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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 ...
