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).
CAR T-Cell Therapy for Relapsed Acute Lymphoblastic Leukemia (ALL)
Dana-Farber/Boston Children’s is actively preparing to offer the recently-FDA-approved CAR T-cell therapy called Kymriah for relapsed B-cell acute lymphoblastic leukemia (ALL). This treatment provides renewed optimism for some of the most challenging cases we face in pediatric ALL. The therapy entails genetic engineering of the patient’s own T-cells to increase targeting of a specific leukemia protein and then accelerate killing of the target. After modification, they are returned to the patient via IV where they can immediately begin destroying circulating cancer cells.
For more information about CAR T-cell therapy, contact our gene therapy program
FDA approves sickle cell gene therapy trial
Another Dana-Farber/Boston Children’s gene therapy innovation — this one designed to correct sickle cell disease — will soon reach the clinic. This therapy suppresses the gene that normally shuts off production of fetal hemoglobin. As a result, healthy fetal hemoglobin can still be produced, compensating and replacing "adult" hemoglobin that stiffen and take on the hallmark "sickle" shape that can trigger an acute sickle cell crisis. The trial has received FDA approval and is expected to open for recruitment in mid-November.
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.
Vietnamese boy thrives after gene therapy for Wiskott-Aldrich syndrome