domingo, 28 de junio de 2020

A Year In, 1st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving

https://www.npr.org/sections/health-shots/2020/06/23/877543610/a-year-in-1st-patient-to-get-gene-editing-for-sickle-cell-disease-is-thriving?utm_source=Nature+Briefing&utm_campaign=d31683ec12-briefing-dy-20200624&utm_medium=email&utm_term=0_c9dfd39373-d31683ec12-44992633

Victoria Gray, who underwent a landmark treatment for sickle cell disease last year, has been at home in Forest, Miss., with her three kids, Jadasia Wash (left), Jamarius Wash (second from left) and Jaden Wash.
Victoria Gray

CRISPR for sickle-cell, one year on

As the one year anniversary of her receiving a ground-breaking treatment for sickle-cell disease approaches, it’s good news for Victoria Gray — the first person in the United States to undergo CRISPR gene therapy to treat a genetic disorder. It’s too soon for scientists to reach firm conclusions about the long-term safety and effectiveness of the approach. But for Gray, life is very different from the severe pain attacks and frequent blood transfusions she experienced before. “It's hard to put into words the joy that I feel — being grateful for a change this big. It's been amazing,” said Gray.
NPR | 7 min read or 6 min listenRead more: Gene therapy is facing its biggest challenge yet: sickle-cell disease (Nature | 13 min read, from December)

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