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

机译：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：本发明涉及在细胞中增加胎儿血红蛋白（HBF）水平的组合物和方法。本发明进一步涉及治疗患有血细胞疾病的患者的方法，包括与患有血红蛋白（HBA）的减少量相关的患者，例如镰状细胞疾病和β-Thalassemias。

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公开/公告号US2022017908A1

专利类型
公开/公告日2022-01-20

原文格式PDF
申请/专利权人 FULCRUM THERAPEUTICS INC.;
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申请/专利号US201917295782
发明设计人 PETER RAHL;ANGELA MARIE CACACE;MICHAEL CAMERON;AKSHAY KAKUMANU;
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申请日2019-11-20
分类号C12N15/67;A61K31/7105;C12N15/11;C12N9/22;A61K38/46;
国家 US
入库时间 2022-08-24 23:24:36

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