发明授权
- 专利标题： Methods and compositions for the treatment of β-thalassemia
- 专利标题（中）： 用于治疗地中海贫血的方法和组合物
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申请号： US14547208申请日： 2014-11-19
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公开(公告)号： US09377471B2公开(公告)日： 2016-06-28
- 发明人: Olivier Hermine , Genevieve Courtois , Jean-Benoit Arlet , Jean-Antoine Ribeil
- 申请人： Institut National de la Sante et de la Recherche Medicale (INSERM) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , Universite Paris Descartes—Paris V , Assistance Publique—Hopitaux de Paris , Fondation Imagine
- 申请人地址： FR Paris FR Paris FR Paris FR Paris FR Paris
- 专利权人： Institut National de la Sante et de la Recherche Medicale (INSERM),CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS),Universite Paris Descartes—Paris V,Assistance Publique—Hopitaux de Paris,Fondation Imagine
- 当前专利权人： Institut National de la Sante et de la Recherche Medicale (INSERM),CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS),Universite Paris Descartes—Paris V,Assistance Publique—Hopitaux de Paris,Fondation Imagine
- 当前专利权人地址： FR Paris FR Paris FR Paris FR Paris FR Paris
- 代理机构： Whitham, Curtis & Cook, P.C.
- 主分类号： G01N33/68
- IPC分类号： G01N33/68 ; A61K38/16 ; G01N33/80
摘要：
Methods and compositions for the treatment of β-thalassemia are provided. Methods and compositions restore or increase erythrocyte maturation in individuals afflicted with β-TM by preventing proteolysis of GATA-1 protein. Screening methods for identifying agents which bind heat shock protein 70 (HSP-70) and inhibit HSP-70 α-globin binding, but which allow GATA-1 protein-HSP-1 binding in a manner that prevents GATA-1 proteolysis.
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