A study out Wednesday details how scientists turned this enemy virus into a savior, altering it so it couldn't cause disease and then using it to deliver a gene the boys lacked.
They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with"bubble boy disease" have had it fixed by a gene therapy made from one of the immune system's worst enemies — HIV, the virus that causes AIDS.
"For a long time we didn't know what was wrong with him. He just kept getting these infections," said his mother, Kristin Simpson. Learning that he had SCID"was just heartbreaking ... I didn't know what was going to happen to him." 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 200,000 newborns, almost exclusively males. Without treatment, it often kills in the first year or two of life.The nickname"bubble boy disease" comes from a famous case in the 1970s — a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs.
A small study of older children suggested it was safe. The new study tried it in infants, and doctors are reporting on the first eight who were treated at St. Jude and at UCSF Benioff Children's Hospital San Francisco.
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