Thalassemia is subdivided into α-thalassemia and β-thalassemia, depending on the underlying genetic mutation and affected globin-chain subunits within the hemoglobin tetramer. The α-thalassemias have ...
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9 ...