Learn about FDA-approved gene therapies from the Gene Therapy Program.
… adrenoleukodystrophy (CALD), but also highlight a risk for cancer. Read more Duchenne muscular dystrophy gene therapy … body's T-cells to better detect and destroy infected or cancerous cells. It is also the first FDA-approved CAR T-cell therapy. The Dana-Farber/Boston Children's Cancer and Blood Disorders Center is offering KYMRIAH® to …