Base editing by nucleotide deaminases linked to programmable DNA-binding proteins
represents a promising approach to permanently remedy blood disorders, although its …

Sickle cell disease (SCD) is a prevalent, life-threatening condition attributable to a heritable
mutation in β-hemoglobin. Therapeutic induction of fetal hemoglobin (HbF) can ameliorate …

Naturally occurring, large deletions in the β-globin locus result in hereditary persistence of
fetal hemoglobin, a condition that mitigates the clinical severity of sickle cell disease (SCD) …

Hematopoietic stem cells (HSCs) have a number of unique physiologic adaptations that
enable lifelong maintenance of blood cell production, including a highly regulated rate of …

Over the last decade, incrementally improved xenograft mouse models, which support the
engraftment and development of a human hemato-lymphoid system, have been developed …

Gene editing following designer nuclease cleavage in the presence of a DNA donor
template can revert mutations in disease-causing genes. For optimal benefit, reversion of the …

Disorders involving β-globin gene mutations, primarily β-thalassemia and sickle cell
disease, represent a major target for hematopoietic stem/progenitor cell (HSPC) gene …

Despite advances in defining diverse somatic mutations that cause myeloid malignancies, a
significant heritable component for these cancers remains largely unexplained. Here, we …

Abstract β-Thalassemia is a genetic form of anemia due to mutations in the β-globin gene,
that leads to ineffective and extramedullary erythropoiesis, abnormal red blood cells and …

Background A point mutation in sickle cell disease (SCD) alters one amino acid in the β-
globin subunit of hemoglobin, with resultant anemia and multiorgan damage that typically …