Gene therapy delivers DNA
into a patient’s cells to replace faulty or missing genes – or adds new genes –
in an attempt to cure diseases or to make changes so the body is better able to
fight off disease. The DNA for a gene or genes is carried into a patient’s
cells by a delivery vehicle called a vector, typically a specially engineered
virus. The vector then inserts the gene(s) into the cells' DNA.
Although gene therapy is relatively
new and often still considered experimental, it can provide a cure for life-threatening
diseases that don’t respond well to other therapies (including
immunodeficiencies, metabolic disorders, and relapsed cancers) and for acute
conditions that currently rely on complex and expensive life-long medication
and management (such as sickle cell disease and hemophilia).
Learn more about our gene therapy clinical trials.
Our CAR T-cell therapy team is made up of experts in leukemia, immunotherapy,
gene therapy, and stem cell transplantation who are experienced in
administering genetically-modified therapies and well-equipped to anticipate
and handle any potential side effects of CAR T-cell therapy. This team is
supported by the #1-ranked children's hospital by U.S. News & World
Report and builds upon Dana-Farber/Boston Children’s world-leading gene therapy program, immunotherapy, expertise, and one of the country's largest stem cell transplant centers. Learn more.
Children’s has one of the most extensive and long-running pediatric gene therapy
programs in the world. Since 2010, we
have treated over 35 patients from 11 countries through eight gene therapy clinical
Why choose Dana-Farber/Boston
Studies led by researchers at Dana-Farber/Boston Children's show the promise of gene therapy in treating an increasing number of pediatric diseases.
CAR (chimeric antigen receptor) T-cell therapy is a promising new treatment for some of the most challenging cases we face in pediatric acute lymphoblastic leukemia (ALL).
Manny shares many loves with his little brother but one thing he wishes they didn't share is sickle cell disease (SCD). By becoming the first patient to participate in a new clinical trial, he's hoping to change that.