First Patient Receives In Vivo CRISPR Editing

Doctors in Oregon delivered the gene editing machinery behind the retina in hopes of treating an inherited form of blindness, according to the companies that developed the therapy.

Jef Akst

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Update (November 17, 2022): Only 3 out of 14 patients experienced “clinically meaningful improvements” when treated with the CRISPR-based therapy in a Phase 1/2 trial, Editas announced today. The company added that it is pausing enrollment but is looking for a collaborator to continue development. “We’ve demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes,” Editas President and CEO Gilmore O’Neill said in the announcement.

Cambridge, Massachusetts–based Editas Medicine and Dublin-based Allergan announced today (March 4) that doctors at the Casey Eye Institute of Oregon Health & Science University in Portland used CRISPR gene editing inside a patient for the first time. They are attempting to treat an inherited form of blindness called Leber congenital amaurosis, the Associated Press reports. The scientists say they will know within a few weeks if the treatment is working and ...

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Meet the Author

Jef Akst
Jef Akst was managing editor of The Scientist, where she started as an intern in 2009 after receiving a master’s degree from Indiana University in April 2009 studying the mating behavior of seahorses.
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