Gene Therapy Program

Gene Therapy Program

What is gene therapy?

Gene therapy is the process of introducing DNA into cells in an effort to treat or prevent disease. When a gene mutation (a permanent alteration in the DNA sequence) causes a protein to be missing or faulty, gene therapy may be able to restore the function of that protein by replacing the gene, introducing a healthy copy of the gene or by turning off genes that are causing issues.

The new genes are inserted into cells with either a viral or non viral genetically-engineered vector, a "delivery vehicle" carrying the genetic material. Modified viruses with the infectious capability turned off are used as vectors, because of their natural ability to invade the target cell, delivering the new gene. The vector can be introduced in one of two ways — outside or inside the body.

What is in-vivo gene therapy?

When it's performed in vivo (inside the body), the vector carrying the therapeutic gene is injected directly into the patient's body. 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 — such as immunodeficiencies, metabolic disorders and hematologic disorders — and certain types of cancers.

What is ex-vivo gene therapy?

When it's performed ex vivo (outside the body), the vector with the corrected gene is introduced to a sample of a patient's cells in a laboratory setting, and then transplanted back into the patient.





Our unique approach to gene therapy

The Boston Children's Hospital Gene Therapy Program is one of the leading and largest gene therapy programs in the world. Founded in 2010, our success is a result of our Program’s research and clinical expertise, combined with our ability to translate laboratory discoveries into advances in patient care. 

What makes us unique is our dedicated infrastructure. We provide a core team that focuses on gene therapy as a treatment, collaborating closely with disease experts at two of the world's 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 one-of-a-kind satellite-training module supports referring physicians.

Collaborating to treat rare and difficult-to-treat conditions

Together, along with the Dana-Farber/Boston Children’s Stem Cell Transplant, Blood Disorders and Hematologic Malignancy Centers, as well as academic and pharmaceutical partners in the U.S. and abroad, we have launched and participated in numerous clinical trials. In addition, we are one of the founding members of the Transatlantic Gene Therapy Consortium (TAGTC), a collaboration that seeks to combine the expertise from a number of centers treating rare diseases with different platforms.

Our success stories

David Williams, MD, chief scientific officer, with Patricio, a participant in the ALD gene therapy clinical trial. Find out how gene therapy is making a difference for our patients and families. 

Dr. David Williams, director of the Gene Therapy Program with Patricio, who participated in the ALD Gene Therapy Clinical Trial.
We are proud to serve a broad and diverse patient population that often comes to us for answers they can't find anywhere else. This top ranking is a credit and a tribute to our extraordinary and deeply committed clinicians, researchers and so many others who dedicate every day to improving and advancing the health and well-being of all children.

Sandra L. Fenwick, CEO

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