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 ...

DMD research has grown significantly, with a surge in studies since 2014, driven by interest in innovative therapies and precision medicine. The US leads in publication output. Advanced therapeutic ...

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 ...

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform gene editing in non-liver tissues, including skeletal muscle, using a ...