Beta-hemoglobinopathies exhibit a heterogeneous clinical picture with varying degrees of
clinical severity. Pertaining to the limited treatment options available, where blood …

Abstract β-thalassemia is a lethal inherited disease resulting from β-globin gene mutations.
Severe β-thalassemia requires regular blood transfusions. Other active interventions …

Abstract Hemoglobin E (HbE)/β-thalassemia is a form of β-hemoglobinopathy that is well-
known for its clinical heterogeneity. Individuals suffering from this condition are often found …

The B-cell lymphoma/leukemia 11A protein (encoded by BCL11A gene) is a key regulator of
fetal-to-adult hemoglobin switching as seen in post-natal life. Although genetic …

Genome-editing technologies have provided researchers with the ability to edit DNA
mutations associated with various genetic diseases. Prime editing, a 'search-and …

In this short review we have presented and discussed studies on pharmacogenomics (also
termed pharmacogenetics) of the drugs employed in the treatment of β-thalassemia or Sickle …

Therapeutic induction of fetal hemoglobin (HbF) is one of the most promising approaches to
ameliorate the severity of hemoglobinopathies like β-thalassemia and sickle cell anemia …

Sickle cell disease (SCD) is the most common severe monogenetic disorder and displays
marked phenotypic heterogeneity. The clinical diversity of SCD derives from the inter …