Third-generation, self-inactivating gp91phoxlentivector corrects the oxidase defect in NOD/SCID mouse–repopulating peripheral blood–mobilized CD34+ cells from patients with X-linked chronic granulomatous disease

Abstract excerpt

HIV-1-derived lentivectors are promising for gene transfer into hematopoietic stem cells but require preclinical in vivo evaluation relevant to specific human diseases. Nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice accept human hematopoietic stem cell grafts, providing a unique opportunity for in vivo evaluation of therapies targeting human hematopoietic diseases. We demonstrate for the first...

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