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US08383604B2 Modulation of BCL11A for treatment of hemoglobinopathies 有权
调节BCL11A治疗血红蛋白病

Modulation of BCL11A for treatment of hemoglobinopathies
Abstract:
The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
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