Our genes, which hold the code for all of our body's functions, are made of DNA. Damage to DNA, such as a mutation, is an underlying
cause of the genetic defects that lead to cancers, blood disorders, and other conditions. Gene therapy delivers DNA into a patient’s cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off disease.
Scientists are investigating a number of different ways to do this:
With gene therapy, 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.
For patients, the process for delivering genes to cells is relatively simple. See below, or view larger.
Through clinical trials and research, Dana-Farber/Boston
Children's is at the forefront of the gene
therapy evolution. Under the direction of David Williams, MD, Dana-Farber/Boston Children's
is one of the global leaders in pediatric gene therapy clinical research. We are currently conducting gene therapy clinical trials for adrenoleukodystrophy,
X-linked severe combined immunodeficiency, and Wiskott-Aldrich syndrome (see
Gene therapy trials for children with other diseases, such as sickle cell disease, chronic
granulomatous disease, relapsed
acute lymphoblastic leukemia, and various malignant and non-malignant blood disorders are also under development. If successful, these gene therapy trials could produce potential cures for these diseases.
To learn more about the trials below, email Colleen Dansereau or call 1-617-919-7008.
Adrenoleukodystrophy (ALD) is a rare and progressive genetic disorder more often diagnosed in boys that affects the nervous system and the adrenal glands (small glands found on top of the kidneys). The cells of a child with ALD are missing the gene for an enzyme called ALDP, which helps break down fatty acids.
Children who meet the following criteria may be eligible to take part in Dana-Farber/Boston Children's gene therapy trial for children with ALD:
X-linked severe combined immunodeficiency (X-SCID) is a rare genetic immunodeficiency that prevents a child's bone marrow from producing infection-fighting white blood cells.
Children who meet the following criteria may be eligible to take part in Dana-Farber/Boston Children's gene therapy trial for children with X-SCID:
Wiskott-Aldrichsyndrome (WAS) is a rare genetic immunodeficiency that keeps a child's
immune system from functioning properly. It also makes it difficult for a
child's bone marrow to produce platelets, making a child prone to bleeding.
Patients who meet the following criteria may be eligible to
take part in Dana-Farber/Boston Children's gene therapy trial for children with WAS:
Patients under age 5 must also:
An international study led by researchers at Dana-Farber/Boston Children's shows that a new type of gene therapy may help boys with a immune disorder commonly known as “bubble boy” disease.