Abstract:Hematopoiesis is sustained throughout fetal and adult life by hematopoietic stem cells (HSCs) that are defined by their self‐renewal capacity, pluripotentiality, and their ability to repopulate myeloablated recipients. HSCs have therefore become important targets for transplantation and somatic gene therapy. Models of HSC gene therapy in mice and large animals have reproducibly demonstrated that a significant proportion of cells participating in long‐term engraftment can be stably transduced by integrating vec… Show more
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