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(Newser)
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Eight for eight. Scientists at the St. Jude Children's Research Hospital are delighted to have used gene therapy to treat eight baby boys from the rare disease known as the "bubble boy", reports the BBC. In addition, researchers have used HIV to do so, notes the AP. Infants had the most common form of severe combined immunodeficiency disease, or SCID, called SCID-X1. Essentially, they are born without a functioning immune system, which means that even a cold could be deadly. The solution? The researchers extracted the blood cells from the babies, inserted a genetic solution into the cells, and then reintroduced them into the infants, NPR explains. In this case, the genetic device was administered via a modified version of HIV, which can not give AIDS to babies.
"It's a game changer," says an immunologist at the Philadelphia Children's Hospital not involved in the study. Washington Post. "It's exciting to see this wave of treatment become a reality." Gene therapy had already been successfully tested on SCID, but many previous patients had developed cancer. The last experience published in the New England Journal of Medicine, has safeguards to prevent this, including a small amount of chemotherapy. Bone marrow transplants have also proven effective in treating the disease, but the best case scenario – a "twin donor with paired tissue" – is not available in most cases, say the researchers in a report. communicated. The eight babies in the study are now young children with a healthy immune system. (Read more stories of discoveries.)
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