This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.

To the research team working to save him, KJ Muldoon was first known only as Patient Eta. But within months, KJ’s name — and ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

It's a watershed moment for in vitro fertilization (IVF), which can be difficult and expensive. Doctors are hopeful AI can ...