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.