Method for treating thalassemia
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.
1. 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.
2. 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).
3. 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.
4. 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.
5. The method of claim 3 , wherein the thalassemia is β-thalassemia.
6. The method of claim 4 , wherein the thalassemia is β-thalassemia.
7. The method of claim 2 , wherein the agent that inhibits mTOR is not an ATP-competitive mTOR kinase inhibitor.