发明公开
- 专利标题： METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT OF MYELODYSPLASTIC SYNDROMES
- 专利标题（中）： 维多利亚药物
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申请号： EP15731531.8申请日： 2015-06-09
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公开(公告)号： EP3154536A1公开(公告)日： 2017-04-19
- 发明人: HERMINE, Olivier , ARLET, Jean-Benoit , GUILLEM, Flavia , COURTOIS, Genevève , FONTENAY, Michaela
- 申请人： Institut National de la Santé et de la Recherche Médicale (INSERM) , Fondation Imagine , Assistance Publique-Hôpitaux de Paris (APHP) , Université Paris Descartes , Centre National de la Recherche Scientifique (C.N.R.S.)
- 申请人地址： 101, rue de Tolbiac 75013 Paris FR
- 专利权人： Institut National de la Santé et de la Recherche Médicale (INSERM),Fondation Imagine,Assistance Publique-Hôpitaux de Paris (APHP),Université Paris Descartes,Centre National de la Recherche Scientifique (C.N.R.S.)
- 当前专利权人： Institut National de la Santé et de la Recherche Médicale (INSERM),Fondation Imagine,Assistance Publique-Hôpitaux de Paris (APHP),Université Paris Descartes,Centre National de la Recherche Scientifique (C.N.R.S.)
- 当前专利权人地址： 101, rue de Tolbiac 75013 Paris FR
- 代理机构： Collin, Matthieu
- 优先权： EP14305873 20140610
- 国际公布： WO2015189186 20151217
- 主分类号： A61K31/165
- IPC分类号： A61K31/165 ; A61P35/02
摘要：
The present invention provides methods and pharmaceutical compositions designed to intervene in this defective process and to promote or restore erythrocyte maturation in individuals suffering from a myelodysplastic syndrome. The methods involve maintaining the activity of GATA-1 by preventing sequestration of Hsp70 in the cytoplasm. Accordingly, it is an object of this invention to provide methods of restoring or increasing erythrocyte maturation in a subject suffering from a myelodysplastic syndrome by preventing proteolytic inactivation of GATA-1. In some embodiments, preventing is achieved by administering to the subject a compound that inhibits the XPO1 nuclear transporter.
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