Repositioning genes awakens fetal hemoglobin to treat disease. CRISPR editing may change future gene therapy. Researchers ...

Recessive dystrophic epidermolysis bullosa (RDEB) is a systemic disease of poor mucocutaneous basement membrane integrity downstream of bi-allelic pathogenic variants in COL7A1. Cutaneous clinical ...

Dr. Omar Llaguna, specialist in surgical oncology, programs a HistoSonics medical device for targeting a medium that mimics a liver tumor in a human body to demonstrate how a histotripsy procedure ...

A Northeast Ohio woman and a 22-year-old man from UAE are among the first patients to receive the innovative CRISPR treatment ...

The HHS said center directors deserve to be supported by managers aligned with aggressive goals “to expeditiously advance ...

CRISPR Therapeutics’ CRSP first marketed product is the one-shot gene therapy Casgevy, approved in late 2023 and early 2024 across the United States and Europe for two blood disorder indications — ...

Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...

The results of gene therapy for hemophilia B contrast with those of factor replacement therapies, where lifelong infusions ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in ...

Huntington's disease has long defied attempts to rescue suffering neurons. A new study in Cell Reports shows that ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would advance one day.

LEXINGTON, Mass. and NEW YORK, May 12, 2025 /PRNewswire/ -- Genezen, a leading gene therapy CDMO, has partnered with Elly's Team, a foundation dedicated to accelerating the translation of medical ...