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Progress Toward the Genetic Treatment of the β‐Thalassemias

1 Nov 2005

Abstract excerpt

The beta-thalassemias are congenital anemias that are caused by mutations that reduce or abolish expression of the beta-globin gene. They can be cured by allogeneic hematopoietic stem cell (HSC) transplantation, but this therapeutic option is not available to most patients. The transfer of a regulated beta-globin gene in autologous HSCs is a highly attractive alternative treatment. This strategy, which is simple...

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PMID: 16339654
DOI: 10.1196/annals.1345.010

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