A new study has found gene therapy can cure a type of “bubble boy disease.”
Wired PR News.com – A potentially deadly condition which affects the body’s immune system, labeled “bubble boy disease” has been found to be treatable with gene therapy. As reported by the Associated Press (AP), a new study which followed the progress of 10 children afflicted with severe combined immunodeficiency (SCID), a genetic disorder that affects between 40 and 100 babies in the United States annually, found the therapy to have successfully treated eight of them. Researchers may now be looking at gene therapy for the treatment of various types of blood cell disorders including sickle cell anemia.
As noted by the AP, SCID was nicknamed “bubble boy disease” after David Vetter, who became known for living in the confines of plastic barriers as a safeguard from germs. Vetter passed away when he was 12 years old in 1984.
The new study tested treatment for a form of SCID less common than the type which affected Vetter. Details about the outcome of the treatment can be found in Thursday’s issue of the New England Journal of Medicine.