Monogenic disorders (MGDs), which are caused by single gene mutations, have a serious
effect on human health. Among these, β-thalassemia (β-thal) represents one of the most …

Abstract β-Thalassemia is one of the most common genetic blood diseases and is caused by
either point mutations or deletions in the β-globin (HBB) gene. The generation of patient …

β-Thalassemia (β-Thal) is one of the most common genetic diseases in the world. The
generation of patient-specific β-Thal-induced pluripotent stem cells (iPSCs), correction of the …

β-thalassemia, one of the most common genetic diseases worldwide, is caused by mutations
in the human hemoglobin beta (HBB) gene. Creation of human induced pluripotent stem …

The generation of beta-thalassemia (β-Thal) patient-specific induced pluripotent stem cells
(iPSCs), subsequent homologous recombination-based gene correction of disease-causing …

The generation of personalized induced pluripotent stem cells (iPSCs) followed by targeted
genome editing provides an opportunity for developing customized effective cellular …

Background Thalassemia is the most common genetic disease worldwide; those with severe
disease require lifelong blood transfusion and iron chelation therapy. The definitive cure for …

Abstract β-thalassemia results from point mutations or small deletions in the β-globin (HBB)
gene that ultimately cause anemia. The generation of induced pluripotent stem cells (iPSCs) …

Precise and accurate gene correction is crucial for enabling iPSC-based therapies, and
Cas9-Nickase based approaches are increasingly considered for in vivo correction of …

Abstract β-thalassemias (β-thal) are a group of blood disorders caused by mutations in the β-
globin gene (HBB) cluster. β-globin associates with α-globin to form adult hemoglobin (HbA …