Researchers Launch First Study of In Vivo CRISPR Therapy in Humans

A trial is recruiting patients to test the gene-editing technology’s ability to treat an inherited form of blindness caused by a mutation in the CEP290 gene.

Written byCatherine Offord

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People with a rare form of inherited blindness are being enrolled for the world’s first in vivo human study of a CRISPR-based therapy, the Associated Press reported yesterday (July 25). The experimental treatment, developed by Massachusetts-based genome-editing company Editas Medicine and Ireland-based pharmaceutical firm Allergan, removes a deleterious mutation from cells in patients’ retinas, and will be tested in 18 adults and children later this year.

“Today marks an important day for the inherited retinal disease community,” Ben Yerxa, CEO of nonprofit Foundation Fighting Blindness, says in a press statement. “We are very excited to have another potentially life changing medicine enter the clinic and join Allergan and Editas in celebrating this milestone.”

The therapy is designed to treat people with a particular form of a disease known as Leber congenital amaurosis that is caused by a point mutation in a gene called CEP290. This particular ...

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Meet the Author

Catherine Offord
After undergraduate research with spiders at the University of Oxford and graduate research with ants at Princeton University, Catherine left arthropods and academia to become a science journalist. She has worked in various guises at The Scientist since 2016. As Senior Editor, she wrote articles for the online and print publications, and edited the magazine’s Notebook, Careers, and Bio Business sections. She reports on subjects ranging from cellular and molecular biology to research misconduct and science policy. Find more of her work at her website.
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