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Visual Function And Quality Of Life In A Cohort Of Swedish Children Wi | OPTH – Dove Medical Press

October 24th, 2019 8:46 am

Rezhna Taha,1 Maria Papadopoulou,1,2 Madeleine Zetterberg,1,2 Solveig Oskarsdottir,3 Marita Andersson Grnlund1,2

1Department of Clinical Neuroscience, Institute of Neuroscience and Physiology, Sahlgrenska Academy at the University of Gothenburg, Gothenburg, Sweden; 2Department of Ophthalmology, Sahlgrenska University Hospital, Mlndal, Sweden; 3Department of Pediatrics, Institute of Clinical Sciences, Sahlgrenska Academy at the University of Gothenburg, Gothenburg, Sweden

Correspondence: Rezhna TahaDepartment of Clinical Neuroscience, Institute of Neuroscience and Physiology, Sahlgrenska Academy at the University of Gothenburg, Gothenburg, SwedenTel +46 31 704 093555Fax +46 31 848952Email rezhna.taha.najim@vgregion.se

Purpose: To evaluate quality of life (QoL) in children with juvenile idiopathic arthritis (JIA).Methods: Forty children with a mean age of 7.9 years were included. The children underwent an ophthalmological examination and completed questionnaires on physical function (CHAQ) and vision-related (VR) QoL (EYE-Q).Results: No differences regarding visual acuity (VA), refraction, intraocular pressure or physical or VRQoL were found between those with JIA without (n=33) and those with JIA-associated uveitis (n=7). When comparing physical function measured by CHAQ disability index and JIA subtype, a difference was found; children with polyarthritis scored the worst (p=0.0098). Children with subnormal VA scored worse on EYE-Q compared with those with normal VA (p=0.013). We found correlations between duration of JIA and CHAQ disability index (r=0.42, p=0.0007) and CHAQ well-being (r=0.34, p=0.022).Conclusion: This study indicates the importance of measuring not only physical function but also VRQoL in children with JIA and JIA-associated uveitis.

Keywords: child arthritis, juvenile idiopathic arthritis, PROM, uveitis, quality of life

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Who are Some of the Most Influential Gen Xers in the Biotech Industry? – BioSpace

October 24th, 2019 8:44 am

Its no secret that Generation X is known for so many things, both positive and negative. Some call members of this group, born between the years of 1961 and 1981, slackers who lack motivation. However, this certainly isnt true for many Generation Xers, including those who have shaped and influenced the biotechnology industry we know today. The five scientists on this list collectively made many exciting strides in the field, notably because they had the technological skills to embrace and utilize advances in the realm of computer programming. Strategically combining technology with biology, which is literally the meaning of biotech, allowed the five intellectuals described here to advance healthcare, one small step at a time.

Sebastian Seung, Princeton University

Sebastian Seung, currently a professor at Princeton University's Neuroscience Institute at the Jeff Bezos Center in Neural Dynamics, works to connect neurons in the brain using computer-based technology. He came up with the Connectome Theory, which is best described as being similar to the Human Genome Project, only for the brain. By determining how the brains neurons function and connect with one another, strides may be made in treating diseases like Alzheimers. Since these neural connections seem to change frequently, computer programs are needed to help create the map. On top of his work in the fields of physics and neuroscience, Seung also gives TED talks and authors books.

Hugh Herr, BiOM, Inc.

Not only is Hugh Herr a well-known biotech leader, but hes also an avid mountain climber. In fact, the latter greatly influenced his work in the field. Back in 1982, he was involved in an incident on New Hampshires Mount Washington that tragically led to the amputation of both of his legs below the knee. But Herr refused to let that stop him from doing what he loved, so he founded BiOM, Inc in conjunction with MITs Media Lab, which makes advanced prosthetics for athletes and individuals who want to keep living their lives, despite their physical disabilities. Among other awards, his work has garnered him the 2016 Princess of Asturias Award for Technical & Scientific Research.

Jennifer Doudna, University of California Berkeley

Serving as the University of California Berkleys Li Ka Shing Chancellor Chair Professor in both the Chemistry and Molecular and Cell Biology Departments, Jennifer Doudna specializes in studying a form of genome editing known as CRISPR. In addition, Doudna determined that the Hepatitis C virus synthesizes viral proteins in a unique way, allowing the development of new vaccines for the disease. Her tireless work in the biotech field has led to several awards, ranging from the American Cancer Societys Medal of Honor to Rockefeller Universitys Pearl Meister Greengard Prize.

Karl Deisseroth, Stanford University

Controlling neurons with optogenetics, which uses light to change how those neurons fire in the brain, is the main breakthrough of Karl Deisseroth, currently the D. H. Chen Professor of Bioengineering and of Psychiatry and Behavioral Sciences at Stanford University. Deisseroth has received a number of awards for his research breakthroughs, including the Fresenius Research Prize in 2017, the 2016 Harvey Prize, and the 2019 Warren Alpert Foundation Prize. Hes also a member of the National Academy of Sciences and the National Academy of Medicine.

Nina Tandon, Epibone

Swooping in at the tail end of Generation X, Nina Tandon is the co-founder of Epibone, a professor of Electrical Engineering at Cooper Union, and a fellow in the Lab for Stem Cells and Tissue Engineering at Columbia. Her background includes degrees in BioMedical Engineering and Electrical Engineering. At Epibone, Tandon and her colleagues create bone tissue out of the stem cells. The tissue then is used for bone grafts in patients, greatly reducing the chances of rejection. Among other accolades, she has been named a Global Thinker by Foreign Policy Magazine, as well as TED Senior Fellow.

Its no secret these five creative innovators choose discovery over complacency. Together and individually, each of these Gen-Xers made inspirational and memorable discoveries that shaped the life-changing biotech industry we appreciate today.

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Latest Report on Stem Cell Therapy Market Future Scope Including Top key Players Anterogen Co., Ltd., RTI SurgicalInc. – The World Industry News

October 24th, 2019 8:44 am

Recent research and the current scenario as well as future market potential ofGlobal Stem Cell Therapy Market Set For Rapid Growth, To Reach Around USD 4759.27 Million By 2024.The global Stem Cell Therapy Market report provides significant information about Stem Cell Therapy Market by fragmenting the market into different segments. GlobalStem Cell Therapy MarketReport concentrates on the strong analysis of the present state ofStem Cell Therapy Marketwhich will help the readers to develop innovative strategies that will act as a catalyst for the overall growth of their industry.(Sample Copy Here)This research report segments theStem Cell Therapy Marketaccording to Type, Application and regions.It highlights the information about the industries and market, technologies, and abilities over the trends and the developments of the industries. After deep research and analysis by the experts, they also disclosed the data about the strong contenders contributing in the market growth and expansion and challenging one another in terms of demand, supply, production, value estimation, revenue, and sales.

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As one of the lead news writers at the world industry news, Hirens specialization lies in the science, technology, Health & business domains. His passion for the latest developments in cloud technology, connected devices, nanotechnology, and virtual reality, among others, shines through in the most recent industry coverage he provides. Hirens take on the impact of digital technologies across the technology, health and business domains gives his writing a fresh and modern outlook.

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Multiple Myeloma Experts, Patients, Advocates and Caregivers Team Up to Hike Through Patagonia – Business Wire

October 24th, 2019 8:44 am

CRANBURY, N.J.--(BUSINESS WIRE)--As a part of a fundraising effort by Moving Mountains for Multiple Myeloma (MM4MM), 13 individuals will traverse Patagonias awe-inspiring and incredible landscape from Nov. 9-19. MM4MM is a joint initiative between the Multiple Myeloma Research Foundation (MMRF), CURE Media Group and Celgene. The upcoming climb includes survivors, caregivers, family members, myeloma doctors and team members from the organizing partners.

Since MM4MM began with its first climb in 2016, the program has raised over $2.7 million. All the funds raised go directly to the MMRF to accelerate new treatment options for patients with multiple myeloma.

As a patient founded organization, the MMRF stands together with those who are battling multiple myeloma patients, families, physicians, researchers, and our pharmaceutical partners. This team represents a microcosm of that myeloma community and demonstrates that together, we can collaborate with ever increasing momentum towards a cure, said Paul Giusti, CEO of the Multiple Myeloma Research Foundation. We are thrilled to enter the fifth year of this inspiring program and to have Celgene join us in this effort to raise awareness and critical funds to continue our mission.

The MM4MM team will include four patients living with multiple myeloma:

We are so honored to be a part of yet another hike with the MMRF and Celgene, said Mike Hennessy Jr., president and CEO of MJH Life Sciences, parent company of CURE magazine. This initiative organized by Moving Mountains for Multiple Myeloma not only raises awareness and research funding for multiple myeloma but has brought together the myeloma community to take action and fight for a cure for myeloma patients.

The team will embark on a five-day trek of a lifetime through Patagonia and take on the rewarding and beautiful landscape that includes glaciers, deep valleys and challenging peaks. During this trek, the team will travel through El Chaltn and acclimatize while they experience the mighty range of peaks dominated by Monte Fitz Roy, an 11,020-foot tower with a sheer face of more than 6,000 feet. Next, the team will reach Lago San Martin, where they will traverse the terrain in daily treks, exploring a 10-mile peninsula, climbing to a condor rookery and reaching remote Andean lakes.

Celgene, Cure and the MMRF share an unwavering commitment to improving the lives of patients with multiple myeloma and we are very proud to continue our role in the Moving Mountains for Multiple Myeloma initiative, said Chad Saward, senior director, patient advocacy at Celgene Corp. We are amazed and inspired by all who are participating in this unique awareness program.

To learn more about MM4MM and to donate to multiple myeloma research, click here.

About Moving Mountains for Multiple Myeloma

Moving Mountains for Multiple Myeloma (MM4MM) is a collaboration between CURE Media Group and the Multiple Myeloma Research Foundation (MMRF) to raise awareness and funds for myeloma research. This year, Celgene Corporation and GSK join the effort as sponsors. In addition to Patagonia, the program also led hikes up Mt. Washington and through Iceland in 2019. To date, MM4MM has raised over $2.7 million for myeloma research and included 51 patients with multiple myeloma on 7 climbs. Funds raised go directly to research, supporting the MMRF mission. For more information, visit https://www.themmrf.org/events/.

About Multiple Myeloma

Multiple myeloma (MM) is a cancer of the plasma cell. It is the second most common blood cancer. An estimated 32,110 adults (18,130 men and 13,980 women) in the United States will be diagnosed with MM in 2019 and an estimated 12,960 people are predicted to die from the disease. The five-year survival rate for MM is approximately 50.7%, versus 31% in 1999.

