Gene Therapy Continues to Benefit Kids with Immunodeficiency

Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.

Written byJef Akst

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More than two years after receiving a gene therapy treatment intended to restore immune functioning, 48 kids with severe combined immunodeficiency are healthy, according to a study published yesterday (May 11) in The New England Journal of Medicine.

“We’re taking what otherwise would have been a fatal disease,” and largely fixing the problem, study coauthor Donald Kohn of Mattel Children’s Hospital at the University of California, Los Angeles, tells the Associated Press. Now, he adds, “[t]hey’re basically ‘free range’—going to school, doing normal things,” without fear of a life-threatening infection.

“People ask us, is it a cure? Who knows long term, but at least up to three years, these children are doing well,” Stephen Gottschalk of St. Jude Children’s Research Hospital in Memphis who was not involved in the study but has tested a similar gene therapy, tells the AP. “The immune function seems stable over ...

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Meet the Author

Jef Akst
Jef (an unusual nickname for Jennifer) got her master’s degree from Indiana University in April 2009 studying the mating behavior of seahorses. After four years of diving off the Gulf Coast of Tampa and performing behavioral experiments at the Tennessee Aquarium in Chattanooga, she left research to pursue a career in science writing. As The Scientist's managing editor, Jef edited features and oversaw the production of the TS Digest and quarterly print magazine. In 2022, her feature on uterus transplantation earned first place in the trade category of the Awards for Excellence in Health Care Journalism. She is a member of the National Association of Science Writers.
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