He Jiankui, who shocked the world in 2018 by announcing the creation of the first gene-edited babies, tells NPR he's now working on a cure for Duchenne muscular dystrophy.
A panel of experts voted 8-6 in favor of Food and Drug Administration approval of the first gene therapy for Duchenne muscular dystrophy, a fatal genetic disease.
The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.
Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.
