Figure 1.
Combining gene therapy with cell therapy for the treatment of genetic bone marrow disorders. Three strategies for generating autologous pluripotent stem cells are shown and described in the text: (1) Somatic cell nuclear transfer (SCNT); (2) parthenogenesis; and (3) direct somatic cell reprogramming with defined genetic factors. Each of these methods produces a pluripotent stem cell that can be cultured in vitro, which facilitates safe and effective correction of single gene defects. Repaired cells can then be differentiated into desired tissues for transplantation. Differentiation into hematopoietic stem cells (HSCs) enables autologous transplantation and genetically normal hematopoiesis.