Gene therapy delivers DNAinto a patients 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 tofight off disease. The DNA for a gene or genes is carried into a patientscells by a delivery vehicle called a vector, typically a specially engineeredvirus. The vector then inserts the gene(s) into the cells' DNA.

Although gene therapy is relativelynew and often still considered experimental, it can provide a cure for life-threateningdiseases that dont respond well to other therapies (includingimmunodeficiencies, metabolic disorders, and relapsed cancers) and for acuteconditions that currently rely on complex and expensive life-long medicationand management (such as sickle cell disease and hemophilia).

CAR T-Cell Therapy for Relapsed Acute Lymphoblastic Leukemia (ALL)

Dana-Farber/Boston Childrens is a certified treatment center for providing the recently-FDA-approved CAR T-cell therapy called KYMRIAH for relapsed B-cell acute lymphoblastic leukemia (ALL). This promising new treatment entails genetic engineering of the patients 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.

Our Gene Therapy Clinical Trials

Learn more about our gene therapy clinical trials.

Dana-Farber/BostonChildrens has one the most extensive and long-running pediatric gene therapyprograms in the world. Since 2010, wehave treated 36 patients from 11 countries through eight gene therapy clinicaltrials.

Why choose Dana-Farber/BostonChildrens:

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Gene Therapy for Pediatric Diseases | DNA Therapy - Dana ...