Recent developments in the use of pyruvate kinase activators as a new approach for treating sickle cell disease
Dina S. Parekh, William A. Eaton, Swee Lay Thein
Abstract
ABSTRACT: Pyruvate kinase (PK) is a key enzyme in glycolysis, the sole source of adenosine triphosphate, which is essential for all energy-dependent activities of red blood cells. Activating PK shows great potential for treating a broad range of hemolytic anemias beyond PK deficiency, because they also enhance activity of wild-type PK. Motivated by observations of sickle-cell complications in sickle-trait individuals with concomitant PK deficiency, activating endogenous PK offers a novel and promising approach for treating patients with sickle-cell disease.
Topics & Concepts
Pyruvate kinase deficiencyPyruvate kinaseGlycolysisSickle cell traitAdenosine triphosphateEnzymeCellDiseaseMedicineBiochemistryChemistryBiologyInternal medicineHemoglobinopathies and Related DisordersErythrocyte Function and PathophysiologyBlood groups and transfusion