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Muscular Dystrophy

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...

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