US2016368975A1PendingUtilityA1
Methods and compositions for the treatment of beta-thalassemia
Assignee: INST NAT DE LA SANTE ET DE LA RECH MEDICALE (INSERM)Priority: Nov 20, 2013Filed: May 23, 2016Published: Dec 22, 2016
Est. expiryNov 20, 2033(~7.3 yrs left)· nominal 20-yr term from priority
G01N 2800/22G01N 33/5094A61K 31/7088A61K 31/70G01N 33/6893G01N 2333/805C07K 16/18A61K 38/02G01N 2500/02G01N 33/80
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Claims
Abstract
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.
Claims
exact text as granted — not AI-modified1 . 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.
2 . The method of claim 1 , wherein said preventing is achieved by administering to said subject a compound that inhibits binding of α globin to Hsp70.
3 . The method of claim 2 , wherein said compound that inhibits binding of α globin to Hsp70 is a small molecule that binds to the α chain binding pocket of Hsp70.
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