Current opinion in hematology
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Curr. Opin. Hematol. · May 2006
ReviewRecent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia.
The beta-thalassemias and sickle cell anemia are severe congenital anemias for which there is presently no curative therapy other than allogeneic hematopoietic stem cell transplantation. This therapeutic option, however, is not available to most patients due to the lack of an HLA-matched bone marrow donor. The transfer of a regulated globin gene in autologous hematopoietic stem cells is therefore a highly attractive alternative treatment. This strategy, simple in principle, raises major challenges in terms of controlling transgene expression, which ideally should be erythroid specific, differentiation and stage restricted, elevated, position independent, and sustained over time. ⋯ These recent advances bode well for the clinical investigation of stem cell-based gene therapy in the severe hemoglobinopathies.