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Archive for the ‘Gene therapy’ Category

Protection of genetic heritage in the era of cloning

Sunday, January 27th, 2013

Research on human beings has expanded greatly due to progress and the evolution of society as well as customs. Not only the unceasing development of research on human beings, but also interference in the beginning and end of life with homologous and heterogonous human reproduction, surrogate motherhood, cloning, gene therapies, eugenics,euthanasia, dysthanasia, orthothanasia, assisted suicide, genetic engineering, reassignment surgery in cases of transsexuality, the use of recombinant DNA technology and embryonic stem cells, transplantation of human organs and tissues, biotechnology and many other scientific advances. Scientific progress goes faster than the real needs of human beings, who are the final recipient of the entire evolutionary progress. Hence, there is the need to scrutinize w...

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Protection of genetic heritage in the era of cloning

Sunday, January 27th, 2013

Research on human beings has expanded greatly due to progress and the evolution of society as well as customs. Not only the unceasing development of research on human beings, but also interference in the beginning and end of life with homologous and heterogonous human reproduction, surrogate motherhood, cloning, gene therapies, eugenics,euthanasia, dysthanasia, orthothanasia, assisted suicide, genetic engineering, reassignment surgery in cases of transsexuality, the use of recombinant DNA technology and embryonic stem cells, transplantation of human organs and tissues, biotechnology and many other scientific advances. Scientific progress goes faster than the real needs of human beings, who are the final recipient of the entire evolutionary progress. Hence, there is the need to scrutinize w...

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Gene Therapies Will Cure Many a Disease (preview)

Sunday, January 13th, 2013

The Science Of The Next 150 Years: 50 Years in the Future [More]

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The Science of the Next 150 Years

Sunday, January 13th, 2013

What scientific and technological milestones can we envision 50, 100 and 150 years hence?

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Gene Therapies Will Cure Many a Disease (preview)

Sunday, January 13th, 2013

The Science Of The Next 150 Years: 50 Years in the Future [More]

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The Science of the Next 150 Years

Sunday, January 13th, 2013

What scientific and technological milestones can we envision 50, 100 and 150 years hence?

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Mucopolysacccharidoses: from understanding to treatment, a century of discoveries

Sunday, December 23rd, 2012

After the first description of a patient recognized as a MPS case was made in 1917, several similar cases were described and identified. Observations reported in the middle of the twentieth century concerning the presence of acid mucopolysaccharides (later called glycosaminoglycans, or GAGs) in tissues and especially in urine of patients were instrumental in providing an identity for these diseases, which became referred as "mucopolysaccharidoses" (MPS). In the late 1960's it was demonstrated that MPS were caused by defects in the breakdown of GAGs, and the specific enzyme deficiencies for the 11 types and subtypes of MPS were identified thereafter. Genes involved in the MPS were subsequently identified, and a large number of disease-causing mutations were identified in each one. Although ...

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Mucopolysacccharidoses: from understanding to treatment, a century of discoveries

Sunday, December 23rd, 2012

After the first description of a patient recognized as a MPS case was made in 1917, several similar cases were described and identified. Observations reported in the middle of the twentieth century concerning the presence of acid mucopolysaccharides (later called glycosaminoglycans, or GAGs) in tissues and especially in urine of patients were instrumental in providing an identity for these diseases, which became referred as "mucopolysaccharidoses" (MPS). In the late 1960's it was demonstrated that MPS were caused by defects in the breakdown of GAGs, and the specific enzyme deficiencies for the 11 types and subtypes of MPS were identified thereafter. Genes involved in the MPS were subsequently identified, and a large number of disease-causing mutations were identified in each one. Although ...