About the Multiple Myeloma Research Foundation

A pioneer in precision medicine, the Multiple Myeloma Research Foundation (MMRF) seeks to find a cure for all multiple myeloma patients by relentlessly pursuing innovations that accelerate the development of precision treatments for cancer. Founded in 1998 by Kathy Giusti, a multiple myeloma patient, and her twin sister Karen Andrews as a 501(c)(3) nonprofit organization, the MMRF has created the business model around cancerfrom data to analytics to the clinic. The MMRF identifies barriers and then finds the solutions to overcome them, bringing in the best partners and aligning incentives in the industry to drive better outcomes for patients. Since its inception, the organization has collected thousands of samples and tissues, opened nearly 100 trials, helped bring 10 FDA-approved therapies to market, and built CoMMpass, the single largest genomic dataset for any cancer. Today, the MMRF is building on its legacy in genomics and is expanding into immune-oncology, as the combination of these two fields will be critical to making precision medicine possible for all patients. The MMRF has raised nearly $500 million and directs nearly 90% of the total funds to research and related programs. To learn more, visit http://www.themmrf.org.

About CURE Media Group

CURE Media Group is the leading resource for cancer updates, research and education. It combines a full suite of media products, including its industry-leading website, CUREtoday.com; innovative video programs, such as CURE Connections; a series of widely attended live events; and CURE magazine, which reaches over 1 million readers, as well as the dynamic website for oncology nurses, OncNursingNews.com, and its companion publication, Oncology Nursing News. CURE Media Group is a brand of MJH Life Sciences, the largest privately held, independent, full-service medical media company in the U.S. dedicated to delivering trusted health care news across multiple channels.

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‘He is not ready to go right now’: John Elway continues to play long game with Drew Lock – The Athletic

October 24th, 2019 8:44 am

John Elway knew this would happen.

After trading up to draft Drew Lock in the second round of the draft, the questions of when his training as the future would start were asked almost immediately.

Lock, the quarterback Elway had eyed throughout the pre-draft process, arrived in Denver after a four-year career in Missouris spread offense. His transition to the Broncos offense an updated version of Mike Shanahans system would take time, from the tiniest details such as his study habits and footwork to the bigger concepts of the offense.

It was paramount, Elway stressed then, that the Broncos remain patient with his development and not rush it.

We believe he has a ton of talent, but we also believe he has a lot left to work on, he said in April. When we look at it, were hoping Drew is the future. But Joe (Flacco) is the starter, is going to be the starter and hes going to...

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CRISPR Therapeutics: At The Top Of My Shopping List – Seeking Alpha

October 24th, 2019 8:44 am

CRISPR Therapeutics (CRSP) has been on my watchlist for a couple of years but the stars never aligned for me to pull the trigger on a buy. Due to some recent updates, I am moving CRSP up to the top of my year-end shopping list and will be stalking an entry point in the coming weeks or months.

I intend to discuss the primary reasons why I have waited for an entry, as well as what has tempted me to start a position. In addition, I discuss some of my leading downside risks associated with this ticker and how I plan to manage my potential position over the next year.

(Image Source: CRISPR)

CRISPR Therapeutics is a leader in gene editing, specifically in the advancement of Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 "CRISPR-Cas9". CRISPR-Cas9 is a gene therapy, however, it is a revolutionary technology that intends to edit the deleterious gene. This is executed by altering explicit genomic sequences that should correct the bad gene or to alleviate the symptoms of the disease. Essentially, CRISPR is attempting to create curative gene therapies for both rare and common diseases.

CRISPR's programs can be divided up into ex vivo, where the company edits cells outside of the body and in vivo, the CRISPR-Cas9-based therapeutic targeted directly into the cells inside the body. CRISPR's leading pipeline programs take aim at hemoglobin related genetic disorders such as transfusion-dependent beta-thalassemia "TDT" and severe sickle cell disease "SCD". Both conditions desperately need a curative therapy because the current standard-of-care is insufficient. In addition to gene editing, the company has several gene-edited allogeneic cell therapy programs, including a few CAR-T candidates for hematological and solid tumor cancers. Furthermore, the company is taking gene-editing into regenerative medicines to address Type 1 diabetes, as well as taking on DMD and few other indications (Figure 1).

Figure 1: CRISPR Pipeline (Source: CRSP)

CRISPR-Cas9 for gene editing was co-invented by Dr. Emmanuelle Charpentier, who is one of CRISPR's founding members. The company now has the rights to Dr. Charpentier's CRISPR-Cas9 technologies, as well as some supplementary technology from other in-licensing efforts. What is more, CRISPR has a strong list of partnerships that will maximize CRISPR-Cas9's ability both clinically and commercially.

CRISPR has always been on my radar and I have admittedly traded the ticker a few times. However, I have been very cautious about investing in the company for a few reasons. One of which, is the fact that it is named after the technology associated with the gene therapies. Although there are other types of gene therapies, CRISPR appears to be the "brand" or term the market associates with all things gene therapy. Consequently, any bad news, setback, or regulatory failure in gene therapy has had a negative impact on the ticker. Essentially, CRISPR becomes a punching bag for anything that goes wrong in the industry. CRSP experienced strong selling pressure following Sarepta Therapeutics (SRPT) receiving a CRL for their DMD gene therapy. As a result, the stock fell from just under $50 a share to ~$43 per share in a few days. Of course, the rest of the industry experienced some selling pressure, but it appears CRSP gets the worst of it. As a result, I have been reluctant to commit to CRSP knowing that it will always be a target for shorts.

Another reason why I have passed on CRSP was due to its early pipeline. The company's pipeline is still mostly pre-clinical, so I would have been investing in science and not a mature product portfolio. Although I am happy to invest in an early-stage biotech/pharma company, I wanted to see if the FDA was willing to allow gene-editing therapies to be put into humans considering the controversy associated with the technology.

My last reason for holding off an investment was the valuation. The company's market valuation appeared to have a premium price on it. Admittedly, the company's high-priced valuation was due to its name and the hype around the gene therapy and not from a fundamental perspective. Although I am sold on the technology, I wasn't going to commit to a long-term investment to an early-stage company that is years away from a pivotal trial, regardless of the technology was from one of the co-founders of the CRISPR-Cas9.

In general, I knew the company was too early in the regulatory process and I expected the ticker to be toyed around with by traders and analysts. Therefore, I wanted to save myself from an unwanted rollercoaster ride of an investment.

The company is now a clinical-stage gene-editing company, which eliminates one of the reasons why I decided to hold off on starting a long-term investment. CRISPR's lead product candidate, CTX001, is ex-vivo CRISPR therapy that is being developed in collaboration with Vertex Pharmaceuticals (VRTX) for TDT or severe SCD. CTX001 is essentially a process that requires the company to engineer a patient's hematopoietic stem cells to increase the levels of fetal hemoglobin "HbF" in red blood cells.

Figure 2: CTX001 SCD (Source: CRSP)

The increase of HbF has the potential to assuage the need for transfusions for TDT and SCD patients.

Figure 3: CTX001 HbF (Source: CRSP)

CRISPR Therapeutics is enrolling in both Phase I/II studies of CTX001 in TDT and severe SCD. The company expects preliminary safety and efficacy data before the year-end. So far, the first TDT patient treated with CTX001 remains "transfusion independent, greater than four months following engraftment."

Now that CXT001 is in the clinic, we can expect several catalysts to come from these programs. Again, I did believe in the technology but the market needs to see that the pre-clinical data will transfer into humans. If the data is promising, we can expect an increase in attention from the market and from Street analysts. The company is expecting to report preliminary results for CTX001 by year-end, so I wouldn't mind having some skin-in-the-game ahead of that readout.

Another reason for a buy is the expanding use of the company's CRISPR-Cas9 technology. In addition to gene editing, the CRISPR-Cas9 has the potential to generate next-gen CAR-T therapies that may be superior to the contemporary autologous CAR-T (Figure 4).

Figure 4: CAR-T Highlights (Source: CRSP)

The company has programs including CTX110, a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting CD19. In pre-clinical mouse studies, the company detected that CTX110 extended the survival in CD19-positive xenograft tumors. In addition to CTX110, the company has CTX120 that is targeting BCMA in multiple myeloma and CTX130 that could target both solid tumors and hematologic malignancies.

Figure 3: CTX110 & CTX120 (Source: CRSP)

In preclinical studies both CTX120 and CTX130 were able to record "complete tumor elimination" in their respective targets.

Figure 4: CTX130 (Source: CRSP)

In addition to cell-therapies, CRISPR is using their ex vivo gene-editing in regenerative medicine by using stem cells to restore or exchange damaged or malfunctioning tissue. Their leading regenerative program is diabetes with their partner, ViaCyte Inc (Figure 5).

Figure 5: In Diabetes (Source: CRSP)

CRISPR has other in vivo disease targets that impact the liver, lung and muscle. These will use the company's lipid nanoparticle-based delivery vehicles "LNPs" and AAV vectors to help deliver the genetic material.

Figure 6: Neuro & Liver (Source: CRSP)

Other notable programs include Glycogen Storage Disease Type Ia "GSDIa", DMD and cystic fibrosis.

Admittedly, I have always seen CRISPR as a pure gene-editing company, however, they have a diverse pipeline and the technology to expand it into numerous other indications and areas of therapy. I expect the company will expand on their research efforts, which should increase the pending value of the company and add some catalysts to the calendar.

The company intends to advance their CRISPR-Cas9 therapeutics both autonomously and in strategic partnerships (Figure 7). The company has closed three major partnerships with Vertex, Casebia/Bayer (OTCPK:BAYRY) and ViaCyte. These partnerships not only provide CRISPR with some support, but they confirm that other reputable companies see CRISPR as a leader in this field.

Figure 7: CRSP Partnerships (Source: CRSP)

As the company expands its pipeline, I anticipate an increase in the number of strategic partnerships and deals. In fact, the company recently announced a partnership with KSQ Therapeutics to in-license IP to advance their cell therapy programs.

My final reason for stalking an entry is the improved risk/reward scenario. The stock appears to have lost some momentum and has had a relatively flat 2019. The stock has recently experienced a strong pull-back, yet, the company has made substantial progress in both the pipeline and strategic collaborations.

Figure 8: CRSP Daily (Source: Trendspider)

This recent pull-back has cut the market cap down to ~$2B which I believe is fair considering the long-term outlook for the company.

Figure 9: CRSP Valuation (Source: Seeking Alpha)

Looking at the CRSP's annual revenue estimates (Figure 10), we can see the Street expects the company to have relatively flat revenues for the next couple of years and will start to experience strong revenue growth in the second half of the next decade. This rapid growth is expected to drop the forward price-to-sales ratio below 1x at some point in 2027. The sector's average price-to-sales is about 5x, so that would equal ~$13B market cap in 2027.

