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Researchers in the U.S. have begun editing the genes of adults with devastating diseases, using a tool known as CRISPR. China has already launched multiple trials of CRISPR in humans. Last year Chinese researcher He Jiankui caused a global outcry when he used the same tool to gene edit twin baby girls when they were just embryos. There is far less concern about other CRISPR trials either in the U.S. or China, in part because genetic changes in the adults treated will not be passed on to future generations. “If it’s done well and carefully, I’m not so worried, to be honest,” says Robin Lovell-Badge, a British geneticist and stem cell scientist, regarding the use of CRISPR in these new trials.

Even so, there are lingering questions about whether it is still too early to move ahead with the technology. CRISPR can, at times, inadvertently edit genes that were not intended to be altered. The fear is that such “off-target edits” could cause other health problems, including cancers. Lovell-Badge, a group leader at the Francis Crick Institute in England, says things can always go wrong, but CRISPR has been adequately vetted in laboratory research, and it is a reasonable time to test the tool in adults.

The fact that CRISPR went from an idea in a lab to a trial in people during this decade speaks to the elegance and versatility of the technology,” says Sam Kulkarni, CEO of the company CRISPR Therapeutics, which is in one  of two groups testing CRISPR-based gene editing approaches in people.

…continue reading ‘Despite Controversy, Human Studies of CRISPR Move Forward in the US’

Image via Flickr AttributionNoncommercial Some rights reserved by National Institutes of Health (NIH)

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Scientific American

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