In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, ...

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 ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed. A one-time gene therapy can markedly slow the progression of Huntington’s ...