Gene Therapy Effective for Severe Combined Immunodeficiency

Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”

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ABOVE: Newborn screening is now used in the US to detect severe combined immunodeficiency before infections set in.
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Treating infants with X-linked severe combined immunodeficiency with low-dose chemotherapy followed by gene therapy gave the children the ability to make the cells needed to mount a normal immune response, researchers report today (April 17) in the New England Journal of Medicine. The finding marks a milestone in the long effort to use gene therapy for the devastating condition, also known as bubble boy disease, which requires untreated patients to be isolated in order to protect them from life-threatening infections. Experts caution that longer follow-up is needed to determine whether the gene therapy–treated patients are truly cured.

“We were able to remove the protective isolation within three to four months post gene therapy and send the babies home to their families,” said Ewelina Mamcarz, a pediatric hematologist-oncologist at St. ...

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Meet the Author

Shawna Williams
Shawna was an editor at The Scientist from 2017 through 2022. She holds a bachelor's degree in biochemistry from Colorado College and a graduate certificate and science communication from the University of California, Santa Cruz.
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