A major goal of hemoglobinopathy research is to develop treatments that correct the
underlying molecular defects responsible for sickle cell disease and β-thalassemia. One …

Abstract The North American Pediatric Aplastic Anemia Consortium (NAPAAC) is a group of
pediatric hematologist‐oncologists, hematopathologists, and bone marrow transplant …

Inflammation, cell adhesion to vascular endothelium, and endothelial injury contribute to
sickle cell anemia (SCA) vaso-occlusion. Although alterations in inflammatory cytokines and …

Tal-effector nucleases (TALENs) are engineered proteins that can stimulate precise genome
editing through specific DNA double-strand breaks. Sickle cell disease and β-thalassemia …

Hydroxyurea, a drug widely used for treating myeloproliferative diseases, has also been
approved for the treatment of sickle cell disease by raising fetal hemoglobin (HbF). We have …

Hydroxyurea has been shown to be efficacious for the treatment of sickle cell anemia (SCA),
primarily through the induction of fetal hemoglobin (HbF). However, the exact mechanisms …

Pharmacological induction of fetal hemoglobin has proven to be a promising therapeutic
intervention in β-hemoglobinopathies by reducing the globin chain imbalance and inhibiting …

Various small molecule pharmacologic agents with different known functions produce
similar outcomes in diverse Mendelian and complex disorders, suggesting that they may …

The switch from fetal (G γ and A γ) to adult (β and δ) globin gene expression occurs at birth,
leading to the gradual replacement of HbF with HbA. Genetic regulation of this switch has …

Abstract Objectives: Fetal hemoglobin (HbF) induction involves NO‐cGMP signaling
pathways. l‐arginine, an NO precursor, and the phosphodiesterase (PDE) 5 inhibitor …