Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for ...

Cameron Mathison is the son of a Hollywood legend, Tim Mathison, so it's no secret that he was blessed with a pretty fine gene pool. And while he's well-known for his role as Ryan Lavery on All My ...

As dystrophin is also deficient in vital organ systems such as the cardiovascular and respiratory systems, the effect is thus inevitably fatal, with an average survival limited to the third decade of ...

Biofortification is more difficult to implement initially because of the high costs of research and development that go into breeding new varieties of plants. But in some ways, it’s also easier ...

The study, “Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy,” was ... s results “set the stage for … the ...

Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy. Nat Commun. 2025;16(1):120. doi: 10.1038/s41467-024-55086-0 ...

More information: Helena Escobar et al, Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy, Nature ...