| Abstract: |
Sickle cell anemia and Cooley's anemia are in principle excellent candidate diseases for a genetic treatment. The much anticipated development of these therapies raises multiple issues in the realms of stem cell biology, molecular genetics, gene transfer technology and transplantation biology. Fundamental questions still remain to be addressed, particularly with respect to the manipulation of hematopoietic stem cells, gene transfer efficiency, and transgene regulation. Novel therapeutic approaches have been proposed, extending beyond the classical strategy of transducing a regulated β-globin gene in hematopoietic stem cells. The repair of mutations in chromosomal sequences or in RNA, as well as indirect approaches aiming to reactivate the expression of endogenous γ-globin genes are all under investigation. However, the cure of sickle cell anemia or β-thalassemia have not yet been reported in animal models. While the prospects of developing a genetic treatments remain promising, one should not underestimate the challenge of implementing in a clinical setting the concepts demonstrated in the laboratory. |
