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NEW COMPOSITIONS AND METHODS FOR TREATING BETA-GLOBINOPATHIES

机译：治疗β-球蛋白病的新成分和新方法

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摘要

The present invention provides new compositions and methods useful for the treatment and potential cure of beta-globinopathies such as sickle cell disease and beta-thalassemia by inhibiting the expression and/or activity of RIOK3.

机译：本发明提供了新的组合物和方法，其通过抑制RIOK3的表达和/或活性，可用于治疗和潜在治愈β-球蛋白病，例如镰状细胞病和β-地中海贫血。

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公开/公告号WO2020097261A1

专利类型
公开/公告日2020-05-14

原文格式PDF
申请/专利权人 THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY OF THE DEPARTMENT OF HEALTH AND HUMAN SERVICES;TISDALE JOHN FITZGERALD;GUDMUNDSDOTTIR BJORG;TUMBURU LAXMINATH;
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申请/专利号WO2019US60151
发明设计人 TISDALE JOHN FITZGERALD;GUDMUNDSDOTTIR BJORG;TUMBURU LAXMINATH;
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申请日2019-11-06
分类号C12N15/113;A61K31/713;A61K31;C07K16;A61P7;
国家 WO
入库时间 2022-08-21 11:11:12

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