Wiskott-Aldrich Syndrome: Lentivirus Gene Therapy Helps 3 Kids
Thursday, July 11, 2013 - 17:40
in Health & Medicine
An international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency has reported results. Wiskott-Aldrich Syndrome is a disorder that weakens the body's immune system. It is caused by a mutation in a gene that encodes the protein WASP. The most often used therapy is a bone marrow or stem cell transplant from a matching donor, often a sibling or relative. It can be curative for some patients, mostly those who have a strongly matching donor. read more