The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

CRISPR gene editing shows potential for treating genetic diseases. Explore how it may impact Down syndrome treatment and ...

CRISPR shows promise for curing rare diseases that have no cure. KJ’s case is a landmark for researchers, who showed that bespoke therapy can be created and delivered to patients in a short ...

Supported by $20 million from the Chan Zuckerberg Initiative, the center focuses on treating rare genetic diseases in children, starting with a group of eight kids who will enroll in a clinical ...

Duchenne Muscular Dystrophy (DMD), hemophilia A and B, HIV, inherited retinal disease (IRD). CRISPR expects its cardiovascular disease treatments CTX310 and CTX320 top-line results to be present ...

Data on DMD Treatment From MDA 2025 Duchenne muscular dystrophy (DMD) is a severe, progressive X-linked genetic disorder caused by mutations in the gene encoding the dystrophin protein. Without ...

Body of work For nearly 20 years, Spuler and her collaborators have been working to understand dysferlin, its role in muscular dystrophy and ways to cure these rare but devastating inherited diseases.

Duchenne Muscular Dystrophy (DMD), hemophilia A and B, HIV, inherited retinal disease (IRD). CRISPR expects its cardiovascular disease treatments CTX310 and CTX320 top-line results to be present ...