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

The clustered regularly interspaced short palindromic repeats (CRISPR) renaissance was
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …

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 …

Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB. We used a
custom adenine base editor (ABE8e-NRCH), to convert the SCD allele (HBB S) into …

Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …

Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …

The majority of studies of genetic association with disease have been performed in
Europeans. This European bias has important implications for risk prediction of diseases …

Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB,
which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 …

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