The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Beta-hemoglobinopathies are the most common genetic disorders worldwide, caused by a
wide spectrum of mutations in the β-globin locus, and associated with morbidity and early …

Ex vivo gene editing in hematopoietic stem and progenitor cells (HSPCs) represents a
promising curative treatment strategy for monogenic blood disorders. Gene editing using the …

Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …

β-Thalassemia is brought about by defective β-globin (HBB [hemoglobin subunit β])
formation and, in severe cases, requires regular blood transfusion and iron chelation for …

β-Thalassemia and sickle cell disease are autosomal recessive disorders of red blood cells
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …

Recent studies have shown that base editing, even with single-strand breaks, could result in
large deletions of the interstitial regions while targeting homologous regions. Several …

β654-thalassemia is caused by a point mutation in the second intron (IVS-II) of the β-globin
gene that activates a cryptic 3′ splice site, leading to incorrect RNA splicing. Our previous …

Abstract Beta-thalassemia (β-thalassemia) is an autosomal recessive disorder caused by
point mutations, insertions, and deletions in the HBB gene cluster, resulting in the …

The European Cooperation in Science and Technology (COST) is an intergovernmental
organization dedicated to funding and coordinating scientific and technological research in …