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World Changing Ideas 2012 (preview)

Sunday, December 2nd, 2012

Scientists and engineers dream about big advances that could change the world, and then they try to create them. On the following pages, Scientific American reveals 10 innovations that could be game changers: an artificial alternative to DNA, oil that cleans water, pacemakers powered by our blood, and more. These are not pie-in-the-sky notions but practical breakthroughs that have been proved or prototyped and are poised to scale up greatly. Each has the potential to make what may now seem impossible possible. -- The Editors

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World Changing Ideas 2012 (preview)

Sunday, December 2nd, 2012

Scientists and engineers dream about big advances that could change the world, and then they try to create them. On the following pages, Scientific American reveals 10 innovations that could be game changers: an artificial alternative to DNA, oil that cleans water, pacemakers powered by our blood, and more. These are not pie-in-the-sky notions but practical breakthroughs that have been proved or prototyped and are poised to scale up greatly. Each has the potential to make what may now seem impossible possible. -- The Editors

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SCMOM 2012_Sistemic – Video

Thursday, November 29th, 2012


SCMOM 2012_Sistemic
Sistemic #39;s world leading expertise is on applying microRNA profiling in context with the biology, to areas where there is currently an unmet need for sophisticated tools delivering instructive and robust knowledge of the cell system. From their SistemQC trade; platform they have derived an extensive suite of tools for the cell therapy, gene therapy and bioprocessing community. SistemQC trade;, molecularly characterises cells including stem cells as well as aids in the optimization and monitoring (QC) of the manufacture process. More specifically the initial focus of SistemQC trade; by clients has been on generation of microRNA based ID markers, purity potency assessment and manufacture monitoring optimization. http://www.sistemic.co.uk Presenter: Jim Reid, Chairman and CEO, SistemicFrom:AllianceRegenMedViews:3 1ratingsTime:11:56More inScience Technology

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How to Grow a Retina from Stem Cells (preview)

Sunday, November 18th, 2012

In the womb, a ball of identical cells gives rise to varied cell types that ultimately form highly ordered structures and then the full panoply of organs in the human body. The process advances according to an internal biological script that directs each fold and crease of tissue to assume exactly the proper shape and dimension.

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Cultivator of Brain Parts

Sunday, November 18th, 2012

Yoshiki Sasai is not just an ordinary tissue engineer who tries to coax stem cells to grow into fully formed bodily structures. It is true that Sasai has made his mark by taking on big projects like using stem cells to whip up a retina, cortical tissue and the cerebellum, involved with balance and movement. But his research has gone deeper by delving into the way stem cells organize themselves into complex structures under the influence of genes and the prenatal environment. Read a profile of Sasai here to accompany “ Grow Your Own Eye ,” Sasai’s own account of growing a retina in the November Scientific American .

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How to Grow a Retina from Stem Cells (preview)

Sunday, November 18th, 2012

In the womb, a ball of identical cells gives rise to varied cell types that ultimately form highly ordered structures and then the full panoply of organs in the human body. The process advances according to an internal biological script that directs each fold and crease of tissue to assume exactly the proper shape and dimension.

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Post-genomic Cardiology – Video

Friday, November 9th, 2012


Post-genomic Cardiology
ll4.me Post-genomic Cardiology Recent advances in molecular and cellular biology have markedly changed our understanding of the heart, and this is having tremendous ramifications for the clinician. This unique reference offers a comprehensive and critical evaluation of this contribution in the field of cardiovascular molecular medicine providing the reader with a sense of new directions in which molecular medicine might be applied. It begins with a detailed primer that makes readily accessible recent molecular, genetic and cellular techniques. Rounding out the coverage of this exciting field are critical and comprenhesive discussions on the use of molecular, genetic and cellular techniques used to identify the etiology and pathophysiology of specific cardiac diseases.* Discusses diagnostic and therapeutic options available not only in the adult and aging individuals but also in infants/children* Numerous illustrations and flow-charts* Explans cutting-edge molecular techniques, including analysis of mitochondria, their role in cardiac dysfunction and updated analysis of Cardioprotection and Metabolic Syndrome* Presentation of recent translational studies for the treatment of cardiovascular diseases is included (eg, gene therapy, pharmacological treatments and stem cell transplantation) Publisher: Academic Press Illustration: Y Language: ENG Title: Post-Genomic Cardiology Pages: 00000 (Encrypted PDF) On Sale: 2007-02-26 SKU-13/ISBN: 9780123736987 Category: Medical ...From:julianlewis9854Views:0 0ratingsTime:00:14More inPeople Blogs

