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O’Reilly Cell Manipulation Laboratory at Dana-Farber (Dana-FarberPhoto)

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus–based γ-retrovirus vector expressing interleukin-2 receptor γ-chain (γc) complementary DNA successfully restored immunity in most patients but resulted in vector-induced leukemia through enhancer-mediated mutagenesis in 25 percent of patients. Researchers assessed the efficacy and safety of a self-inactivating retrovirus for the treatment of SCID-X1.

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