StemCell Therapy

Updated | Late last week, reports emerged that scientists in Oregon had used gene-editing technology, known as CRISPR-Cas9, to edit a human embryo. While research like this is already occurring in China and Great Britain, this is the first time scientists in the U.S. have edited an embryo.

The move raises thequestion of whether regulations are strict enough in the U.S. Both Congress and the National Institutes of Health have explicitly said they would not fund research that uses gene-editing to alter embryos. But laws and guidelines are not keeping pace with this fast-moving and controversial work.

CRISPR is an experimental biomedical technique in which scientists are able to alter DNA, such as change the misspellings of a gene that contributes to mutations. The technology has the potential to reverse and eradicate congenital diseases if it can be used successfully on a developing fetus.

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Here's how CRISPR gene editing works. REUTERS

The news frenzy that followed this announcement was based on a leak from unknown sources. Initial reports emerged from a number of less known sources, including MIT Technology Review, that Shoukhrat Mitalipov of Oregon Health and Science University used the technology to change the DNA of not just one, but a number of embryos. But the news stories about this research werent based on a published study, which means they dont provide the full picture. No one yet knows what the researchers did or what the results were.

Until now, most of the breakthrough research on CRISPRaside from the discovery itself, which is attributed to multiple research groups in the U.S. has occurred in China. InApril 2015, Chinese scientists reported that theyd edited the genome of human embryos, a world first, in an attempt to eliminate the underlying cause of a rare blood disorder.

Researchers there have also been experimenting with CRISPR technology to treat cancer. Last spring, a team of scientists at Sichuan Universitys West China Hospital used the approach to modify immune cells in a patient with an aggressive form of lung cancer. The researchers altered genes in a bid to empower the cells to combat the malignancy. Another group of Chinese scientists tried changing genes in blood that were then injected into a patient with a rare form of head and neck cancer to suppress tumor growth.

Despite potential of CRISPR to cure fatal diseases, the technology has fast become one of the most significant challenges for bioethicists. Some people view its power as potentially dangerous because it could allow scientists to cherry-pick genetic traits to create so-called designer babies.

Arthur Caplan, a professor of bioethics at New York University's Langone Medical Center and founding director of NYULMC's division of medical ethics thinks the fears are overblown. Gene-editing technology, says Caplan, is nowhere near this sci-fi fantasy.

If you would compare this to a trip to Mars, you're basically launching some satellites, says Caplan. He suggests that much of the media coverage on CRISPR is melodramatic, including last weeks coverage of researchers meddling with an embryo. We haven't shown that you can fix a disease or make someone smarter.

Lack of Guidelines

CRISPR technology isnt ready for clinical use, whether to stop serious genetic diseases or simply make brown eyes blue. But geneticists are working toward these goals, and the scientific community is ill-prepared to regulate this potentially powerful technology.

So far guidelines for using CRISPR are minimal. In 2015, the National Institutes of Health issued a firm statement. Advances in technology have given us an elegant new way of carrying out genome editing, but the strong arguments against engaging in this activity remain, the NIH said in its statement. These include the serious and unquantifiable safety issues, ethical issues presented by altering the germline in a way that affects the next generation without their consent, and a current lack of compelling medical applications justifying the use of CRISPR/Cas9 in embryos.

But although the NIH wont back CRISPR research for embryo editing, that doesnt mean such research is prohibited in the U.S. Private organizations and donors fund researchers. Caplan suspects this is how the team in Oregon managed to carry out their experiment.

In February 2017, the National Academy of Sciences and the National Academy of Medicinetwo leading medical authorities that propose medical and research guidelines for a wide range of research and medical topics issued sweeping recommendations for the use of CRISPR technology. In their joint Human Genome Editing: Science, Ethics, and Governance report, the panel of experts deemed the development of novel treatments and therapies an appropriate use of the technology. The recommendations also approve investigating CRISPR in clinical trials for preventing serious diseases and disabilities and basic laboratory research to further understand the impact of this technology.

The authors of the report caution against human genome editing for purposes other than treatment and prevention of diseases and disabilities. But the line between treatment and enhancement isnt always clear, says Caplan. And policing so-called ethical uses of CRISPR technology will be increasingly difficult because single genes are responsible for a myriad diseases and traits. You don't realize that you're changing DNA in places you don't want to, he says.

A source familiar with the controversial Oregon research reported last week told Newsweek that a major journal will publish a paper on the work by the end of this week. According to The Niche, a blog produced by the Knoepfler Lab at University of California Davis School of Medicine in Sacramento, California, the paper is slated to be published in Nature . Mitalipov did not respond to Newsweek s requests for comment or confirmation.

Caplan hopes that publication of the paper will initiate further discussion about the ethics of experimenting with CRISPR including practical measures such as a registry for scientists conducting studies through private funding. We need to have an international meeting about what are the penalties of doing this, he says. Will you go to jail or get a fine?

This story has been updated to note that the initial report of the CRISPR research in Oregon was based on a leak, but did not necessarily misconstrue the research.

Originally posted here:
Gene Editing Is Revolutionizing Medicine but Causing a Government Ethics Nightmare - Newsweek

This entry was posted on August 1, 2017 at 7:51 pm and is filed under Genetic medicine. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |