Jing Hu, Yebing Zhong, Pengxiang Xu, Liuyan Xin, Xiaodan Zhu, Xinghui Jiang, Weifang Gao, Bin Yang, Yijian Chen
β-Thalassemia is the world's number 1 single-gene genetic disorder and is characterized by suppressed or impaired production of β-pearl protein chains. This results in intramedullary destruction and premature lysis of red blood cells in peripheral blood. Among them, patients with transfusion-dependent β-thalassemia face the problem of long-term transfusion and iron chelation therapy, which leads to clinical complications and great economic stress. As gene editing technology improves, we are seeing the dawn of a cure for the disease, with its reduction of ineffective erythropoiesis and effective prolongation of survival in critically ill patients...
May 3, 2024: Medicine (Baltimore)