Figure 10: CRSP Annual Revenue Estimates (Source: Seeking Alpha)

I understand this is years away, but I expect these estimates to only improve as the company continues to add pipeline programs and other strategic partnerships. Indeed, I could wait another year or two to further reduce my risk and cost for time. However, I don't want to be left out of this ticker if the company starts reporting curative results in multiple programs. What would happen if the company demonstrates the potential to cure Type 1 diabetes? Do you think it will be trading at a $2B market cap? What about if they cure cystic fibrosis? Sickle Cell? Eliminate renal cancer with an autologous CAR-T therapy? I believe the company would be undervalued at $2B even if they only cured one of these indications. Assuming the company has at least one winner in their current pipeline, I have to say the current risk/reward is enticing enough for me to start a speculative position.

I believe the biggest threat to CRISPR comes from the impressive competition from other gene therapy companies and institutions. These include companies that are working on CRISPR-Cas9 technology such as Intellia Therapeutics (NTLA) and most notably Editas Medicine (EDIT). Other gene-editing companies such as bluebird bio (BLUE), Sangamo Therapeutics (SGMO), Cellectis (CLLS), Precision BioSciences (DTIL), and Allogene Therapeutics (ALLO) use other gene-editing platforms. There is a multitude of gene therapy companies that I didn't mention, but I think you get my point. Some of these companies are not in the same league as CRISPR, however, I believe CRISPR's biggest threat is something that is currently in a theoretical stage or perhaps hasn't been thought of yet. As gene therapy products begin to hit the market and start dethroning current standard-of-care therapies, I expect big pharma to start devoting a larger percentage of their R&D to gene therapies. As a result, more universities and institutions will start pioneering new CRISPR tech and perhaps another level of gene editing. If they are successful, they will most likely dethrone CRISPR-Cas9 as cutting-edge technology.

CRISPR is a developmental biotech company, so of course, the financials will be an issue until the company has a product on the market. At the end of Q2, the company had $427.9M in the bank. The company's R&D expenses were $39.5M for Q2, which led to a net loss of $53.7M. Usually, I would consider $428M to be a strong cash position for pre-revenue biotech, but I don't think we are seeing peak cash-burn. The company has several programs that haven't even hit the clinic yet, so we can expect that cash position to melt rapidly in the coming years. In fact, the company recently secured a $200M at-the-market sale of common stock, so we should expect the company to tap the market when they need to. Even then, investors need to accept the strong possibility the company will execute secondary offerings in the future.

CRISPR Therapeutics has evolved over the past couple of years and so has my view of a potential investment. The company's premium name brought a premium price, which generated a substantial amount of attention from traders who have created detached or arbitrary valuations for the ticker. Now, the company has moved into the clinic and will be generating data that could reveal curative level results for dreadful diseases and conditions. If CRISPR is able to gain regulatory approval, the product could replace the current standard-of-care for the indication and eventually dominate as patients demand a cure, rather than dealing with a lifetime of medications or procedures.

Due to this potential, I am moving CRSP up to the top of my shopping list as we close out 2019. I expect many of the speculative biotech names to experience end-of-the-year tax selling, so I am going to keep a close eye on the charts to see if I can get a nice discount to establish a large position. In the meantime, I rely on the chart's technicals to find an entry. Returning to figure 8, we can see the stock has returned to its previous trading range after a month of selling pressure. If the share price is able to hold this area over the next couple of weeks, I will look to click the buy button on a low volume day. If the stock breaks below $35, I will most likely hold off on a buy until the end of the year. Once I have established a position, I will look to add to my position following data readouts with a goal to have a full-sized position by the end of 2021.

Disclosure: I am/we are long SRPT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: I am also long BLUE.

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CRISPR Therapeutics: At The Top Of My Shopping List - Seeking Alpha

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Ewing sarcoma: Causes, symptoms, and treatment – Medical News Today

October 24th, 2019 8:44 am

Ewing sarcoma is a form of bone cancer that usually affects children and adolescents.

Ewing sarcoma can be very aggressive, but the cells tend to respond well to radiation therapy. Ideally, doctors will diagnose the cancer before it has spread.

According to the National Library of Medicine, an estimated 250 children in the United States receive a diagnosis of Ewing sarcoma each year.

In this article, learn more about Ewing sarcoma, including the symptoms, causes, and treatment options.

Ewing sarcoma is a rare type of cancer that usually starts in the bone typically in the pelvis, chest wall, or legs and occurs mostly in children and teenagers.

Dr. James Ewing first described Ewing sarcoma in 1921. He identified cancer cells that looked different than the cells in osteosarcoma, another type of bone tumor.

Doctors may also refer to this cancer type as the Ewing family of tumors. These tumors have distinct cells that usually respond well to radiation treatments.

This rare cancer type accounts for just 1.5% of all childhood cancers and is the second most common bone cancer type in childhood, after osteosarcoma.

Although researchers are unsure why some people develop Ewing sarcoma, they have identified mutations in certain genes in the tumor cells that cause this cancer.

These include the EWSR1 gene on chromosome 22 and the FLI1 gene on chromosome 11.

These genetic mutations occur spontaneously during a person's lifetime. The individual does not inherit them from a family member.

There are no known risk factors for Ewing sarcoma that make one person more likely than another to develop this cancer.

Ewing sarcoma can cause the following symptoms:

An estimated 87% of Ewing sarcomas are sarcoma of the bone. The other types form in the soft tissues, such as cartilage, that surround the bones.

Ewing sarcoma can spread to other areas of the body. Doctors call this process metastasis.

Areas that the cancer can spread to include other bones, bone marrow, and the lungs.

Doctors categorize Ewing sarcoma as one of three types according to its extent:

Before diagnosing Ewing sarcoma, a doctor will take a person's full medical history and ask them what symptoms they are having, when they noticed them, and what makes them better or worse. They will also perform a thorough physical exam, focusing on the area of concern.

A doctor will usually recommend an imaging study to view the bone or bones. These tests include:

If it looks as though a tumor may be present, a doctor will perform a biopsy, which involves taking a sample of bone tissue. They will send this tissue to a laboratory, where a specialist called a pathologist will check it for the presence of cancerous cells.

A doctor may also order blood tests, a bone marrow biopsy, and other scans when necessary. These tests can help determine whether the cancer has spread to other locations.

A doctor will work with a team of cancer specialists and surgeons to recommend and implement particular treatments.

Possible treatments for Ewing sarcoma include:

Doctors may use a combination of treatments depending on how far the cancer has spread and a person's overall health.

Research into new treatments for Ewing sarcoma is ongoing. Some doctors may inform their patients about clinical trials, which help test new treatments.

Possible complications of Ewing sarcoma include:

If Ewing sarcoma has spread to other areas of the body, it can be life threatening. For this reason, it is vital for a doctor to evaluate any symptoms as quickly as possible.

According to the American Academy of Orthopaedic Surgeons, an estimated two-thirds of people in whom cancer has not spread to other areas of the body survive at least 5 years after their diagnosis.

People who are more likely to have positive outcomes include those who have:

The likelihood of successful treatment is different for every individual, so people should speak to a doctor about their or their child's expected outlook.

Ewing sarcoma is a rare type of cancer that mostly affects young people.

When doctors detect it early enough, the condition usually responds well to treatment.

Anyone who notices signs or symptoms of Ewing sarcoma, such as a bone that breaks for no apparent reason or a painful lump or swelling, should speak to a doctor.

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Global Wound Care Market Outlook and Forecasts, 2018 & 2019-2024 – ResearchAndMarkets.com – Business Wire

October 24th, 2019 8:44 am

DUBLIN--(BUSINESS WIRE)--The "Wound Care Market - Global Outlook and Forecast 2019-2024" report has been added to ResearchAndMarkets.com's offering.

The global wound care market is growing at a CAGR of over 5% during the forecast period 2018-2024

The adoption of wound care biologics is augmenting the growth of the global wound care market. The commercial availability of a wide array of wound biologics is likely to encourage many end-users to use them in treating wounds as they are clinically proven, safe, and effective than other products.

The growing incidence of infections caused in lesions is another factor accelerating the growth of anti-microbial dressings market segment. Anti-microbial agents such as chlorhexidine, maggots, silver, iodine, and honey are increasingly becoming important in the global wound care market. Therefore, the incorporation of anti-microbial agents in wound dressing products is improving clinical outcomes for the treatment of wounds, thereby driving the global wound care market.

Key Vendor Analysis

The global wound care market is highly competitive and dynamic characterized by the presence of several global, regional, and local vendors, offering a diverse range of products and devices for treating various acute and chronic wounds. There are approximately more than 400 vendors providing a wide array of wound care products worldwide. Multiple product launches, strategic acquisitions, and differentiated products have fueled the growth in recent years.

New product launches and strategic acquisitions, collaborations will be crucial for companies to maintain revenue growth in the coming years. Large and diversified companies account for nearly 48% of the market share. Also, the majority of key players have demonstrated consistent growth in the last three years. Moderate to the high growth of major players will continue to boost the market growth. In addition, both established and emerging players are developing or commercializing wound care products and devices.

Major players are focusing on technological innovations, thereby investing substantial amounts on R&D activities for offering advanced wound care products such as advanced wound dressings, skin grafts, and wound biologics.

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage

4.1 Market Definition

4.2 Base Year

4.3 Scope of the study

4.4 Market Segments

5 Report Assumptions & Caveats

5.1 Key Caveats

5.2 Currency Conversion

5.3 Market Derivation

6 Market at a Glance

7 Introduction

7.1 Wound Care: An Overview

7.1.1 Background

7.1.2 Wound Care for Acute & Chronic Wounds

7.1.3 Wound Care: Market Snapshot

8 Market Dynamics

8.1 Market Growth Enablers

8.1.1 Increasing Prevalence of Acute & Chronic Wounds

8.1.2 New Product Approvals/Launches

8.1.3 Increasing Number of Surgical Procedures

8.2 Market Growth Restraints

8.2.1 High Cost of Advanced Wound Care Products & Devices

8.2.2 Limitations & Complications with Wound Care Products & Devices

8.2.3 Intense Competition & Pricing Pressure

8.2.4 Shortage of Resources for Wound Care Treatments

8.3 Market Opportunities & Trends

8.3.1 Focus on Development & Commercialization of Wound Biologics

8.3.2 High Demand for Anti-microbial Wound Dressing Products

8.3.3 Growing Popularity of Natural Surgical Sealants

8.3.4 Emergence of Stem Cell Therapy For Wound Healing

9 Global Wound Care Market

9.1 Market Overview

9.2 Market Size & Forecast

9.3 Five Forces Analysis

10 By Product Type

10.1 Market Snapshot & Growth Engine

10.2 Market Overview

11 Advanced Wound Care Products

11.1 Market Snapshot & Growth Engine

11.2 Market Overview

11.3 Advanced Wound Care Segmentation by Product Type

11.4 Advanced Wound Dressings

11.5 Wound Therapy Devices

11.6 Wound Care Biologics

12 Traditional Wound Care Products

12.1 Market Overview

12.2 Market Size & Forecast

13 Sutures & Stapling Devices

13.1 Market Snapshot & Growth Engine

13.2 Market Overview

13.3 Market Size & Forecast

13.4 Segmentation by Product Type

13.5 Sutures

13.6 Stapling Devices

14 Hemostats & Surgical Sealants

14.1 Market Snapshot & Growth Engine

14.2 Market Overview

14.3 Market Size & Forecast

14.4 Segmentation by Product Type

14.5 Hemostats

14.6 Surgical Sealants

15 By Wound Type

15.1 Market Snapshot & Growth Engine

15.2 Market Overview

15.3 Acute Wounds

15.4 Chronic Wounds

16 By End Users

16.1 Market Snapshot & Growth Engine

16.2 Market Overview

16.3 Hospitals & Specialty Wound Care Clinics

16.4 Long-term Care Facilities

16.5 Home Healthcare

16.6 Others

17 By Geography

17.1 Market Snapshot & Growth Engine

17.2 Market Overview

Key Company Profiles

Other Prominent Vendors

For more information about this report visit https://www.researchandmarkets.com/r/gxho7k

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Molecular Diagnostics Market is Anticipated to Expand at a CAGR of 11.6% from 2016 to 2025 – Health News Office

October 24th, 2019 8:44 am

The ELECTRONIC HEALTH RECORDS (EHR) market research report added by Report Ocean, is an in-depth analysis of the latest trends, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research study provides market introduction, ELECTRONIC HEALTH RECORDS (EHR) market definition, regional market scope, sales and revenue by region, manufacturing cost analysis, Industrial Chain, market effect factors analysis, ELECTRONIC HEALTH RECORDS (EHR) market size forecast, 100+ market data, Tables, Pie Chart, Graphs and Figures, and many more for business intelligence.

