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) results from a single base pair change in the sixth codon of the β-
globin chain of hemoglobin, which promotes aggregation of deoxyhemoglobin, increasing …

For over two decades, the EBMT has updated recommendations on indications for
haematopoietic cell transplantation (HCT) practice based on clinical and scientific …

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

Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …

Gene therapy (GT) provides a potentially curative treatment option for patients with sickle
cell disease (SCD); however, the occurrence of myeloid malignancies in GT clinical trials …

Abstract CRISPR/Cas9-mediated beta-globin (HBB) gene correction of sickle cell disease
(SCD) patient-derived hematopoietic stem cells (HSCs) in combination with autologous …

Background: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that
results in significant complications and affects quality of life. Hematopoietic stem cell …

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 …

Sickle cell disease (SCD) is a group of inherited autosomal recessive disorders, including
sickle cell anemia, sickle beta-thalassemia, and other hemoglobinopathies, which are …