Gene Therapy Program

Gene therapy introduces DNA into a child’s cells in an effort to treat or prevent disease. Although still relatively new and often considered experimental, gene therapy is a promising treatment option for a number of diseases that don’t respond well to other therapies, including inherited conditions, immunodeficiencies, metabolic disorders and certain types of cancers.

Our expertise in gene therapy

The gene therapy program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center is one of the largest and experienced pediatric gene therapy programs in the world. In addition, we are a certified treatment center for providing the recently FDA-approved CAR T-cell therapy called KYMRIAH for relapsed B-cell acute lymphoblastic leukemia (ALL).

Learn more about our gene therapy clinical trials.

Why choose the Gene Therapy Program at Dana-Farber/Boston Children’s

The Dana-Farber/Boston Children’s Gene Therapy Program offers immediate access to the in- and out-patient services at two world-leading academic medical institutions — Boston Children’s Hospital and Dana-Farber Cancer Institute. Our efficient enrollment processes allow children to move smoothly and quickly from study enrollment to treatment, and our unique satellite-training module supports referring physicians.

Get more in-depth information about our Gene Therapy Program on the Dana-Farber/Boston Children's Cancer and Blood Disorders website. Learn about: