发明名称 Modulation of BCL11A for treatment of hemoglobinopathies
摘要 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.
申请公布号 US8383604(B2) 申请公布日期 2013.02.26
申请号 US200913063524 申请日期 2009.09.14
申请人 CHILDREN'S MEDICAL CENTER CORPORATION;PRESIDENT AND FELLOWS OF HARVARD COLLEGE;ORKIN STUART H.;SANKARAN VIJAY G. 发明人 ORKIN STUART H.;SANKARAN VIJAY G.
分类号 C07H21/02;C07K14/00;C12N5/071;C12N15/113 主分类号 C07H21/02
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