Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …

For over 100 years, clinicians and scientists have been unravelling the consequences of the
A to T substitution in the β-globin gene that produces haemoglobin S, which leads to the …

Abstract Background Deoxygenated sickle hemoglobin (HbS) polymerization drives the
pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has …

Polymerization of deoxygenated hemoglobin S underlies the pathophysiology of sickle cell
disease (SCD). In activating red blood cell pyruvate kinase and glycolysis, mitapivat (AG …

Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic
repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene …

The issue of treating sickle cell disease with drugs that increase hemoglobin oxygen affinity
has come to the fore with the US Food and Drug Administration approval in 2019 of …

Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …

Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases
affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is …

Sickle cell disease is an umbrella term for a group of hemoglobinopathies characterized by
the presence of 2 β-globin gene mutations or deletions, at least 1 of which is the point …

In the 100 years since sickle cell anemia (SCA) was first described in the medical literature,
studies of its molecular and pathophysiological basis have been at the vanguard of scientific …