Method for treating thalassemia
AssigneeSt. Jude Children's Research Hospital
InventorsMitchell J. Weiss, Mondira Kundu, Christophe Lechauve
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Abstract
Methods for removing excess free α-globin in erythroid cells and treating a thalassemia using an agent that activates Unc-51 Like autophagy activating Kinase (ULK) are described.
Claims7
A method for removing excess free α-globin in reticulocytes or erythrocytes comprising contacting reticulocytes or erythrocytes with an effective amount of an agonist that directly activates Unc-51 Like autophagy activating Kinase (ULK), AMP-Activated Protein Kinase (AMPK), or Glycogen synthase kinase 3 (GSK3) thereby removing excess free α-globin in the reticulocytes or erythrocytes.
A method for removing excess free, insoluble α-globin in reticulocytes or erythrocytes comprising contacting reticulocytes or erythrocytes with an effective amount of an agent that activates Unc-51 Like autophagy activating Kinase (ULK) thereby removing excess free, insoluble α-globin in the reticulocytes or erythrocytes, wherein the agent inhibits Mechanistic Target Of Rapamycin (mTOR).
A method for treating a thalassemia comprising administering to a subject in need thereof an effective amount of an agonist that directly activates Unc-51 Like autophagy activating Kinase (ULK), AMP-Activated Protein Kinase (AMPK), or Glycogen synthase kinase 3 (GSK3) and stimulates ULK1-dependent autophagy thereby treating the subject's thalassemia.
A method for treating a thalassemia comprising administering to a subject in need thereof an effective amount of an agent that activates Unc-51 Like autophagy activating Kinase (ULK), wherein the agent inhibits Mechanistic Target Of Rapamycin (mTOR) and is not an ATP-competitive mTOR kinase inhibitor.
Dependencies are inferred from the claim text.