One mom calls it 'amazing'
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. 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.
"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."bubble boy disease"
When doctors first tried it 20 years ago, the treatment had unintended effects on other genes, and some patients later developed leukemia. The new therapy has safeguards to lower that risk.
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