Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...

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

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

Dec 6 (Reuters) - Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with ...

A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...

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

Exa-cel could provide benefit before kids develop chronic organ damage ...

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger ...

A one-year-old baby boy has received an incredible honour, for helping researchers get one step closer to finding treatment ...

Researchers at UT Southwestern Medical Center have identified a protein that causes human cell membranes to break open in a ...