Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we ...

One of Sam Altman’s pet projects aims to engineer the brain to better work with brain-computer interface implants.

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer’s disease that could help protect the brain from damage and preserve cognitive function ...