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Gene Therapy Through Autologous Transplantation of Gene-Modified Hematopoietic Stem Cells

29 Sept 2012

Abstract excerpt

Various genetic diseases of blood cells have been successfully treated with allogeneic hematopoietic stem cell (HSC) transplantation (HSCT) (Table 1). Given that all mature blood cells and their progenitors are derived from HSCs, replacement of genetically defective HSCs with normal donor HSCs replenishes the hematopoietic and lymphoid systems with genetically normal blood cells.

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PMID: 23032601
DOI: 10.1016/j.bbmt.2012.09.021

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