The global EHR market was valued at $23,592 million in 2016, and is expected to reach $33,294 million by 2023, growing at a CAGR of 5.0% from 2017 to 2023.

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ELECTRONIC HEALTH RECORDS (EHR) Market: Competitive Analysis by Key Players:

AdvancedMD, Inc.Allscripts Healthcare Solutions, Inc.Cerner CorporationComputer Programs and Systems, Inc.CureMD CorporationeClinicalWorksEpic Systems CorporationGeneral Electric CompanyGreenway Health, LLCQuality Systems, Inc.

The Global ELECTRONIC HEALTH RECORDS (EHR) Market also explains the competitive landscape among the major key players of the market, based on various parameters, such as:

ELECTRONIC HEALTH RECORDS (EHR) Market Segments:

By Product (Cloud-Based Software and Server-Based/On-Premise Software), Type (Inpatient EHR and Ambulatory EHR), Application (Clinical Application, Administrative Application, Reporting in Healthcare System, Healthcare Financing, and Clinical Research Application), and End User (Hospitals, Clinics, Specialty Centers, and Other End Users)

ELECTRONIC HEALTH RECORDS (EHR) Market: Insights

The global EHR market was valued at $23,592 million in 2016, and is expected to reach $33,294 million by 2023, growing at a CAGR of 5.0% from 2017 to 2023.

An electronic health record (EHR) is a digital version of the paper chart of a patient. It contains all the data related to a patients medical history including medications, diagnosis, immunization dates, treatment plans, radiology images, allergies, and test results from laboratories. These also allow access to evidence-based tools used by healthcare providers to make decisions about a patients treatment. EHRs have also helped to streamline and automate the workflow in a healthcare setting.

Rise in adoption of EHR, increased use of cloud based EHR software, and rapid surge in aging population and subsequent rise in the number of chronic diseases drive the market growth. However, high cost of EHR and increase in concerns regarding the patient data safety & security are expected to impede the market growth. Moreover, huge market potential in the developing regions are expected to offer further opportunities for market growth during the forecast period.

The report segments the market based on product, type, application, end user, and region. Based on product, the market is bifurcated into cloud-based software and server-based/on-premise software. The type segment is categorized into inpatient EHR and ambulatory EHR. Based on application, the market is segmented into clinical application, administrative application, reporting in healthcare system, healthcare financing, and clinical research application. EHRs may be used in hospitals, clinics, specialty centers, and other medical settings. Based on region, the market is analyzed across North America (U.S., Canada, and Mexico), Europe (Germany, France, UK, Spain, and rest of Europe), Asia-Pacific (China, Japan, Australia, India, and rest of Asia-Pacific), and LAMEA (Brazil, South Africa, Saudi Arabia, and rest of LAMEA).

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Furthermore, the years considered for the study are as follows:

Historical year 2013-2017

Base year 2018

Forecast period** 2019 to 2025 [** unless otherwise stated]

Regional Analysis:

The market research study offers in-depth regional analysis along with the current market scenarios. The major regions analyzed in the study are:

Key highlights and important features of the Report:

Overview and highlights of product and application segments of the global ELECTRONIC HEALTH RECORDS (EHR) Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Explore about Sales data of key players of the global ELECTRONIC HEALTH RECORDS (EHR) Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the ELECTRONIC HEALTH RECORDS (EHR) Market.

Explore about gross margin, sales, revenue, production, market share, CAGR, and market size by region.

Describe ELECTRONIC HEALTH RECORDS (EHR) Market Findings and Conclusion, Appendix, methodology and data source;

Research Methodology:

The market research was done by adopting various tools under the category of primary and secondary research. For primary research, experts and major sources of information have been interviewed from suppliers side and industries, to obtain and verify the data related to the study of the Global ELECTRONIC HEALTH RECORDS (EHR) Market. In secondary research methodology, various secondary sources were referred to collect and identify extensive piece of information, such as paid databases, directories and annual reports and databases for commercial study of the Global ELECTRONIC HEALTH RECORDS (EHR) Market. Moreover, other secondary sources include studying technical papers, news releases, government websites, product literatures, white papers, and other literatures to research the market in detail.

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There are 15 Chapters to display the Global ELECTRONIC HEALTH RECORDS (EHR) Market:

Chapter 1, to describe Definition, Specifications and Classification of Global ELECTRONIC HEALTH RECORDS (EHR), Applications of, Market Segment by Regions;Chapter 2, to analyze the Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, to display the Technical Data and Manufacturing Plants Analysis of , Capacity and Commercial Production Date, Manufacturing Plants Distribution, Export & Import, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, to show the Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, to show the Regional Market Analysis that includes United States, EU, Japan, China, India & Southeast Asia, Segment Market Analysis (by Type);Chapter 7 and 8, to explore the Market Analysis by Application Major Manufacturers Analysis;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type, Market Trend by Application;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, to analyze the Consumers Analysis of Global ELECTRONIC HEALTH RECORDS (EHR) by region, type and application;Chapter 12, to describe ELECTRONIC HEALTH RECORDS (EHR) Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, to describe ELECTRONIC HEALTH RECORDS (EHR) sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

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Molecular Diagnostics Market is Anticipated to Expand at a CAGR of 11.6% from 2016 to 2025 - Health News Office

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NIH, Gates Foundation aim to bring genetic cures to the poor – STAT

October 24th, 2019 8:41 am

The National Institutes of Health and the Bill and Melinda Gates Foundation will together invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV with an attribute even rarer in the world of genetic medicine than efficacy, the groups announced on Wednesday: The cures, they vowed, will be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

The effort reflects growing concerns that scientific advances in genetic medicine, both traditional gene therapies and genome-editing approaches such as CRISPR, are and will continue to be prohibitively expensive and therefore beyond the reach of the vast majority of patients. Spark Therapeutics Luxturna, a gene therapy for a rare form of blindness, costs $425,000 per eye, for instance, and genetically engineered T cells (CAR-Ts) to treat some blood cancers cost about the same.

With CRISPR-based treatments already being tested in clinical trials for sickle cell disease, the blood disorder beta thalassemia, and another form of blindness, and with additional CRISPR treatments in development, scientists, ethicists, and health policy experts have grown increasingly concerned that the divide between haves and have-nots will grow ever-wider.

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Gene-based treatments are largely inaccessible to most of the world by virtue of the complexity and cost of treatment requirements, which currently limit their administration to hospitals in wealthy countries, the NIH said in a statement. To help right that, its collaboration with the Gates Foundation aims to develop curative therapies that can be delivered safely, effectively and affordably in low-resource settings.

Scientists whose research focuses on gene-based cures welcomed the infusion of funding and the recognition that genetic cures are on track to be unaffordable to the majority of patients. But they noted one irony. The most effective sickle cell drug, hydroxyurea, has hardly even been studied in sub-Saharan Africa, let alone made widely available. Yet a 2019 study found that giving children the drug cut their death rate by two-thirds and halved the pain crises that are common in sickle cell disease, caused by misshapen red blood cells that cannot flow through blood vessels.

The NIH-Gates collaboration is tremendously exciting and has the potential to have a great impact on sickle cell disease in sub-Saharan Africa, said Dr. Vijay Sankaran of the Dana-Farber/Boston Childrens Cancer and Blood Disorders Center, who has done pioneering research on genetic cures for the disease. But my hesitation is that even the inexpensive therapies we have today, such as hydroxyurea, are largely unavailable there. The question is, how do we best approach this disease, with therapies that are working today or with genetic therapies that might work?

The same concerns surround HIV. Very inexpensive less than $100 per year in the U.S. antiretroviral drugs can keep the virus in check, but only 67% of HIV-positive adults and 62% of HIV-positive in children in east and southern Africa are estimated to be on antiretroviral treatment.

The new collaboration aims to move gene-based cures into clinical trials in the U.S. and countries in sub-Saharan Africa within the next seven to 10 years, and to eventually make such treatments available in areas hardest hit by sickle cell disease and HIV/AIDS. The idea is to focus on access, scalability, and affordability to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries, NIH Director Francis Collins said in a statement. We aim to go big or go home. But the challenge is enormous, he told reporters on Wednesday: Im not going to lie. This is a bold goal.

An estimated 95% of the 38 million people with HIV live in the developing world, with 67% in sub-Saharan Africa. Up to 90% of children with sickle cell disease in low-income countries die before they are 5 years old. In the U.S., the life expectancy for people with sickle cell disease is in the low 40s.

The NIH and the Gates Foundation will fund research to identify potential gene-based cures for sickle cell and HIV, and also work with groups in Africa to test those cures in clinical trials.

The science of genetic cures for both diseases is within reach, experts say. CRISPR Therapeutics and Vertex (VRTX) are already running a clinical trial for sickle cell disease, using the CRISPR genome editor to do an end-run around the disease-causing mutation in the hemoglobin gene: The therapy releases the brake on red blood cells production of fetal hemoglobin, whose production shuts off in infancy but which does not have the sickling damage of adult hemoglobin.

Developing effective, safe genetic cures for sickle cell and HIV would be only a first step, however. As currently conceived, such therapies require advanced medical facilities to draw blood from patients, alter their cells genomes in a lab, give the patients chemotherapy to kill diseased blood-making cells, and then perform whats essentially a bone marrow transplant, followed by monitoring patients in a hospital for days to prevent infection and provide intensive medical support, said Dr. Dan Bauer, a sickle cell expert at Boston Childrens.

