Hematopoietic stem cell gene therapy

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 vectors based on mammalian retroviruses. Several clinical studies have also shown that conventional gene transfer technology can produce major beneficial therapeutic effects in human patients.