Gene Therapy Program

Our unique approach to gene therapy

Boston Children's Hospital is an international leader in gene therapy with one of the largest pediatric gene therapy programs in the world. Founded in 2010, our success is a result of our program’s clinical expertise, pioneering gene therapy research, and commitment to caring for rare and difficult-to-treat conditions.

What makes us unique is our dedicated infrastructure. Our core team collaborates 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. We also offer a one-of-a-kind satellite training module to support referring physicians.

Watch: How is gene therapy transforming pediatric health care?

Dr. Christine Duncan, medical director of clinical research and clinical development at the Gene Therapy Program, delves into the transformative impact of gene therapy.

A microscope looking at a drop of blood made of sickled red blood cells.

Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history

The first gene editing therapy for sickle cell disease to be approved by the Food and Drug Administration has deep roots at Boston Children’s Hospital.

Learn more

We have launched and participated in numerous clinical gene therapy trials together with the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. We also work closely with Boston Children’s Departments of Ophthalmology, Neurology, Neurosurgery, and Pulmonary Medicine; Division of Genetics and Genomics; and Metabolism Program, as well as academic and pharmaceutical partners in the U.S. and abroad. We are a founding member of the Transatlantic Gene Therapy Consortium (TAGTC), which seeks to combine the expertise from a number of centers treating rare diseases with different platforms.

Below are some conditions we are currently treating with gene therapy or for which we plan to offer gene therapy in the near future. Some of these are currently being tested in clinical trials, and some have received approval from the U.S. Food and Drug Administration.

Adrenoleukodystrophy (ALD)
Aromatic L-amino acid decarboxylase (AADC) deficiency
Beta thalassemia
Duchenne muscular dystrophy
Hemophilia A
Inherited retinal disorders
Metachromatic leukodystrophy
Sickle cell disease
Spinal muscular atrophy (SMA)
Wiskott-Aldrich syndrome
X-linked severe combined immunodeficiency (SCID-X1)
X-linked chronic granulomatous disease (X-CGD)
Relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL)
Relapsed or refractory large B-cell lymphoma (patients 18 and older)

Ongoing gene therapy research

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 in 2010, we have treated more than 70 patients from 20 countries through more than 19 gene therapy clinical trials.

What is gene therapy?

Read about the different types of gene therapy and how they work.

As gene therapy continues to evolve and improve, our team is optimistically embracing new gene-correction technologies such as gene editing, base editing, and gene silencing and conducting active research to make these techniques ever more precise and safe. We feel fortunate to be at the forefront of this field with our patients and families providing motivation and purpose.