For a decade after its discovery, CRISPR gene editing was stuck on the cusp of transforming medicine. Then, in 2023, scientists started using it on sickle-cell disease, and Victoria Gray, a patient who lived with constant pain'like lightning inside her body, she has said'got the first-ever FDA-approved CRISPR gene-editing treatment. Her symptoms vanished; so did virtually everyone else's in the clinical trial she was a part of. This year, the technology has started to press beyond its next barrier. Most of the 8 million people globally who have sickle-cell disease share the same genetic mutation; treating rare disorders will require dealing with many different mutations, even within the same disease. And although rare diseases affect 30 million Americans in total, relatively few people are diagnosed with each one. Fyodor Urnov, a scientific director of UC Berkeley's Innovative Genomics Institute (IGI), showed me a list of rare diseases and pointed to one carried by only 50 people....
learn more
