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COMPOSITIONS AND METHODS FOR INCREASING FETAL HEMOGLOBIN AND TREATING SICKLE CELL DISEASE

机译：用于增加胎儿血红蛋白和治疗镰状细胞病的组合物和方法

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摘要

The present invention relates to compositions and methods of increasing levels of fetal hemoglobin (HbF) in cells. The present invention further relates to methods for treating patients suffering from blood cell diseases, including those associated with reduced amounts of functional adult hemoglobin (HbA), such as sickle cell disease and β-thalassemias.

机译：本发明涉及增加细胞中胎儿血红蛋白（HbF）水平的组合物和方法。本发明还涉及用于治疗患有血细胞疾病的患者的方法，所述血细胞疾病包括与镰状细胞疾病和β地中海贫血等功能性成人血红蛋白（HbA）的量减少有关的患者。

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公开/公告号WO2020106876A2

专利类型
公开/公告日2020-05-28

原文格式PDF
申请/专利权人 FULCRUM THERAPEUTICS INC.;
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申请/专利号WO2019US62461
发明设计人 RAHL PETER;CACACE ANGELA;CAMERON MICHAEL;KAKUMANU AKSHAY;
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申请日2019-11-20
分类号A61K38/46;A61K31/713;A61K48;A61P7;A61P7/06;C12N9/22;C12N15/113;C12N15/63;
国家 WO
入库时间 2022-08-21 11:10:59

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