• Gene Therapy for Children


    Through clinical trials and research, Dana-Farber/Boston Children's Cancer and Blood Disorders Center is at the forefront of the gene therapy evolution. Under the direction of David Williams, MD, Dana-Farber/Boston Children's is one of only three hospitals in the country conducting gene therapy clinical trials for X-linked severe combined immunodeficiency, Wiskott-Aldrich syndrome, adrenoleukodystrophy and relapsed acute lymphoblastic leukemia. Coming soon are gene therapy trials for children with sickle cell disease, which, if successful, could produce potential cures for these diseases.

    Gene Therapy Overview

    Gene therapy introduces genetic material (DNA) into a patient’s cells to prevent or fight cancer. Our genes contain DNA, the code that controls much of our body’s functions. DNA damage is an underlying cause of the genetic mutations that lead to cancer. Gene therapy replaces faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off disease.

    Scientists are investigating a number of different ways to do this:

    • Replacing missing or mutated genes, which is the most common approach
    • Changing the regulation of genes; mutated genes that cause cancer could be turned off so they don’t cause disease or turned on to fight disease.
    • Making cancer cells more recognizable to the body’s immune system to improve the bodies disease-fighting response

    With gene therapy, genes are driven inside a patient’s cells using a delivery vehicle called a vector. These genes have been modified in a laboratory to fortify the bodies’ healthy cells in their fight against the malignant cells. For the patient, the process for delivering genes to cells is relatively simple. Blood is drawn or a liquid sample of bone marrow (bone marrow aspiration) is taken from a patient. In the laboratory, cells from the blood or bone marrow are exposed to a virus or other type of vector containing the desired genetic material. Once this material has entered the cells in the lab, they are injected back into the patient’s body through a vein.

  • Need Help? Speak with Us.

    Our specialized new patient coordinators can answer your questions about treatment options and becoming a patient. In urgent cases, we typically can see new patients within 24 hours.
  • Contact Us: Gene Therapy

    To learn more or refer a patient for a gene therapy clinical trial, contact Colleen Dansereau.
    Phone: 617-919-7008
    Email: colleen.dansereau@childrens.harvard.educallout bg
  • Advances in Gene Therapy: "Bubble Boy Syndrome"

    Dr. David Williams 

    An international study led by researchers at Dana-Farber/Boston Children's shows that a new type of gene therapy may help boys with a immune disorder commonly known as “bubble boy” disease.