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DNA Hacking Is Now Street Legal

Tuesday, November 6th, 2012

The world's first mass-market gene therapy has been legalized in Europe. Glybera, which treats a painful disorder that leads to pancreatitis, will be available in hospitals beginning next year.

Chances are you haven't heard much about lipoprotein lipase deficiency (LPLD), a disease that leads to pancreatitis. This rare disease, however, is at the center of world-changing medical advances.

Last week the European Union approved a gene therapy treatment for LPLD--and this marks the first time any medical treatment that rewrites a patients' DNA has been approved for commercial use.

The treatment, called Glybera, will be released by Dutch firm uniQure in the second half of 2013. Glybera will be administered to patients by specially trained doctors at a limited number of European hospitals. Patients receiving treatment have their DNA altered by a series of injections into their leg muscles, which helps normalize the metabolism of fat particles carried in the blood. LPLD prevents sufferers from properly metabolizing these particles, leading to a host of side effects including pancreatitis.

Gene therapy is an emerging form of medicine, focusing on the use of DNA to rewrite or supplement existing genes. This experimental practice is expected to lead to a series of breakthroughs over the next 50 or so years for various types of cancers, Parkinson's disease, sickle cell anemia, and a host of inherited conditions. Studies involving different forms of gene therapy are currently under way in Europe and the United States--one famous case in 2007 involved a man who was cured of HIV through stem cell transplantation. Stem cell transplants are one type of gene therapy used to give patients therapeutic DNA; other methods use different sources.

LPLD affects approximately one in 1,000,000 people worldwide. Apart from (frequently fatal) acute pancreatitis, patients regularly suffer from yellow spots on their skin, swollen abdomens, enlarged livers, severe abdominal pain, and diarrhea. The condition is currently treated through diet--patients are encouraged to go on a strict low-fat, alcohol-and-red-meat-free diet to mitigate symptoms.

By helping to normalize the metabolism of fat, Glybera prevents inflammation of the pancreas thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities, said Professor John Kastelein of the University of Amsterdam in a statement.

Gene therapy treatments in the United States are primarily focused on cancer, including leukemia. No other gene therapy products are expected to be approved by major medical regulator agencies in 2012. The European Commission, which handles medical approvals for Europe, is widely considered to have a bureaucratic pipeline that is far more open to gene therapy than the United States.

[Image: DNA damage via Tom Ellenberger/Washington University School of Medicine]

For more stories like this, follow @fastcompany on Twitter. Find Neal Ungerleider, the author of this article, on Twitter.

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Disorders Of Hemoglobin – , Bernard G. Forget – Video

Sunday, November 4th, 2012


Disorders Of Hemoglobin - , Bernard G. Forget
ll4.me Disorders Of Hemoglobin - , Bernard G. Forget This book is a completely revised new edition of the definitive reference on disorders of hemoglobin. Authored by world-renowned experts, the book focuses on basic science aspects and clinical features of hemoglobinopathies, covering diagnosis, treatment, and future applications of current research. While the second edition continues to address the important molecular, cellular, and genetic components, coverage of clinical issues has been significantly expanded, and there is more practical emphasis on diagnosis and management throughout. The book opens with a review of the scientific underpinnings. Pathophysiology of common hemoglobin disorders is discussed next in an entirely new section devoted to vascular biology, the erythrocyte membrane, nitric oxide biology, and hemolysis. Four sections deal with #65533;? #65533; #65533;? #65533; and #65533;? #65533; thalassemia, sickle cell disease, and related conditions, followed by special topics. The second edition concludes with current and developing approaches to treatment, incorporating new agents for iron chelation, methods to induce fetal hemoglobin production, novel treatment approaches, stem cell transplantation, and progress in gene therapy.Author: Steinberg, Martin H. Author: Forget, Bernard G. Author: Higgs, Douglas R. Publisher: Cambridge University Press Illustration: N Language: ENG Title: Disorders of Hemoglobin Pages: 00848 (Encrypted PDF) On Sale: 2009-06-30 SKU-13/ISBN: 9780521875196 ...From:marylloyd321Views:0 0ratingsTime:00:13More inPeople Blogs

