Results 21 to 30 of about 398,944 (237)
Targeting sickle cell disease root-cause pathophysiology with small molecules
Haematologica, 2019 The complex, frequently devastating, multi-organ pathophysiology of sickle cell disease has a single root cause: polymerization of deoxygenated sickle hemoglobin.
Yogen Saunthararajah
doaj +1 more source
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.
New England Journal of Medicine, 2023 BACKGROUND Sickle cell disease is caused by a defect in the β-globin subunit of adult hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed red cells that hemolyze and cause vaso-occlusion that results in progressive ...
Akshay Sharma +21 more
semanticscholar +1 more source
Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM): a trial for children with sickle cell anemia [PDF]
, 2017 Hydroxyurea treatment is recommended for children with sickle cell anemia (SCA) living in high-resource malaria-free regions, but its safety and efficacy in malaria-endemic sub-Saharan Africa, where the greatest sickle-cell burden exists, remain unknown.
Hodges, James S. +8 more
core +2 more sources
The Scientific World Journal, 2008 The root cause of sickle cell disease is a single β-globin gene mutation coding for the sickle β-hemoglobin chain. Sickle hemoglobin tetramers polymerize when deoxygenated, damaging the sickle erythrocyte.
Martin H. Steinberg
doaj +1 more source
Rapid and inefficient kinetics of sickle hemoglobin fiber growth
Science Advances, 2019 Nanoscale analysis reveals the rapid and inefficient sickle hemoglobin self-assembly process. In sickle cell disease, the aberrant assembly of hemoglobin fibers induces changes in red blood cell morphology and stiffness, which leads to downstream ...
B. Castle, D. Odde, D. K. Wood
semanticscholar +1 more source
Haematologica, 2012 Background Little is known about the effects of blood rheology on the occurrence of acute chest syndrome and painful vaso-occlusive crises in children with sickle cell anemia and hemoglobin SC disease.Design and Methods To address this issue, steady ...
Yann Lamarre +13 more
doaj +1 more source
Advances in Hematology, 2021 Sickle cell disease is a hereditary disease that predominantly affects black people. It is very widespread in sub-Saharan Africa, particularly at the Lehmann “sickle belt” level, where the prevalence of the hemoglobin S involves at least 10% of the ...
Jeannette Bassimbié Kakou Danho +4 more
doaj +1 more source
Drugs for preventing red blood cell dehydration in people with sickle cell disease. [PDF]
, 2016 BACKGROUND: Sickle cell disease is an inherited disorder of hemoglobin, resulting in abnormal red blood cells. These are rigid and may block blood vessels leading to acute painful crises and other complications.
Ballas, Samir K., Nagalla, Srikanth
core +2 more sources
Discovery of GBT440, an Orally Bioavailable R-State Stabilizer of Sickle Cell Hemoglobin
ACS Medicinal Chemistry Letters, 2017 We report the discovery of a new potent allosteric effector of sickle cell hemoglobin, GBT440 (36), that increases the affinity of hemoglobin for oxygen and consequently inhibits its polymerization when subjected to hypoxic conditions.
B. Metcalf +23 more
semanticscholar +1 more source
Understanding Sickle cell disease: Causes, symptoms, and treatment options
Medicine, 2023 Sickle cell disease (SCD) is a hereditary blood disorder characterized by the production of abnormal hemoglobin molecules that cause red blood cells to take on a crescent or sickle shape.
Chukwuka Elendu +8 more
semanticscholar +1 more source