Doctors use HIV in gene therapy to fix 'bubble boy' disease

Combined Shape

Eight babies who were born without a working germ-fighting system had their disease corrected by a gene therapy that was made from one of the immune system’s worst enemies — HIV, the virus that causes AIDS.

The boys had severe combined immunodeficiency syndrome, caused by lack of a gene that helps the immune system form. Without it, babies are unable to fight off infections. It’s also known as “bubble boy disease” because of a famous case in which a boy lived in a protective bubble to shield him from germs.

The therapy uses a version of HIV that’s been altered so it cannot cause disease but can carry the needed gene into the boys’ blood cells.

Results were published Wednesday by the New England Journal of Medicine. The treatment was pioneered by a doctor at St. Jude Children’s Research Hospital in Memphis.

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