Celebrating 10 Years of Gene Therapy










Gene Therapy Program | A Decade of Progress

When 5-month-old Agustín Cáceres received his new gene on Dec. 17, 2010, correcting a severe immune deficiency, he was among the first patients to benefit from a rebirth of gene therapy, and the first patient to receive gene therapy at Boston Children’s Hospital. 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 that historic day, 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.

What a decade of gene therapy means to us...

“The gene therapy team is unlike any other group I’ve been a part of — this team cares about people. The relationships between physician and patient as well as nurse and patient are unmatchable. I’m proud to be a part of a strong, female-dominant research group that changes people’s lives.”
Bridget Kerwin, Clinical Research Coordinator II

“10 years of an amazing journey providing life changing treatments to patients and families, so grateful I have been a part of it!"
Colleen H. Dansereau, Director of Clinical Operations

“10 years of gene therapy means 10 years of unwavering commitment to a smooth patient journey from start to finish."
Pauleen Faynberg, Program Coordinator I

“The opportunity to help bring new curative therapies to children with deadly diseases is both a thrill of a lifetime and a privilege. One only has to see the video of Agustin to understand the impact our program has had on children and their families. It is also a blessing to have such an outstanding team to work with on a daily basis.”
David A. Williams, MD, Chief Scientific Officer and Chief of Hematology/Oncology

10 years of second chances

Gene therapy is bringing new hope for patients 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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