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).
Our Gene Therapy Clinical Trials
Learn more about our gene therapy clinical trials
Children’s has one the most extensive and long-running pediatric gene therapy
programs in the world. Since 2010, we
have treated 25 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.
Sung-Yun Pai, MD, explains gene therapy.
Born with “bubble boy” disease, Agustin now has a normal life after gene therapy.