| 发明名称 |
METHODS AND COMPOSITIONS FOR THE TREATMENT OF BETA-THALASSEMIA |
| 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. |
| 申请公布号 |
US2015141341(A1) |
申请公布日期 |
2015.05.21 |
| 申请号 |
US201414547208 |
申请日期 |
2014.11.19 |
| 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 |
发明人 |
HERMINE Olivier;COURTOIS Genevieve;ARLET Jean-Benoit;RIBEIL Jean-Antoine |
| 分类号 |
A61K38/16;G01N33/68 |
主分类号 |
A61K38/16 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method of restoring or increasing erythrocyte maturation in a subject suffering from β-thalassemia major (β-TM) by preventing proteolytic inactivation of GATA-1. |
| 地址 |
Paris FR |
