10 newborns with 'bubble boy' immune disease cured with gene therapy

10 newborns with 'bubble boy' immune disease cured with gene therapy
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A group of newborns with a rare immune disease known as "bubble boy" have been cured using gene therapy, researchers revealed Wednesday.

Ten babies with severe combined immunodeficiency disease (SCID)–essentially born without a functioning immune system–were treated with a new gene therapy that uses the HIV virus, according to the Washington Post.

Scientists from St. Jude Children’s Research Hospital and UCSF Benioff Children’s Hospital found that the new treatment completely rid the babies of the genetic disorder with no side effects.


The rare genetic condition, which earned its nickname because those affected need to be kept in sterile environments, affects about 1 in 200,000 babies, primarily boys, according to the Associated Press. Those with the disease rarely survive past their first birthdays.

Ewelina Mamcarz, one of the study leaders, told the AP that "the children are cured."

"They came to us as little infants, some of them as young as 2 months, with severe infections," she said. "Now they are home, living normal lives, attending daycare."

A previous attempt to use gene therapy on the disease was cancelled in 2003 after it was found to result in some patients developing leukemia.

Experts say the treatment unveiled Wednesday does not appear have those kinds of side effects, and could be used to find cures for an array of other diseases.

“It’s a game changer,” Jennifer Hemall, a pediatric immunologist at Children’s Hospital of Philadelphia who was not involved in the study, told the Post.

“For immunologists following this disease, gene therapy has always been out there as the hope of the future. It’s exciting to see this wave of treatments actually becoming a reality.”

Other treatments, such as bone marrow transplants, are risky and difficult to obtain, according to reports.