Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain.

Children and adults born with severe hearing loss caused by mutations in the OTOF gene now have a treatment option that did ...

A new gene therapy is giving people born deaf the chance to hear, often within just weeks. In a small but groundbreaking study, researchers delivered a working copy of a key hearing gene directly into ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

Please provide your email address to receive an email when new articles are posted on . SALT LAKE CITY — In this video, Jay Chhablani, MD, discusses preliminary data from the ArMaDa study of OCU410 ...

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...