Journal article

siRNA-mediated reduction of alpha-globin results in phenotypic improvements in beta-thalassemic cells

Hsiao Phin Joanna Voon, Hady Wardan, Jim Vadolas

HAEMATOLOGICA-THE HEMATOLOGY JOURNAL | FERRATA STORTI FOUNDATION | Published : 2008

Abstract

beta-thalassemia is an inherited hemoglobinopathy caused by defective synthesis of the beta-globin chain of hemoglobin, leading to imbalanced globin chain synthesis. Excess alpha-globin precipitates in erythroid progenitor cells resulting in cell death, ineffective erythropoiesis and severe anemia. Decreased alpha-globin synthesis leads to milder symptoms, exemplified in individuals who co-inherit alpha- and beta-thalassemia. In this study, we investigated the feasibility of utilizing short-interfering RNA (siRNA) to mediate reductions in alpha-globin expression. A number of siRNA sequences targeting murine alpha-globin were tested in hemoglobinized murine erythroleukemic cells. One highly e..

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