By Heidi Ledford – Nature –
The CRISPR family’s most versatile member has made its medical debut: a cutting-edge gene-editing technique known as prime editing has been used to treat a person for the first time. The recipient is a teenager with a rare immune disorder.
Researchers designed the treatment to correct a mutation that causes chronic granulomatous disease, a dangerous condition that disables a variety of immune cells, including those called neutrophils. A month after the teenager received the treatment, he had experienced no serious side effects. And the therapy seemed to have restored the function of a crucial enzyme in two-thirds of his neutrophils — more than enough to provide a significant boost to his immune system.
/cloudfront-us-east-2.images.arcpublishing.com/reuters/U5VZBBJ5SBJARD5TTCJHHDF6VM.jpg "FDA panel says Vertex/CRISPR to assess safety risks of gene therapy in follow-up study")