Membership
Get unlimited digital access. Try it today for only $0.99.
Topics

Medical Research

With a deft snip, potential treatment emerges for deadly childhood Duchenne Muscular Dystrophy

With a deft snip, potential treatment emerges for deadly childhood Duchenne Muscular Dystrophy

Using cells from patients with Duchenne Muscular Dystrophy, a genetic disease that affects one in 5,000 boys, UCLA scientists have devised a strategy for creating "corrected" stem cells that could restore tissue under attack by the deadly muscle-wasting disorder.

A gene-editing procedure described by researchers Thursday in the journal Cell Stem Cell resulted in a gene that functions properly, coding for the production of proteins called dystrophins, which are deficient in people with Duchenne Muscular Dystrophy (DMD). It could lead to one of the...

Loading
59°