Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”
Apr 17, 2019
SHAWNA WILLIAMS
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.
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