Research ArticleTHALASSEMIA

The autophagy-activating kinase ULK1 mediates clearance of free α-globin in β-thalassemia

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Science Translational Medicine  21 Aug 2019:
Vol. 11, Issue 506, eaav4881
DOI: 10.1126/scitranslmed.aav4881

Unclogging red blood cells

In β-thalassemia, a genetic disorder caused by mutations in the β-globin subunit of adult hemoglobin, the pathological consequences are caused by two problems. One is a shortage of adult hemoglobin that can function to transport oxygen, while the other is a buildup of excess α-globin subunits, which damages the red blood cells and thus further impairs oxygen transport in the body. Using mouse models of β-thalassemia as well as patient-derived cells, Lechauve et al. determined that autophagy-activating kinase ULK1 plays a key role in the clearance of accumulated α-globin. The authors also showed that the drug rapamycin stimulates ULK1-dependent autophagy and thus facilitates α-globin clearance.

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