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Researchers Launch First Study of In Vivo CRISPR Therapy in Humans
Researchers Launch First Study of In Vivo CRISPR Therapy in Humans

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.

Catherine Offord
Catherine Offord

Catherine is a senior editor at The Scientist.

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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.”

See “CRISPR Inches Toward the Clinic

The therapy is designed to treat people with a particular form of a disease known as Leber congenital amaurosis that is caused by...

The trial differs from previous attempts to use CRISPR for therapeutic purposes, which have either been ex vivo (using cells extracted from patients and then replaced post-editing), or, controversially, in embryonic cells. Because the treatment is only applied locally to somatic cells, the changes it makes will not be heritable.

See “CRISPR Scientists Slam Methods Used on Gene-Edited Babies

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