Movies love a good distraction. While you’re watching the action, something subtle plays out in the corner. That quiet thing ...

A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy.

Nicole Verdun, top gene therapy regulator at the FDA, has been forced out of the agency.

Following a second patient death, Sarepta is temporarily suspending use of Elevidys in non-ambulatory DMD patients, pending evaluation of an enhanced immunosuppression regimen.

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular dystrophy.

Sarepta halts Elevidys shipments for non-ambulatory DMD patients after a second patient death in three months post gene therapy treatment.

SALT LAKE CITY — In this Healio Video Perspective from the ARVO meeting, SriniVas R. Sadda, MD, FARVO, discusses a long-term study evaluating MCO-010 for the treatment of retinitis pigmentosa.

Researchers at the University of Pennsylvania believe they’re one step closer to finding a treatment that fights glioblastoma, the deadliest form of brain cancer.

Baby KJ Muldoon was born with a rare genetic disorder called CPS1 deficiency and spent nearly the entire first year of his life in the hospital. Now, he’s finally home after becoming the first ...

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 ...

By reprogramming brain cells, a new gene therapy approach for Alzheimer's developed by UC San Diego researchers could address the root cause of the disease to halt its progression.