He called the NIH-Gates effort terrific, but cautioned that delivering advanced gene therapies requires tremendous effort, extended hospitalization, and large supplies of blood products. All of those requirements mean that even if a CRISPR-based cure for sickle cell disease or HIV were provided at cost, there will still be barriers to access.

Recognizing that, Collins said, a genetic cure would have to be given directly into patients (in vivo), presumably through an infusion, rather than by treating blood or other cells removed from patients and genetically transformed in a lab (ex vivo). That could avoid the resources needed for and the complications that can occur with ex vivo therapies, said Sankaran, who has discussed the approach with Gates officials.

This story has been updated with additional comments.

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Occurrence, Genetic Diversities And Antibiotic Resistance Profiles Of | IDR – Dove Medical Press

October 24th, 2019 8:41 am

Stephen Abiola Akinola,1 Mulunda Mwanza,2 Collins Njie Ateba1

1Antimicrobial Resistance and Phage Bio-Control Group, Department of Microbiology, Faculty of Natural and Agricultural Sciences, North West University, Mmabatho 2745, South Africa; 2Center for Animal Health Studies, Faculty of Natural and Agricultural Sciences, North West University, Mmabatho 2745, South Africa

Correspondence: Collins Njie AtebaAntimicrobial Resistance and Phage Bio-control Group, Department of Microbiology, Faculty of Natural and Agricultural Sciences, North West University, Private Bag X2046, Mmabatho 2745, South AfricaTel +27 18 489 2720Email collins.ateba@nwu.ac.za

Purpose: Contamination with Salmonella on food products and poultry in particular has been linked to foodborne infections and/or death in humans. This study investigated the occurrence, genetic diversities and antibiotic resistance profiles of Salmonella strains isolated from chickens.Patients and methods: Twenty each duplicate faecal swab samples were collected from five different poultry pens of broilers, layers and indigenous chickens in the North-West Province, South Africa. Isolates identities were confirmed through amplification and sequence analysis of 16S rRNA and the invA gene fragments after which phylogenetic tree was constructed. Salmonella enteritidis (ATCC:13076TM), Salmonella Typhimurium (ATCC:14028TM) and E. coli (ATCC:259622TM) were used as positive and negative controls, respectively. The serotypes of Salmonella isolates were determined. Antibiotic-resistant profiles of the isolates against eleven antimicrobial agents were determined.Results: Eighty-four (84%) of representative isolates possessed the invA genes. The percent occurrence and diversity of Salmonella subspecies in chickens were 1.8130.9% and was highest in Salmonella enterica subsp. enterica. Notably, the following serotypes Salmonella bongori (10.09%), Salmonella Pullorum (1.81%), Salmonella Typhimurium (12.72%), Salmonella Weltevreden, Salmonella Chingola, Salmonella Houten and Salmonella Bareily (1.81%). Isolates (96.6%) displayed multidrug resistance profiles and the identification of isolates with more than nine antibiotic resistance was a cause for concern.Conclusion: This study indicates that isolates had pre-exposure histories to the antibiotics tested and may pose severe threats to food security and public health.

Keywords: Salmonella, diversity, antimicrobial resistance, phylogenetic, chickens

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Police were cracking cold cases with a DNA website. Then the fine print changed. – NBC News

October 24th, 2019 8:41 am

In April 2018, California authorities revealed that theyd used a novel investigative technique to arrest a man they called the Golden State Killer, a serial murderer whod escaped capture for decades.

For the first time, police had submitted DNA from a crime scene into a consumer DNA database, where information about distant relatives helped them identify a suspect.

The announcement kindled a revolution in forensics that has since helped solve more than 50 rapes and homicides in 29 states.

But earlier this year, that online database changed its privacy policy to restrict law enforcement searches, and since then, these cold cases have become much harder to crack. The change is allowing some criminals who could be identified and caught to remain undetected and unpunished, authorities say.

There are cases that wont get solved or will take longer to solve, Lori Napolitano, the chief of forensic services at the Florida Department of Law Enforcement, said.

The switch was imposed by GEDmatch, a free website where people share their DNA profiles in hopes of finding relatives. The company had faced criticism for allowing police to search profiles without users permission, and decided that it would rather make sure members understood explicitly how investigators were using the site. So, it altered its terms of service to automatically exclude all members from law enforcement searches and left it to them to opt in.

Overnight, the number of profiles available to law enforcement dropped from more than 1 million to zero. While the pool has grown slowly since then, as more people click a police-shield icon on GEDmatch allowing authorities to see their profile, cases remain more difficult to solve, investigators say.

CeCe Moore, a leading specialist in using DNA evidence and family trees to identify criminal suspects a method known as investigative genetic genealogy depends on GEDmatch for her work. After entering a suspects DNA profile into the site, she reviews the results and assesses the likelihood of law enforcement being able to determine the suspects identity. She then scores each case from 1 to 5, 1 being a sure thing and 5 a long shot.

Im giving a lot more fives than I used to, said Moore, who helped solve several cases using GEDmatch before the site changed its terms of service, including the 1987 killing of a young Canadian couple, the 1988 murder of an 8-year-old Indiana girl and the 1992 rape and strangulation of a Pennsylvania schoolteacher.

This sharp drop in the usefulness of a promising technology has sparked an effort by law enforcement authorities and researchers like Moore to convince the public to take action. These groups hope to persuade more Americans to obtain their DNA profiles from direct-to-consumer genetic testing companies most of which have large databases but dont allow law enforcement searches and share them publicly, including with law enforcement, on databases like GEDmatch. One direct-to-consumer company, FamilyTreeDNA, allows law enforcement to search its database, but charges for it and limits results.

Some people are reluctant, worried that their DNA profiles will be hacked or used against their wishes, whether in the pursuit of a criminal or in the sale of data to health care companies. There are also concerns that DNA sharing will lead to the end of anonymity.

But law enforcement authorities and genetic sleuths who work with them argue that there is greater public good in helping to keep killers and rapists off the streets.

In the interest of public safety, dont you want to make it easy for people to be caught? said Colleen Fitzpatrick, a genetic genealogist who co-founded the DNA Doe Project, which identifies unknown bodies, and runs IdentiFinders, which helps find suspects in old crimes. Police really want to do their job. Theyre not after you. They just want to make you safe.

To illustrate those points, investigators tell the story of Angie Dodge.

Dodge, 18, was raped and murdered in 1996 in her Idaho Falls, Idaho, apartment. A year later, a man confessed to the crime, and although he later recanted and his DNA didnt match that of semen left on Dodges body, he was convicted of participating in the killing and sentenced to life in prison.

Dodges mother grew convinced that the prisoner, Christopher Tapp, was not her daughters killer. She pressed authorities to reopen the case. In 2017, Tapp was freed in a deal with prosecutors in which his conviction of aiding and abetting the murder remained.

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So did the question of who left their DNA at the crime scene.

Almost a year later, California authorities said theyd used genetic genealogy to catch the Golden State Killer.

The announcement generated a surge of interest in the technique, as genetic genealogists teamed up with private companies to sell their services to law enforcement. (Public crime labs are not equipped to do the kind of DNA analysis required, and police generally arent fluent in methods used to build family trees.) Parabon NanoLabs was the first, hiring Moore to run its genetic genealogy services. Idaho Falls police asked the company to try it.

Parabon submitted the Dodge suspects DNA profile into GEDmatch in May 2018, but the DNA was so degraded that, even with more than 1 million profiles to compare against, the connections were sparse. Moore decided that genetic genealogy wouldnt work and declined to take up the case.

But Dodges mother, Carol, begged Moore to keep trying. Moore relented and examined the connections more closely. With help from her team of genetic genealogists, she explored a series of leads that didnt pan out. They kept at it for months, eventually discovering a new branch of the suspects family tree and a potential suspect.

Police followed that man, collecting a cigarette butt he discarded and using it to obtain his DNA. It matched the crime scene profile, and in May 2019 Brian Leigh Dripps confessed, police said. A few weeks later, Tapp was exonerated. Dripps is awaiting trial.

Moore chronicled that search at a recent gathering of genetic researchers, investigators, prosecutors and lab technicians in Palm Springs, California. If she had been working on the Dodge case after GEDmatch limited access to its database, she told attendees of the International Symposium on Human Identification, this case would not have been able to be solved by genetic genealogy.

She said she understood why GEDmatchs owners made the decision, but the result was allowing some violent criminals to remain free for longer than they would have been with the full power of genetic genealogy. She pleaded with her audience to take DNA tests and upload their profiles into GEDmatch.

We dont want this very valuable tool to slip out of our hands, Moore said.

Curtis Rogers didnt ask for this.

Rogers, 81, works in Florida as a court-appointed guardian for the elderly. He founded GEDmatch as a free public service in 2010 after being inspired by his own experience connecting with people who shared his last name. He partnered with a computer programmer who wrote software that made it easy for people to find relatives through certain shared pieces of genetic material. The site became popular among professional and amateur genealogists, and as direct-to-consumer genetic testing services grew, GEDmatch enabled people to compare their DNA profiles in a single place.

Rogers knew little of law enforcements interest in his website until the Golden State Killer announcement. The news upset Rogers and some members. But he eventually accepted the sites role in solving violent crimes where other forensic techniques including searches for matches in criminal DNA databases had failed.

I am not totally comfortable with GEDmatch being used to catch violent criminals but I doubt it would be possible to prevent it, Rogers told NBC News last year. I feel it is important to make sure all our users are educated to the possible uses of GEDmatch so they can make up their own minds.

As law enforcement searches of his site surged, Rogers imposed a few restrictions. He allowed investigators to pursue leads on homicides and rapes, but not less serious crimes like assaults.

Then, late last year, police in Utah asked Rogers to use the site to investigate an attack on an elderly church organist, who was seriously hurt but survived. Rogers agreed, and police used GEDmatch to identify a 17-year-old suspect, who was arrested in April.

But that bending of Rogers own rules on how police could use GEDmatch triggered a backlash that led him to change the sites terms of service. On May 18, all GEDmatch members were removed from law enforcement searches and offered a chance to opt in if they wanted to be included.

Ethicists said the decision ensured that users would be properly informed about how their profiles would be used.

People using genetic genealogy databases for their own purposes never anticipated this kind of access to their genetic information or that information being used to identify people theyre related to, said Amy McGuire, director of the Center for Medical Ethics and Health Policy at the Baylor College of Medicine.

There is a genuine tension between wanting to protect consumers and be respectful of their wishes and recognizing that working with law enforcement provides a social benefit, she said.

A Baylor College of Medicine survey last year found 91 percent of respondents favored law enforcement using consumer DNA databases to solve violent crimes, and 46 percent for nonviolent crimes.

