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Autologous HSC Transplant May Treat Sickle Cell Disease

(SCD) is an autosomal recessive disorder caused by mutations in (HBB) that deform .

A small number of patients have been successfully treated with allogeneic hematopoietic stem cell (HSC) transplantation; however, there are several drawbacks and complications associated with this procedure, including graft vs. host disease and long-term immune suppression. Many of complications could potentially be avoided by the use of autologous HSC transplant, which uses patient cells that have been treated genetically modified to replace defective hemoglobin.

In this issue of the , and colleagues investigated the utility of a genetic vector encoding a human hemoglobin gene engineered to impede sickle hemoglobin and prevent the sickling of red blood cells. The vector was used to efficiently transduce bone marrow cells from and the transduced cells were successfully transplanted into immunocompromised mice, suggesting that this method could potentially be used to treat SCD.

TITLE: β-globin gene transfer in human bone marrow for sickle cell disease



Journal of Clinical Investigation