Gene Therapy Program










Gene Therapy Program

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.

Gene Therapy: Magnifying glass zooming in on a DNA strand

Understanding gene therapy

What is  gene therapy? How does gene therapy work? And how is genetic material delivered to a person's cells?

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Entering a new decade

After steady improvements in technique, gene therapy represents a major shift in medicine, offering cures for conditions not previously treatable and for diseases often demanding heroic efforts. Since our founding a decade ago, we have treated more than 70 patients from 20 countries through 19 gene therapy clinical trials.

As the Gene Therapy Program enters its second decade, our team is optimistically embracing new gene-correction technologies such as gene editing and gene silencing. We feel fortunate to be at the forefront of this field with our patients and families providing motivation and purpose.

Because of gene therapy...

Paul

Matthew

Harry

Paul

Matthew

Harry

Our stories

Gene therapy is bringing new hope for children and adults with a wide range of difficult diseases, from sickle cell disease and hemophilia to debilitating brain and muscle diseases to life-threatening immune deficiencies.
Matt, who received gene therapy for hemophilia A, with his family

Hemophilia A

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Brenden Whittaker, who was born with CGD, with David Williams

Chronic Granulomatous Disease

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Eva, who received gene therapy for ALL

Acute Lymphoblastic Leukemia

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Doctor examining a patient who was treated using a gene therapy for sickle cell disease,

Sickle Cell Disease

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Mom holding daughter who has SMA

Spinal Muscular Atrophy

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sofia in her mother

Severe Combined Immunodeficiency

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Family with two sons with ALD

Adrenoleukodystrophy

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Emir, who received WAS gene therapy

Wiskott-Aldrich Syndrome

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Dr. David Williams posing with 2 patients and their family

Other Stories

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The commitment and compassion with which we care for all children and families is matched only by the pioneering spirit of discovery and innovation that drives us to think differently, to find answers, and to build a better tomorrow for children everywhere.

Kevin B. Churchwell, President and CEO

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