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(301) 337-2100 "Gene Therapy Rockville, 20850" "Cell therapy Rockville, 20850" – Video

Thursday, November 1st, 2012


(301) 337-2100 "Gene Therapy Rockville, 20850" "Cell therapy Rockville, 20850"
http://www.AmericanGeneTechnologies.com American Gene Technologies International Inc. (AGTII) is developing bio-safe, high efficiency lentivirus therapies to treat cancer and other chronic human diseases. AGTII was founded in 2007 to develop bio-safe, high-efficiency, multi-gene vectors for human gene delivery in-vivo. AGTII #39;s products incorporate a novel, proprietary technology for the clinical application of gene expression technology in cancer therapy, autosomal disorders, stem cell treatment, and cosmetic applications. In vivo studies completed at the University of Maryland have demonstrated the efficacy of AGTII #39;s prototype prostate cancer therapeutic by eliminating Human Prostate Cancer tumors in mouse hosts. The active treatment group showed complete resolution of disease without harm to healthy tissue - even in large, late-stage tumors. AGTII also has prototype therapeutics in testing for Pancreatic Cancer. These prototypes have demonstrated efficacy in In vitro testing, as well. Additional designs for therapeutics addressing other cancers are in the design phase. AGTII #39;s vision is to use its technology to enable the one hundred billion dollar genetic drug market. CONTACT US! American Gene Technologies International Inc. Maryland Technology Development Center 9700 Great Seneca Highway Rockville, MD 20850 Phone: (301) 337-2100 Fax: (301) 337-2101 Email: info@agtii.com ----------------------------------------------------- Produced by: Create A Social Buzz SEO Social ...From:CreateAsocialBuzzViews:16 0ratingsTime:01:38More inScience Technology

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bluebird bio Awarded $9.3 Million From the California Institute for Regenerative Medicine to Further Gene Therapy …

Thursday, November 1st, 2012

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that the California Institute for Regenerative Medicine (CIRM) has approved an award to the company for $9.3 million for the first round of its new Strategic Partnership Awards initiative. The award is to support a Phase 1/2 study to evaluate the safety and efficacy of LentiGlobin, the companys development-stage program for the treatment of beta-thalassemia, which will be initiated in the United States in 2013.

We are very encouraged by the clinical data generated to date demonstrating the potential of LentiGlobin as a one-time transformative gene therapy for patients with beta-thalassemia, said David Davidson, M.D., chief medical officer, bluebird bio. bluebird bio has made significant advances in lentiviral vector design, transduction efficiency, and in our manufacturing process, enabling the production of gene-modified products that can be scaled and deployed for many different clinical indications. We are delighted that CIRM has chosen to recognize the importance of this innovative approach for the treatment of one of the most commonly inherited blood disorders, and we are excited to work with CIRM to continue the development of LentiGlobin in the U.S.

bluebird bios LentiGlobin product introduces a fully functional human beta-globin gene into the patient's own hematopoietic stem cells. These corrected stem cells ultimately produce fully functioning red blood cells. bluebird bio is currently conducting a Phase 1/2 trial examining the feasibility, safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell disease. Results of the first patient were reported in Nature in 2010, showing dramatic results, including stable expression of functional beta-globin resulting in transfusion independence which now extends for greater than four years following a single treatment.

