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Primary Immunodeficiency Disorders Program Research and Innovation

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The Primary Immunodeficiency Disorders Program at Boston Children’s Hospital is involved in all areas of basic science, clinical research and state of the art care for patients with primary immunodeficiency. We were one of the first major hospitals to have a clinical immunology program.

X-linked Severe Combined Immune Deficiency Gene Therapy Trial

Boston Children’s is one of three hospitals in the world involved in a gene therapy trial for X-linked Severe Combined Immunodeficiency ? or SCID-X1 — better known as “bubble boy” disease, which makes the body’s immune system very weak and unable to fight common germs. In 2010, Agustin Caceres, a child from Argentina, was able to participate in the SCID-X1 gene therapy trial at Children’s and the Dana Farber’s Cell Manipulation Core Facility. Partnering with physicians in England and France, this international gene therapy trial has treated Agustin and allowed him to get his first hug from his parents.

TACI gene and common variable immunodeficiency

The Geha laboratory is looking at how mutations of the TACI gene are connected to common variable immunodeficiency, one of the most prevalent forms of primary immunodeficiency.

Stem Cell Transplantation

Stem cell transplantation is the only known cure for many of the most severe primary immunodeficiency disorders. Learn more about the stem cell transplantation program at Boston Children’s.

PID Transplant Consortium

Boston Children’s is part of the Primary Immunodeficiency Disorders Transplant Consortium that provides nationwide data about stem cell transplants. Our physicians contribute to and have access to the most recent discoveries and standards of care for stem cell transplantation for primary immunodeficiency disorders.

The future of pediatrics will be forged by thinking differently, breaking paradigms and joining together in a shared vision of tackling the toughest challenges before us.”
- Sandra L. Fenwick, President and CEO