Article
Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene
15 Mar 2002
Abstract excerpt
The beta-thalassemias are caused by more than 200 mutations that reduce or abolish beta-globin production. The severity of the resulting anemia can lead to lifelong transfusion dependency. A genetic treatment based on globin gene transfer would require that transgene expression be erythroid specific, elevated, and sustained over time. We report here that long-term synthesis of chimeric hemoglobin...
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