From its earliest conception, gene therapy held the promiseof correcting inherited diseases by inserting a normal copyof the relevant gene into somatic cells.1 Common monogenic diseasesof blood cells, such as sickle cell disease or β-thalassemia,were originally considered important candidates for gene therapybecause they were well understood at the molecular level andbecause the target cell, the hematopoietic stem cell, is easilyaccessible and can be explanted, genetically corrected in thelaboratory, and then retransplanted.2 The advantage of genetherapy over the conventional transplantation of hematopoieticstem cells from compatible donors is that gene therapy is . . . [Full Text of this Article]
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From the Division of Research Immunology–Bone Marrow Transplantation, Childrens Hospital Los Angeles, and the Department of Pediatrics, Keck School of Medicine of the University of Southern California — both in Los Angeles (D.B.K.); and the Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD (F.C.).
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