Gene therapy restores immune function to infants with SCID-X1 (X-linked severe combined immunodeficiency)
Haberman Associates | Biopharmconsortium Blog
by Allan Haberman, Ph.D
4y ago
IL2RG protein, encoded by tL2RG complementary DNA. ( As reported in the 18 April issue of the New England Journal of Medicine, researchers at the St. Jude Children’s Research Hospital (Memphis, TN) and their colleagues have used gene therapy to restore immune function to eight infants with newly diagnosed X-linked severe combined immunodeficiency (SCID-X1). SCID-X1 is sometimes called “bubble-boy disease”, because of the case of a boy born in 1971 with SCID-X1, who had to be isolated in a plastic bubble while awaiting a bone-ma more
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