A team of researchers from the U.S. and Germany describes a novel CRISPR approach to produce healthy heart muscle using pluripotent stem cells from Duchenne muscular dystrophy (DMD) patients. In their ...

ALAMEDA, Calif.--(BUSINESS WIRE)-- Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced its participation in the ...

Left: Muscle fibers expressing dysferlin (purple) made from gene-edited muscle stem cells transplanted into a mouse that lacks dysferlin. Right: Muscle fibers from the recipient mouse that are ...

NEW HAVEN, Conn. — Terry Horgan is a 26-year-old who works at Cornell University. He’s very tech savvy. Currently, he gets around in a motorized wheelchair due to a rare disease called Duchenne ...

Disorders like muscular dystrophies are difficult to treat using gene therapies because of a size problem. The dysfunctional genes in these conditions are often very big, and current methods used in ...

Armed with $68 million in Series B funding, Epicrispr Biotechnologies (Epic) is initiating human trials of its lead candidate EPI-321, an epigenetic therapy for the neuromuscular disorder, ...

A new study from UT Southwestern suggests that more people with Duchenne muscular dystrophy could live longer by identifying and more aggressively treating patients with certain risk factors. The ...