Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...

Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

Over 500 million people worldwide suffer from diabetes—a disease that contributes to major complications such as stroke, ...

Discover the major highlights from ASCO 2025, according to researchers and drug developers who attended the convention.

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

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study ...

Gene therapies challenge ERT dominance in Gaucher’s disease as new data reveals strategic insights, key players, and future ...