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Gene Therapy for SCID-X1

Scientists from St. Jude Children's Research Hospital have used a gene therapy to treat infants newly diagnosed with X-linked severe combined immunodeficiency (SCID-X1), also known as "bubble boy disease," the Memphis Commercial Appeal reports. Infants with this condition are born without working immune system, NPR adds.

The team led by St. Jude's Brian Sorrentino — who recently died — treated bone marrow stem cells from eight infants with SCID-X1 with a lentiviral vector carrying normal copy of the IL2RG gene. Those altered cells were then transferred back to the infants after they were treated with low levels of busulfan, a chemotherapy drug. As they report in the New England Journal of Medicine this week, the researchers followed these infants for a median 16.4 months to find seven infants had normal levels of certain immune cells three to four months later, while the eighth infant needed an additional boost of treated cells before responding.

"This novel approach has shown really outstanding results for the infants," James Downing, the CEO of St. Jude's, tells the Commercial Appeal. "The treatment has fully restored the immune system in these patients, which wasn't possible before, and has no immediate side effects."

The Associated Press adds that the hospital has licensed the rights to the treatment to Mustang Bio.