Will consider data that includes "the experience of individuals," FDA commissioner says ...

An Ice Age double burial in Italy has yielded a stunning genetic revelation. DNA from a mother and daughter who lived over 12,000 years ago shows that the younger had a rare inherited growth disorder, ...

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

The study, published in Nature Communications, used family studies and genetically engineered mice to understand how gene ...

New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for BEAM‑304 Anticipated in 2026 ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...

Opus Genetics targets inherited retinal diseases with one-dose AAV gene augmentations. Read why IRD stock is a Buy.

Eric Dane died after battling ALS. Here’s what to know about ALS, the progressive neurological disease that affects nerve ...