Importance Sickle cell disease (SCD) is an inherited disorder of hemoglobin, characterized
by formation of long chains of hemoglobin when deoxygenated within capillary beds …

Sickle cell disease (SCD) is a widespread disease caused by a mutation in the beta-globin
gene that leads to the production of abnormal hemoglobin called hemoglobin S. The …

Background Previous global analyses, with known underdiagnosis and single cause per
death attribution systems, provide only a small insight into the suspected high population …

Genome editing has therapeutic potential for treating genetic diseases and cancer.
However, the currently most practicable approaches rely on the generation of DNA double …

Study objective We provide an updated assessment of trends in sickle cell disease (SCD)–
related mortality, a significant source of mortality in the United States among black persons …

Sickle cell disease (SCD) disproportionately affects Black or African American persons in the
United States and can cause multisystem organ damage and reduced lifespan. Among 178 …

Background Many children with sickle cell disease living in sub-Saharan Africa die before
reaching age 5 years. We estimate the child mortality associated with sickle cell anaemia …

Background For decades, patients with sickle cell disease have had only a limited number of
therapies available. In 2019, voxelotor (1500 mg), an oral once-daily sickle haemoglobin …

Sickle cell disease (SCD) is a severe monogenic disease associated with high morbidity,
mortality, and a disproportionate burden on Black and Hispanic communities. Our objective …

Importance Sickle cell disease (SCD) is a monogenic disorder, yet clinical outcomes are
influenced by additional genetic factors. Despite decades of research, the genetics of SCD …