Abstract
Sickle cell disease (SCD) is caused by a point mutation in the β-globin gene that creates hemoglobin S (HbS). Upon deoxygenation, HbS forms long polymers that distort the shape of red blood cells, causing hemolysis and vaso-occlusion. Voxelotor inhibits HbS polymerization, the root cause of SCD complications. To view this Bench to Bedside, open or download the PDF.
Copyright © 2020. Published by Elsevier Inc.
MeSH terms
-
Anemia, Sickle Cell / epidemiology
-
Anemia, Sickle Cell / genetics*
-
Benzaldehydes / therapeutic use*
-
Hemoglobin, Sickle / antagonists & inhibitors*
-
Hemoglobin, Sickle / genetics
-
Humans
-
Point Mutation / genetics
-
Polymerization / drug effects
-
Pyrazines / therapeutic use*
-
Pyrazoles / therapeutic use*
-
beta-Globins / genetics*
Substances
-
Benzaldehydes
-
Hemoglobin, Sickle
-
Pyrazines
-
Pyrazoles
-
beta-Globins
-
voxelotor