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Methods and pharmaceutical compositions for the treatment of myelodysplastic syndromes
专利权人:
发明人:
Olivier Hermine,Flavia Guillem,Jean-Benoit Arlet,Genevève Courtois,Michaela Fontenay
申请号:
US15316890
公开号:
US09675590B2
申请日:
2015.06.09
申请国别(地区):
US
年份:
2017
代理人:
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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