• Gene Therapy for Children, Teens and Young Adults

    Gene therapy delivers DNA into a patient’s cells to replace faulty or missing genes – or adds new genes – in an attempt to cure diseases or to make changes so the body is better able to fight off disease. The DNA for a gene or genes is carried into a patient’s cells by a delivery vehicle called a vector, typically a specially engineered virus. The vector then inserts the gene(s) into the cells' DNA.

    Although gene therapy is relatively new and often still considered experimental, it can provide a cure for life-threatening diseases that don’t respond well to other therapies (including immunodeficiencies, metabolic disorders, and relapsed cancers) and for acute conditions that currently rely on complex and expensive life-long medication and management (such as sickle cell disease and hemophilia).

    Our Gene Therapy Clinical Trials

    Learn more about our gene therapy clinical trials

    Gene Therapy at Dana-Farber/Boston Children’s

    Dana-Farber/Boston Children’s has one the most extensive and long-running pediatric gene therapy programs in the world. Since 2010, we have treated 25 patients from 11 countries through eight gene therapy clinical trials.

    Why choose Dana-Farber/Boston Children’s:

    • Our patients have immediate access to the in- and outpatient services of two world leading academic medical institutions – Boston Children’s Hospital and Dana-Farber Cancer Institute.
    • Our concierge services make gene therapy treatment easier for families. These include:
      • assistance with travel and housing for both national and international patients
      • assistance with the insurance process; we have successfully negotiated insurance approval for 80% of our U.S. patients – more than any other gene therapy program
    • We have one of the most efficient enrollment processes in any pediatric gene therapy program – allowing children to move quickly and smoothly from study enrollment into treatment, without losing precious time.
    • Our unique satellite training module supports referring physicians who care for our patients. This approach allows children to return home more quickly without compromising clinical monitoring for safety.
    • We provide a lifetime commitment to care – and will continue to answer questions and address concerns related to the patient’s gene therapy treatment throughout the patient’s life.

    Learn more