The Latest Breaking News from Gene Therapy

A new gene therapy targeting an enzyme involved in familial hypercholesterolemia slashes bad cholesterol in a small clinical trial.

‘It’s really important that we get the first gene therapy treatment right’

A small, proof-of-concept study in monkeys reveals the potential of a one-off gene therapy to treat people with alcohol use disorder who haven't responded to other treatments.

Narayana Nethralaya to start human clinical trials of indigenous gene therapy for ocular diseases; first such effort in India. Cost of therapy to reduce from ₹7 crore to 1/20th-1/50th of international costs. Meeting of 25 clinicians & scientists held to come out with consensus & establish national registry for rare…

A teen with dystrophic epidermolysis bullosa, or "butterfly disease," was legally blind but has now had his sight restored with gene therapy.

Antonio Vento Carvajal has been legally blind for much of his 14 years

The “chemical cocktail” helped reverse aging in mice, but some experts are skeptical about whether it will work in humans.

U.S. health regulators have approved a gene therapy for the most common form of hemophilia

All five people aboard the Titan are believed dead after an implosion. Saturday marks a year since the Supreme Court overturned Roe. The FDA approves the first gene therapy for muscular dystrophy.