新兴疗法。
Emerging Therapies.
作者信息
Guerra Amaliris, Musallam Khaled M, Taher Ali T, Rivella Stefano
机构信息
Department of Pediatrics, Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
International Network of Hematology, 31-33 High Holborn, London WC1V 6AX, UK.
出版信息
Hematol Oncol Clin North Am. 2018 Apr;32(2):343-352. doi: 10.1016/j.hoc.2017.11.002. Epub 2017 Dec 15.
Abstract
At present, the only definitive cure for β-thalassemia is a bone marrow transplant (BMT); however, HLA-blood-matched donors are scarcely available. Current therapies undergoing clinical investigation with most potential for therapeutic benefit are the β-globin gene transfer of patient-specific hematopoietic stem cells followed by autologous BMT. Other emerging therapies deliver exogenous regulators of several key modulators of erythropoiesis or iron homeostasis. This review focuses on current approaches for the treatment of hemoglobinopathies caused by disruptions of β-globin.
摘要
目前，β地中海贫血唯一确切的治愈方法是进行骨髓移植（BMT）；然而，几乎没有人类白细胞抗原（HLA）配型相合的供体。目前正在进行临床研究且最具治疗益处潜力的疗法是对患者特异性造血干细胞进行β珠蛋白基因转移，然后进行自体骨髓移植。其他新兴疗法则提供几种红细胞生成或铁稳态关键调节因子的外源性调节剂。本综述重点关注目前治疗由β珠蛋白破坏引起的血红蛋白病的方法。
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CD34+ cells from dental pulp stem cells with a ZFN-mediated and homology-driven repair-mediated locus-specific knock-in of an artificial β-globin gene.来自牙髓干细胞的CD34+细胞，其通过锌指核酸酶介导和同源驱动修复介导的位点特异性敲入人工β-珠蛋白基因。
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