Epigenome editing has rapidly evolved in recent years, with diverse applications that
include elucidating gene regulation mechanisms, annotating coding and noncoding …

Advances in the technologies and informatics used to generate and process large biological
data sets (omics data) are promoting a critical shift in the study of biomedical sciences. While …

Gene editing to disrupt the GATA1-binding site at the+ 58 BCL11A erythroid enhancer could
induce γ-globin expression, which is a promising therapeutic strategy to alleviate β …

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

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

Background Sickle cell disease is characterized by hemolytic anemia, pain, and progressive
organ damage. A high level of erythrocyte fetal hemoglobin (HbF) comprising α-and γ …

Fetal hemoglobin (HbF, α 2 γ 2) level is genetically controlled and modifies severity of adult
hemoglobin (HbA, α 2 β 2) disorders, sickle cell disease, and β-thalassemia. Common …

The human gut microbiome is a complex ecosystem that is involved in its host's metabolism,
immunity and health. Although interindividual variations in gut microbial composition are …

β-Thalassemias Defective synthesis of the β-globin chain causes recessively inherited
disorders characterized by inadequate hemoglobin production and chronic anemia …

Abstract β-hemoglobinopathies such as sickle cell disease (SCD) and β-thalassemia result
from mutations in the adult HBB (β-globin) gene. Reactivating the developmentally silenced …