But with fewer links from GEDmatch to examine, investigators now have to spend more time seeking connections that could lead them to a killer. That means chasing down more leads, or asking innocent distant relatives to submit their DNA for genetic testing in hopes that their profile will help fill out the family tree. That kind of target testing raises its own set of privacy concerns.

You only need to look at the pace of press releases since the time of the Golden State Killer and see that there was virtually one every week, if not more, and the pace of those cases being solved has reduced itself, said Anne Marie Schubert, the district attorney in Sacramento County, California, where she oversaw the Golden State Killer investigation.

The limited GEDmatch access ultimately means higher costs for law enforcement who turn for help to Parabon, which dominates the private market for such work, or FamilyTreeDNA, one of the earliest direct-to-consumer genetic testing companies.

It also means that more cases might remain unsolved.

If I had a larger team, we would certainly try more cases, Moore said. But because agencies are paying Parabon, we dont want them to waste valuable resources or give false hopes.

Rogers says he has done the right thing for his members, and for the field he loves.

We are here for genealogists, not for law enforcement, he said. On the other hand, law enforcement is here to stay. I feel a big obligation to make sure its used properly. I dont want some half-cocked law enforcement person to do something that creates a story and ruins everything for everyone.

Rogers has sent emails to members urging them to allow law enforcement to search their profiles, linking to a video message from a relative of one of the Golden State Killers victims.

Many of these families have suffered for decades. They need your support, he wrote in an email to members. We hope you will encourage others who have been genealogically DNA tested to also add their information. We believe it is the caring thing to do.

So far, Rogers said, 181,000 members have opted in. Thats far from the critical mass of 1 million that some researchers say is needed to solve cold cases with regularity. It could be many months, and perhaps years, before GEDmatchs law enforcement-accessible database reaches that size.

Im sorry we had to do this. However, I feel very strongly that when we bit the bullet and did what we did, we set the whole future on a much stronger base, Rogers said of genealogy. Two or three years from now, this whole thing will be forgotten.

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Association study of genetic variants of the ANGPTL3 gene and suscepti | NDT – Dove Medical Press

October 24th, 2019 8:41 am

Qi Gong,1,* Liping Ye,2,* Huiwen Gui,1 Jing Liu,1 Huanyin Li,1 Qian Sun1

1Department of Neurology, Minhang Branch, Zhongshan Hospital, Fudan University, Shanghai 201199, Peoples Republic of China; 2Nursing Department, Minhang Branch, Zhongshan Hospital, Fudan University, Shanghai 201199, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Huanyin LiDepartment of Neurology, Minhang Branch, Zhongshan Hospital, Fudan University, 170 Shenlong Road, Minhang District, Shanghai 201199, Peoples Republic of ChinaTel/Fax +86 216 492 3400Email li_huanyin@sina.com

Background: Stroke ranks as the third-leading cause of years of life lost worldwide. ANGPTL3 plays important roles in lipid metabolism, atherosclerosis, and occurrence of stroke. The purpose of this study was to evaluate associations of genetic variants in the ANGPTL3 gene with ischemic stroke (IS) risk.Methods: A casecontrol study was conducted to evaluate the associations of tag single-nucleotide polymorphisms (SNPs) of the ANGPTL3 gene and risk of IS, as well as serum lipid levels. Dual-luciferase reporter assays in the HEK293T cell line was conducted to evaluate the promoter activity of ANGPTL3 rs6690733.Results: We found rs6690733 (C vs A: OR 1.34, 95% CI 1.131.59; P=0.001) and rs12563308 (C vs T: OR 0.77, 95% CI 0.640.93, P=0.007) were significantly associated with susceptibility to IS. Even corrected for Bonferroni adjustment, the two variants were still significant (0.0074=0.028). Carriers of the minor allele of SNP rs6690733 had significantly higher levels of TC and LDL-C, while carriers of the minor allele of SNP rs12563308 had significantly lower levels of TC and LDL-C (all P<0.05). For rs6690733, the luciferase assay showed that promoter activity was significantly increased by 67% of plasmids containing the minor C allele compared with the major A allele in HEK293 cells.Conclusion: Our study revealed genetic variants of the ANGPTL3 gene could contribute to susceptibility to IS through participating in the regulation of lipid metabolism.

Keywords: ischemic stroke, ANGPTL3, genetic, atherosclerosis

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Only 30 children have been diagnosed with this rare genetic disease. Why are they coming to Dallas? – WFAA.com

October 24th, 2019 8:41 am

DALLAS How far would you go to save your child from a rare genetic disease with no known cure and no real option for treatment?

I have three kids and Id go to the end of the earth for them, said Stephen Grey, associate professor of pediatrics at UT Southwestern Medical Center.

Our tour of Grey's lab was the first, and likely only, time outsiders will ever be given access.

Nobody will be allowed in here without being qualified or gowned up appropriately, he explained. Its a sealed HEPA filter facility.

Inside the facility on the UT Southwestern campus, Grey will manufacture a virus that will be injected into children.

Stephen Grey's lab is on the campus of UT Southwestern Medical Center.

Teresa Woodard

The virus doesnt replicate or spread or cause any sort of infection.

It has one specific purpose: to deliver missing DNA, in the hopes of treating rare diseases caused by DNAs absence.

Grey calls it a molecular mail truck.

The simplest way I can explain what we do is children are born, they have a defect where theyre missing a gene, and we put it back, he explained.

Literally hundreds of trillions of virus particles carry the DNA and will go across a nervous system to bring the DNA into the cells where its missing and then it permanently fixes things, he said. Then, its really treating the disease at the root of everything thats wrong.

One disease Grey will tackle in his lab is often described as ALS, or Lou Gehrig's Disease, in children.

Theyre going to lose control of their legs, lose control of arms, and basically move to being essentially in a quadriplegic state, Grey described. Its ultimately fatal by the second or third decade of life.

Its called CMT4J and Talia Duff, 13, of Boston, is currently battlingthe disease.

She learned to walk and dance as a little girl. She was growing and gaining independence, but that progression stopped a few years ago.

Instead of gaining strength with age, Talia started to lose it. She is now in a wheelchair.

Kinley Kitchens, 14, lives in North Texas.

Her CMT4J is not as far along as Talia's, but each day is a battle.

You have very bad balance and a lack of coordination, she said, "so I fall a lot.

Her family worries about the future.

The progression is just the unknown, said Kinleys mother, Sabrina. You just dont know when its going to progress.

Only 30 kids in the world have CMT4J.

Recently, on the campus of UT Southwestern, two of them - Talia and Kinley and their families, came face to face for the first time.

It just makes you feel not so alone, said Jocelyn Duff, Talias mother, And I think its pretty special for both Talia and Kinley for that same reason.

Our researchers are often asked why we should care about a disease thats so rare, Jocelyn said.

Its really those rare diseases that are the guinea pigs for more common diseases. So these clinical trials are critically important for teasing apart what gene therapies might work for what diseases.

Because theyve been given such a grim prognosis and because no treatment currently exists these parents are willing to take great risks that could return remarkable results.

If we can do this for these rare diseases, then we can move on and expand the treatment to things like Alzheimers, autism, schizophrenia, epilepsy things that are very common, Grey said.

Grey doesn't aim to cure diseases, he said. His goal is to stop a disease's progression.

Cure is a four-letter word for me because it sets some expectations, but I think were looking at a comprehensive treatment for some really devastating diseases, he said.

Sure, the science is cool. Im a scientist. I love the curiosity and the problem solving but seeing what the families go through theyll never quit and they feel like my kids.

Grey hopes to begin clinical trials on about eight different rare diseases in early 2020. He hopes to have about a half dozen kids in each trial.

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1 Billion Years of Evolution Illuminated by Genetic Sequencing of 1,100 Plants – SciTechDaily

October 24th, 2019 8:41 am

The 1KP initiative, a collaborative effort of nearly 200 scientists, spans green algae to land plants, providing a framework for examining 1 billion years of plant evolution. Credit: Eric Zamora/Florida Museum

Plants are evolutionary champions, dominating Earths ecosystems for more than a billion years and making the planet habitable for countless other life forms, including us. Now, scientists have completed a nine-year genetic quest to shine a light on the long, complex history of land plants and green algae, revealing the plot twists and furious pace of the rise of this super group of organisms.

The project, known as the One Thousand Plant Transcriptomes Initiative (1KP), brought together nearly 200 plant biologists to sequence and analyze genes from more than 1,100 plant species spanning the green tree of life. A summary of the teams findings published on October 23, 2019, in Nature.

In the tree of life, everything is interrelated, said Gane Ka-Shu Wong, lead investigator of 1KP and professor in the University of Albertas department of biological sciences. And if we want to understand how the tree of life works, we need to examine the relationships between species. Thats where genetic sequencing comes in.

Much of plant research has focused on crops and a few model species, obscuring the evolutionary backstory of a clade that is nearly half a million species strong.

To get a birds-eye view of plant evolution, the 1KP team sequenced transcriptomes the set of genes that is actively expressed to illuminate the genetic underpinnings of green algae, mosses, ferns, conifers, flowering plants and all other lineages of green plants.

One hallmark of plant evolution is the frequency of genome duplication. Flowering plants are renowned for making multiple copies of their genome, which may contribute to the evolution of new gene functions. The 1KP project uncovered previously unknown duplication events in this group. Credit: Kristen Grace/Florida Museum

This gives a much broader perspective than what you could get by just looking at crops, which are all concentrated in one little part of the evolutionary tree, said study co-author Pamela Soltis, University of Florida distinguished professor and Florida Museum of Natural History curator. By having this bigger picture, you can understand how changes occurred in the genome, which then allows you to investigate changes in physical characteristics, chemistry or any other feature youre interested in.

One challenge was the projects sheer size, said study co-author Douglas Soltis, UF distinguished professor, and Florida Museum curator.

To look at that many genomes is unparalleled, he said. Its not a jump in technology as much as a jump in scale.

Sequencing transcriptomes requires freshly collected tissues, which is how Soltis found himself trekking through Gainesvilles greenery with containers of liquid nitrogen. Back at the laboratory, a team extracted genetic material from the frozen plant clippings and shipped the extractions to China for sequencing. All over the world, their colleagues followed suit.

Analyzing the sequences also required a reworking of existing software, which wasnt designed to handle such an unprecedented volume of genetic data, and without funding for the analysis, the researchers chipped away at the data as they had spare time.

But the labor was worth it, Pamela Soltis said.

The plant community got more than 1,000 sets of sequences, said Soltis, who also directs the UF Biodiversity Institute. Who could argue with that? All these branches of the plant tree of life have been filled in.

One hallmark of plant evolution and a feature rarely seen in animals is the frequency of genome duplication. Over and over again, lineages doubled, tripled or even quadrupled their entire set of genes, resulting in massive genome sizes. While the purpose of whole genome duplication is still unclear, scientists suspect that it may drive evolutionary innovation: If you have two copies of genes, one copy can gradually evolve a new function.

