医学
地中海贫血
贫血
造血
遗传增强
造血干细胞移植
珠蛋白
血红蛋白
干细胞
移植
免疫学
基因
内科学
遗传学
生物
作者
Alì Taher,Khaled M. Musallam,Maria Domenica Cappellini
标识
DOI:10.1056/nejmra2021838
摘要
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.
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