- US scientists have used HIV to make a gene therapy that cured eight infants of Severe Combined Immunodeficiency (SCID), or “bubble boy” disease. Results of the research have been published in the New England Journal of Medicine.
- The gene therapy involved collecting the babies’ bone marrow and correcting the genetic defect in their DNA soon after their birth. The “correct” gene which was inserted into was an altered version of HIV, the virus that causes AIDS.
- SCID is caused by a genetic flaw that keeps the bone marrow from making effective versions of blood cells that comprise the immune system. It affects 1 in 2, 00,000 new-borns, mostly males. A bone marrow transplant from a genetically matched sibling can cure SCID, but most people lack a suitable donor and the process is also risky.
- The nickname “bubble boy disease” comes from a famous case in the 1970s where a Texas boy with SCID lived for 12 years in a protective plastic bubble to isolate him from germs.
