β-Thalassemias
Alì Taher, Khaled M. Musallam, Maria Domenica Cappellini
Abstract
Defective synthesis of the β-globin chain causes recessively inherited disorders characterized by inadequate hemoglobin production and chronic anemia. Transfusion may lead to iron overload. Hematopoietic stem-cell transplantation may be curative. Gene therapy holds promise for reversing anemia.
Topics & Concepts
ThalassemiaHaematopoiesisAnemiaMedicineHematopoietic stem cell transplantationGenetic enhancementGlobinHemoglobinImmunologyStem cellBiologyTransplantationGeneGeneticsInternal medicineHemoglobinopathies and Related DisordersIron Metabolism and DisordersPrenatal Screening and Diagnostics