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ex-vivo-gene-therapy-retroviral

Ex Vivo Retroviral Gene Therapy for Ada Deficiency Gene therapy for SCID requires the removal of bone marrow cells from the patient. The cells are cultured and the mutant Ada gene is replaced with a functional copy. The bone marrow culture is treated with neomycin to kill nontransformed cells. The bone marrow cells are then replaced in the patient and repopulate the blood supply with normal blood cells.

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