Developing treatment for sickle cell disease.
Sickle cell disease pathophysiology results from sickle haemoglobin polymerisation and its effects on the sickle erythrocyte and the vasculature. Many of the abnormalities of sickle cell disease are secondary to the damage caused by the polymer and the injured red cell. Pharmacological treatment of the disease is focused on the inhibition of sickle haemoglobin polymerisation, prevention or repair of red cell dehydration and interruption of the interaction of sickle cells with the endothelium.
Steinberg
Martin H
MH
Boston University School of Medicine, Boston, Massachusetts 02118, USA. msteinberg@medicine.bu.edu
Brugnara
Carlo
C
D000755:Anemia, Sickle Cell / Q000097:blood / Q000188:drug therapy* / Q000378:metabolism; D000818:Animals; D005319:Fetal Hemoglobin / Q000378:metabolism; D006451:Hemoglobin, Sickle / Q000378:metabolism*; D056572:Histone Deacetylase Inhibitors; D006801:Humans; D007473:Ion Channels / Q000037:antagonists & inhibitors; D008745:Methylation; D009569:Nitric Oxide / Q000378:metabolism
D006451:Hemoglobin, Sickle; D056572:Histone Deacetylase Inhibitors; D007473:Ion Channels; D009569:Nitric Oxide; D005319:Fetal Hemoglobin