This CIRM award is among the first awards under the agencys Strategic Partnership Awards initiative, which is designed to engage more effectively with industry and to increase outside investment in CIRM-funded stem cell research. The funding awards were made at the October 25, 2012 meeting of the stem cell agencys governing board, the Independent Citizens Oversight Committee (ICOC).

About CIRM

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. A list of grants and loans awarded to date may be seen here: http://www.cirm.ca.gov/for-researchers/researchfunding.

About bluebird bio

bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bios product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The companys novel approach uses stem cells harvested from the patients own bone marrow into which a healthy version of the disease causing gene is inserted. bluebird bios approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and potentially presenting a one-time transformative therapy using a patients own stem cells. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease. Led by a world-class team, its operations are located in Cambridge, Mass., San Francisco and Paris, France. For more information, please visit http://www.bluebirdbio.com.

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New gene therapy method tested in human cells … and it works, researchers report

Thursday, November 1st, 2012

ScienceDaily (Oct. 24, 2012) Oregon Health & Science University's development of a new gene therapy method to prevent certain inherited diseases has reached a significant milestone. Researchers at the university's Oregon National Primate Research Center and the OHSU Department of Obstetrics & Gynecology have successfully demonstrated their procedure in human cells. It's believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects.

The research results are online Oct. 24, in the journal Nature. Dr. Mitalipov also will present the results of his research at the American Society for Reproductive Medicine Conference in San Diego Oct. 24

The OHSU gene therapy method was initially devised through research in nonhuman primates led by Shoukhrat Mitalipov, Ph.D., associate scientist in the Division of Reproductive & Developmental Sciences at ONPRC, Oregon Stem Cell Center and OHSU School of Medicine departments of Obstetrics and Gynecology and Molecular and Medical Genetics.

The procedure was specifically developed to prevent diseases related to gene defects in the cell mitochondria. Mitalipov's previous work was published in the August 2009 edition of Nature. In the current study, Mitalipov, in collaboration with Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women's Health, demonstrated efficacy of this therapy in human gametes and embryos.

"Cell mitochondria contain genetic material just like the cell nucleus and these genes are passed from mother to infant," explained Mitalipov. "When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations."

To conduct this research, Mitalipov and his colleagues obtained 106 human egg cells from study volunteers recruited through OHSU's Division of Fertility and Reproductive Endocrinology. The researchers then used a method developed in previous nonhuman primate studies, to transfer the nucleus from one cell to another. In effect, the researchers "swapped out" the cell cytoplasm, which contains the mitochondria. The egg cells were then fertilized to determine whether the transfer was a success and whether the cells developed normally. Upon inspection, it was demonstrated that it was possible to successfully replace mitochondrial DNA using this method.

"Using this process, we have shown that mutated DNA from the mitochondria can be replaced with healthy copies in human cells," explained Mitalipov. "While the human cells in our study were allowed to develop to the embryonic stem cell stage, this research shows that this gene therapy method may well be a viable alternative for preventing devastating diseases passed from mother to infant."

The current Nature paper also expanded upon the previously reported nonhuman primate work by demonstrating that the method was possible using frozen egg cells. Mitochondria were replaced in a frozen/thawed monkey egg cell, resulting in the birth of a healthy baby monkey named Chrysta.

The second portion of the study, which was completed at ONPRC, is also considered an important achievement because egg cells only remain viable for a short period of time after they are harvested from a donor. Therefore, for this therapy to be a viable option in the clinic, preservation through freezing likely is necessary so that both the donor cell and a mother's cell are viable at the time of the procedure.

While this form of therapy has yet to be approved in the United States, the United Kingdom is seriously considering its use for treating human patients at risk for mitochondria-based disease. It's believed that this most recent breakthrough, combined with earlier animal studies, will help inform that decision-making process.

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