Addressing the frequency of whole genome duplication in plants was one of 1KPs goals, Douglas Soltis said. While flowering plants and ferns were already famous for genome duplication, Soltis said 1KP uncovered a number of previously unknown duplication events in these groups, as well as in the gymnosperms, the group of plants that includes conifers.

Other plant lineages took a different route, expanding certain gene families rather than copying their entire genome. This, too, is thought to provide new avenues for evolutionary development, and not surprisingly, the research team uncovered a major expansion of genes just before the appearance of vascular plants, land plants with xylem and phloem special cells for transporting water and nutrients.

But Douglas Soltis said gene expansions did not always correspond to major plant evolutionary milestones.

Theres not much of an expansion before seed plants appear or for flowering plants, he said. In fact, flowering plants actually shrank certain gene families, which may be a sign that they just co-opted existing genes for new functions.

Another surprise finding was that mosses, liverworts and hornworts form a single related group, confirming a centuries-old hypothesis that had been reversed in recent decades.

Wed done a partial analysis in 2014 that suggested these plants were close relatives, but a lot of people didnt believe it. These results underscore those findings, Pamela Soltis said. Its going to rock the moss world.

While the project refines our understanding of plant evolution and relationships between lineages, these data are also invaluable tools for advancing crop science, medicine, and other fields, the researchers said.

Identifying genes that have been duplicated in flowering plants could help scientists better understand their function, which could lead to crop improvements, Pamela Soltis said.

And because many plants have medicinal benefits, the genetic data offered by the 1KP project could lead to new discoveries that improve human health.

We focused on getting a lot of wild samples collected from plant lineages known to have important chemistry in hopes that people could mine this material for new compounds, Douglas Soltis said.

The sequences generated by the 1KP team are publicly accessible through the CyVerse Data Commons.

Probably hundreds of papers have used the data in ways we dont even know about, Pamela Soltis said. That is a super cool aspect of this study.

But the 1KP team has little time to celebrate its achievement. The next goal? Sequencing 10,000 genomes.

###

Reference: One thousand plant transcriptomes and the phylogenomics of green plants by One Thousand Plant Transcriptomes Initiative, 23 October 2019, Nature.DOI: 10.1038/s41586-019-1693-2

Matthew Gitzendanner, Evgeny Mavrodiev and Grant Godden of the Florida Museum and Emily Sessa of UFs department of biologyalso co-authored the study. James Leebens-Mack of the University of Georgia is a co-corresponding author.

The 1KP initiative was funded by the Alberta Ministry of Advanced Education and Alberta Innovates, Musea Ventures, the National Key Research and Development Program of China, the Ministry of Science and Technology of the Peoples Republic of China, the State Key Laboratory of Agricultural Genomics and the Guangdong Provincial Key Laboratory. Sequencing activities at BGI were also supported by the Shenzhen Municipal Government of China. Computational support was provided by the China National GeneBank, the Texas Advanced Computing Center, WestGrid and Compute Canada. Additional support was provided by the National Science Foundation, the NSF-funded iPlant Collaborative, the National Institutes of Health, German Research Foundation and the Natural Sciences and Engineering Research Council of Canada.

The quote above from Gane Ka-Shu Wong first appeared in a joint press release published by the University of Georgia and the University of Alberta.

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How Is Genetic Genealogy Shaping The Way Cold Cases Are Solved? – Oxygen

October 24th, 2019 8:41 am

In early 2018, investigators in California uploaded DNA from one of the Golden State Killer crime scenes to GEDmatch, an open data personal genomics database and genealogy website. They found a mutual descendant of the suspects great-great-great grandparents, and enlisted genetic genealogists to construct a family tree.

This eventually led authorities to Joseph James DeAngelo, a 72-year-old former police officer. DeAngelo was arrested that April and charged with 13 counts of murder, including one committed during burglary and rape, and 13 counts of kidnapping for robbery, according to CNN. He is currently awaiting trial.

Since DeAngelos arrest, law enforcement has investigated numerous cold cases using genealogical DNA databases, whether from open-use platforms like GEDmatch or commercial websites like FamilyTreeDNA. While an impressive number of cases have been solved, finding usable matches that lead to an arrest is labor intensive and often difficult.

Its a steep learning curve, CeCe Moore, head of genetic genealogy at Parabon NanoLabs, told Oxygen.com. Its been two years since genetic genealogists have started to be used. There are some law enforcement officials who are getting good at it. We just need more people that really put the time in to do it because there are a limited number of us out there that can work these cases skillfully.

To help law enforcement identify unknown suspect or victim DNA from a crime scene, genealogists upload the DNA sample to a database like GEDmatch. Then, they reverse-engineer the family tree of the unidentified person by comparing it to the family trees of people with whom they share DNA.

Youre hoping your top match is going to be at least a third cousin, and you can go back to a second great-grandparent. I use a lot of supporting matches as well, said Moore, who has worked on more than 200 cases. If youve got a lot of really close genetic matches, its obviously going to be a lot easier.

While genetic genealogists have proved critical in helping investigations, arrests and convictions are ultimately the result of police work.

It sometimes can only narrow it down so far, and then you turn it over to the investigators, Moore said. Were really just a lead generator. Were trying to get tips, and then investigators have to determine whether thats a viable tip or not.

The key to more successful identifications is access to DNA databanks, according to Moore. As more people upload their genetic information, there will be more profiles to compare them to.

The techniques are already very powerful, and the science is very powerful. We just need more DNA to compare it to, Moore told Oxygen.com. Thats what it relies on. We need more data and more people who are willing to contribute their DNA profiles to the effort.

As forensic genealogy has grown in usage, however, many genealogical websites have wrestled with the privacy concerns of their users. Increasingly, websites only allow access to users who have manually opted-in to share their profiles with law enforcement, thus limiting the data pool.

While Moore is hopeful that more cold cases will be solved in the coming years thanks to breakthroughs in DNA technology, she is realistic about the challenges ahead.

It takes a lot of skill unless you get very, very lucky, she said. Theres been some cases over the last year or so that law enforcement agencies have been able to figure it who it is themselves without a genetic genealogist because it was such a close match, but the vast majority of the cases 99 percent of them are going to need someone who is experienced and skilled, or they're just going to be wasting their time spinning their wheels. Its not that it will lead them in the wrong direction, it just wont lead them anywhere.

For more cold case investigations, follow Paul Holes as he explores the physical and emotional "DNA" of crime scenes in "The DNA of Murder with Paul Holes," premiering Saturdaysat 7/6c on Oxygen.

Crime Time is your destination for true crime stories from around the world, breaking crime news, and information about Oxygen's original true crime shows and documentaries. Sign up for our Crime Time Newsletter and subscribe to our true crime podcast Martinis & Murder for all the best true crime content.

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Here’s Why We’re Not At All Concerned With Seattle Genetics’s (NASDAQ:SGEN) Cash Burn Situation – Yahoo Finance

October 24th, 2019 8:41 am

There's no doubt that money can be made by owning shares of unprofitable businesses. For example, biotech and mining exploration companies often lose money for years before finding success with a new treatment or mineral discovery. But while the successes are well known, investors should not ignore the very many unprofitable companies that simply burn through all their cash and collapse.

Given this risk, we thought we'd take a look at whether Seattle Genetics (NASDAQ:SGEN) shareholders should be worried about its cash burn. For the purposes of this article, cash burn is the annual rate at which an unprofitable company spends cash to fund its growth; its negative free cash flow. First, we'll determine its cash runway by comparing its cash burn with its cash reserves.

View our latest analysis for Seattle Genetics

A cash runway is defined as the length of time it would take a company to run out of money if it kept spending at its current rate of cash burn. When Seattle Genetics last reported its balance sheet in June 2019, it had zero debt and cash worth US$376m. Looking at the last year, the company burnt through US$172m. That means it had a cash runway of about 2.2 years as of June 2019. Importantly, though, analysts think that Seattle Genetics will reach cashflow breakeven before then. If that happens, then the length of its cash runway, today, would become a moot point. Depicted below, you can see how its cash holdings have changed over time.

NasdaqGS:SGEN Historical Debt, October 21st 2019

On balance, we think it's mildly positive that Seattle Genetics trimmed its cash burn by 4.3% over the last twelve months. On top of that, operating revenue was up 32%, making for a heartening combination Considering the factors above, the company doesnt fare badly when it comes to assessing how it is changing over time. While the past is always worth studying, it is the future that matters most of all. For that reason, it makes a lot of sense to take a look at our analyst forecasts for the company.

Seattle Genetics seems to be in a fairly good position, in terms of cash burn, but we still think it's worthwhile considering how easily it could raise more money if it wanted to. Generally speaking, a listed business can raise new cash through issuing shares or taking on debt. Commonly, a business will sell new shares in itself to raise cash to drive growth. By comparing a company's annual cash burn to its total market capitalisation, we can estimate roughly how many shares it would have to issue in order to run the company for another year (at the same burn rate).

Seattle Genetics has a market capitalisation of US$15b and burnt through US$172m last year, which is 1.2% of the company's market value. So it could almost certainly just borrow a little to fund another year's growth, or else easily raise the cash by issuing a few shares.

It may already be apparent to you that we're relatively comfortable with the way Seattle Genetics is burning through its cash. In particular, we think its cash burn relative to its market cap stands out as evidence that the company is well on top of its spending. Its weak point is its cash burn reduction, but even that wasn't too bad! It's clearly very positive to see that analysts are forecasting the company will break even fairly soon Taking all the factors in this report into account, we're not at all worried about its cash burn, as the business appears well capitalized to spend as needs be. Notably, our data indicates that Seattle Genetics insiders have been trading the shares. You can discover if they are buyers or sellers by clicking on this link.

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Of course, you might find a fantastic investment by looking elsewhere. So take a peek at this free list of interesting companies, and this list of stocks growth stocks (according to analyst forecasts)

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned. Thank you for reading.

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Study: The Likelihood of Developing PTSD Following Trauma Is Party Determined by Genetics – The Swaddle

October 24th, 2019 8:41 am

Our understanding of post-traumatic stress disorder (PTSD), until now, is that it is a mental health disorder that occurs as a consequence of exposure to extreme, life-threatening stress, and/or serious injury. This exposure, by definition, is requisite for the development of PTSD, but not all those who face trauma necessarily develop it the individual susceptibility to PTSD varies widely. Since the turn of the century, scientists have been trying to find evidence for genetic influence on PTSD risk, with the last decade witnessing concerted efforts to identify specific DNA variants that can influence ones genetic susceptibility to develop PTSD.

New research, findings of which were published in Nature Communications, has, for the first time, identified a clear biological pathway for the mental health disorder, despite a section of science still viewing it as an entirely social construction. In the largest and most diverse genetic study of PTSD to date, scientists from the University of California, San Diego, School of Medicine and more than 130 additional institutions have concluded that genetics do, in fact, play a role in determining whether or not a person will develop PTSD, similar to the biological pathways of depression and other forms of mental illness.

