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 ...
The Belmonte lab's advanced in vivo Cas9-based epigenetic gene activation system enhances skeletal muscle mass (top) and fiber size growth (bottom) in a treated mouse (right) compared with an ...
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 ...
For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...
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, ...
NIH-funded UT team and colleagues from Metagenomi Thereapeutics discovered highly efficient enzyme could enable targeted gene editing within the human body.
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 ...