*A gene therapy that may eliminate, or significantly reduce, the need for daily insulin injections is preparing to enter ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Scientists have developed a new gene therapy that quiets pain at its source in the brain—without the addictive risks of opioids. Using AI to map how pain is processed, they created a targeted “off ...

A phase 1/2 trial of a novel viral vector gene therapy in adults with type 1 diabetes is scheduled to begin this year.

The rare pediatric disease affects approximately one in one million children globally. Mutations in the ITGB2 gene disrupt ...

NANJING, China — A groundbreaking clinical trial has achieved what many thought impossible: restoring meaningful hearing in people born profoundly deaf, including teenagers and young adults who were ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine This voice experience is generated by AI. Learn more. This ...

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

I am deeply humbled by the decades of support from people around the world. It is an honor to share this painful yet ...

It involves substances “not for human use.” ...