Our long-term goal is to develop tools that might help clinicians predict who is at greatest risk for PTSD and personalize their treatment approaches. We cant always protect people from trauma. But we can treat them in the best ways possible, at the best time, Dr. Caroline Nievergelt, the studys first author, associate professor of psychiatry at UC San Diego School of Medicine and associate director of neuroscience in the Center of Excellence for Stress and Mental Health at the Veterans Affairs San Diego Healthcare System, said in a press release.

In collaboration with the Psychiatric Genomics Consortiums PTSD working group and Cohen Veterans Bioscience, a non-profit organization dedicated to accelerating PTSD and traumatic brain injury research, the studys authors built a 12-country network of more than 200 researchers, assembling data and DNA samples from more than 20,000 people with PTSD and 170,000 control subjects (those who did not develop PTSD following trauma).

At more than 200,000 people, the latest studys sample size is 10 times larger than the firstPsychiatric Genomics Consortium PTSD study, published in 2017, and includes both civilians and members of the military. The release notes the cohort is also the most ancestrally diverse for any psychiatric genetics study to date, with more than 23,000 people with PTSD of European ancestry and more than 4,000 of African ancestry.

Related on The Swaddle:

Researchers Find Genetic Hotspot Behind Autism

Putting this large database through statistical analyses, the studys authors measured the effect of gene variants at millions of different points on chromosomes across the human genomes on someones chances of developing PTSD.

According to the findings, PTSDs heritability the level of influence genetics has on the variability of PTSD risk among people is between 5% and 20%. Scientists found that, like other psychiatric disorders and several human traits, the risk of developing PTSD following trauma is a highly polygenic trait. This means there exist thousands of genes at different loci on different chromosomes that make tiny contributions to the disorder and when expressed together, add up to the heritable trait. Scientists have found gene variants at six loci that were strongly associated with PTSD risk.

Three of the six loci were specific to certain ancestral backgrounds two European and one African and three were only detected in men. The six loci hint that inflammatory and immune mechanisms may be involved in the disorder, which is consistent with findings from previous studies, the release summarises.

Further exploring the relatively nascent belief in science that many psychiatric disorders and behavioral traits have important molecular similarities at DNA-level, the study also analyzed genetic correlations between PTSD and 235 other disorders behaviors and physical traits. They found a significant overlap with 21, including depression, schizophrenia, insomnia, asthma, and coronary artery disease. Additionally, a Parkinsons disease gene involved in dopamine regulation was also found to be associated with PTSD. Similar to other mental disorders, the genetic contribution to PTSD correlates with that for many other traits. Further research is needed to determine what this means whether some of the same genes that influence risk for PTSD also influence risk for other diseases like, for example, depression, Karestan Koenen, a senior author of the study and associate member of the Stanley Center for Psychiatric Research at MIT and Harvard University, and a professor of psychiatric epidemiology in the Harvard T.H. Chan School of Public Health, said in the release.

Koenen adds: Based on these findings, we can say with certainty that there is just as much of a genetic component to PTSD risk as major depression and other mental illnesses. Our limited ability to study the living human brain and uncover the biological roots of PTSD has contributed to the lack of treatments and the stigma around this debilitating condition. Genetics helps us make new discoveries, find opportunities for new therapies, and counter that stigma.

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23andMe Wants Everyone to Get Used to Sharing Their Genetic Data – Motherboard

October 24th, 2019 8:41 am

What Anne Wojcicki, co-founder and CEO of 23andMe, would like you to consider is this: Once upon a time, people were kinda freaked out by the idea of typing their credit card info into a website. Now this is done basically all the time. Your phone is a digital wallet; your web browsers will memorize your card numbers for you. Might your bank information get cribbed or hacked? Has it already been? Yeah, probably so, but this is How We Live Now. Its weird to think it was ever not normal.

This little historic tale is how Wojcicki addresses the current hesitance a majority of people feel to submitting their genetic material to a database by spitting into a tube and mailing that tube off to a companys lab. In a brief discussion at the TIME 100 Health Summit in New York City on Thursday, Wojcicki explained that shes actually grateful for the 2030 percent of the population thats down to spit (according to a Twitter poll TIME ran ahead of Wojcickis talk). At-home DNA kits like 23andMe are still a relatively new technology. People simply need to grow accustomed to the ideas of genetic testing and sharing genomic data with public databases, so that researchers can observe patterns across the population, and ultimately make people healthier, she said.

Its essentially an argument for sharing DNA data as good for public health, like a new-age equivalent of getting vaccinated. To drive the point home further, Eric Lander, a geneticist and director of the Broad Institute of MIT (and someone who once sat around a table with Jeffrey Epstein), mentioned a potential breakthrough in treating angiosarcoma, a rare, highly fatal cancer by using using DNAthat, Lander argued, may never have happened if it werent for peoples willingness to fork over health and genetic information.

Its hard to find a sensible, non-demonic argument against something that could lead to expedited breakthroughs in cancer treatment. But what has to be kept in mind is that 23andMe is a private company. These anecdotesvirtuous as they may soundare marketing techniques. Wojcicki rightfully believes that no singular institution will be able to harvest the amount of DNA that her company has. According to 23andMes About page, more than 10 million people have spit into its tubes and thereby handed their genetic information over. Of those 10 million, 80 percent have opted in to participate in research, via that spit data. For context: The National Institute of Health is currently in the midst of enrolling its largest DNA-related study to date (All Of Us), which will reach full enrollment at one million participants. About a year into their recruiting efforts, theyre about a quarter of the way there, according to what NIH Director Francis Collins said in a separate panel at Thursdays TIME 100 Health Summit.

Wojcicki emphasized that only those who opt in for ancestry information have their data entered into public databases, which are subject to subpoena (the likes of which helped identify the Golden State Killer). She further emphasized that, because DNA is highly similar among family members, submitting your spit implicates your relatives genetic information. (23andMe consents around this.) Thats a hell of a lot of data thats sitting around for seemingly forever, and since 23andMe is, once again, a private company, theres no telling what happens to this info if/when the company goes under, or if they decide to change their policies.

What 23andMe is sitting on now is perhaps the most valuable pool of genetic data in the world. Earlier this year, the company partnered with TrialSpark, an NYC-based research company, in order to use its large database of data to fit its opted-in consumers to studies. Mind you, Wojcicki used to work on Wall Street. She is, at her core, a businessperson. Charging customers to have their data, and then partnering with another company with an interest in that data, sounds lucrative as fuck; a hell of a business deal.

Wojcicki and Lander concluded their talk on Thursday with a heartwarming sentiment: The virtue of something like 23andMe is that consumers (or participants, however youd prefer to look at it) have access to their genetic data, rather than submitting it to a study and never getting feedback. Wojcicki refers to this as empowering; its empowering to know whats going on in the little strands that make you the person you are. It also sounds very empowering, monetarily speaking, for those in the game, marketing spit kits and trading (totally consented for!!) genetic data.

Go here to see the original:
23andMe Wants Everyone to Get Used to Sharing Their Genetic Data - Motherboard

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Type 2 diabetes: Eating this superfood could help fight the condition – Express

October 22nd, 2019 7:46 pm

Type 2 diabetes is a condition in which the body cant control the amount of glucose (sugar) in the blood, causing levels to become too high, and left untreated, serious complications such as kidney failure, nerve damage, heart disease and stroke can occur. High blood sugar levels can be prevented or managed with lifestyle changes such as regular exercise, but while the NHS recommends 30 minutes of moderate aerobic exercise five days a week, a recent report by the World Health organisation estimates a third of UK adults arent active enough. Now, new research by experts in New Zealand says consuming a specific type of blackcurrant could help sedentary adults walk for longer without getting tired. The effects took hold just an hour after taking the blackcurrant supplement.

Meanwhile the superfood also resulted in a dramatic 90 percent reduction in the build of enzymes linked to the onset of dementia and depression.

The studys lead author Dr Suzanne Hurst said: "Identifying foods or dietary supplements that specifically support the desire to exercise daily will enable an individual to adhere to an exercise programme and maintain and active and healthy lifestyle."

And she found that drinking blackcurrant juice one hour before exercise supports positive affective responses during a low impact walking exercise in healthy sedentary adults.

The blackcurrants themselves were grown in New Zealand - and because of the Southern Hemispheres intense UV light, this produces unusually high levels of protective and health-giving nutrients compared with berries grown elsewhere.

The key ingredients are anthocyanins, pigments in the fruit's skin responsible for their dark purple colouring, which belong to a group of flavonoids called polyphenols and possess high levels of antioxidants.

The participants - a group of male and female adults aged between 20 and 59 - were given either blackcurrant juice or a placebo an hour before they were asked to walk on a treadmill.

The average time walked by those whod taken blackcurrants was 11 minutes longer than those who hadnt.

While only 10 percent of those whod had the placebo walked a distance greater than 10km - compared with 30 percent of participants in the blackcurrant group.

The treadmill walkers whod had blackcurrants also reported lower exertion scores - and higher overall mood scores - than those whod had the placebo. Test subjects also underwent a blood test both before and after the experiment.

And its here they noted a huge decrease in something called monoamine oxidase-B, or MAO-B, activity - something they believe could help fight neurological diseases like dementia.

Dr Hurst, of the New Zealand Institute for Plant & Food Research, says: "We found that plasma collected 1 hour after blackcurrant consumption showed a dramatic acute decline - 90 percent - in platelet MAO-B activity, which was still detectable in participants plasma once they had stopped exercise.

"The pharmacological inhibition of brain MAO-B activity has been used to treat those diagnosed with neurological diseases and depression, potentially through their neuroprotective properties in reducing the metabolism of monoamines."

The findings were reported in the Journal of the International Society of Sports Nutrition and they have been welcomed by Nicki Bundock, of Surrey-based firm CurraNZ, the UKs leading supplier of New Zealand blackcurrants supplements.

Nicki explained: "Regular exercise is one of the best ways to reduce your chances of health problems like heart disease or type 2 diabetes. But if youre not an active person, starting an exercise plan and actually sticking with it can be incredibly difficult.

Studies have shown more than half of us drop out of exercise plans after just six months, reverting to inactive lifestyles. And its therefore vital we find ways to make exercise more tolerable and enjoyable.

This study suggests blackcurrant supplements can boost time spent exercising while also making it feel easier and more pleasurable. And the less stressful exercise is, the more likely it is to become a habit.

"The fact the study also suggested benefits to neurological health make it even more welcome."

Previous studies have also demonstrated the positive effect of blackcurrants on blood sugar.

A clinical trial carried out at the University of Aberdeen showed the fruit could affect how the body breaks down carbohydrates and sugars, reducing the amount of sugar that ends up in the bloodstream after a person eats a meal.

Another fruit found to have a positive impact on blood sugar is guava.

Read the rest here:
Type 2 diabetes: Eating this superfood could help fight the condition - Express

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