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For Gene Editing, a 'New Frontier'

If deemed safe, scientists hope for widespread use of CRISPR procedure inside patients' bodies
By Jenn Gidman,  Newser Staff
Posted Mar 4, 2020 8:34 AM CST

(Newser) – "We literally have the potential to take people who are essentially blind and make them see." Those are the attention-grabbing words of Charles Albright, the chief scientific officer at Editas Medicine, which is sponsoring a study to wield DNA technology in a novel way: edit genes using the CRISPR tool inside the patients' bodies. The gene-editing procedure was recently carried out for the first time inside a patient, who happened to have a congenital eye disorder that causes blindness, with the AP calling it a "new frontier" for DNA finagling. It's not clear when the procedure at Oregon's Casey Eye Institute took place, but scientists hope they'll know within months if it worked and how much of the patient's vision was restored, per NPR. "We're really excited about this," says Harvard Medical School's Dr. Eric Pierce, who's in charge of a study about the procedure.

The hourlong surgery involved doctors injecting into the eye three drops of fluid containing a genetically engineered virus, which then travels under the retina and cuts out a mutation in the CEP290 gene, believed to cause Leber congenital amaurosis. And researchers have 18 other patients, both kids and adults, lined up for a similar procedure if the first procedure is deemed a success after a waiting period. The AP notes that even though this is the first time CRISPR has done its work inside a patient's body, in-the-body gene editing took place in 2017 using another tool called zinc fingers. "We're helping open, potentially, an era of gene-editing for therapeutic use that could have impact in many aspects of medicine," Pierce adds to NPR, which adds that brain disorders and muscle conditions like muscular dystrophy could all benefit from such a procedure. (Read more discoveries stories.)

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