Treating sickle cell disease by targeting HbS polymerization
Treating sickle cell disease by targeting HbS polymerization
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DOI：
10.1182/blood-2017-02-765891
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发表时间：
2017-05-18
期刊：
影响因子：
20.3
通讯作者：
Bunn, H. Franklin
中科院分区：
文献类型：
--
作者：
Eaton, William A.;Bunn, H. Franklin
尽管镰状细胞病的根本原因是血红蛋白S（HbS）聚合形成纤维，使红细胞缺乏弹性，但目前在临床试验中评估的大多数药物都针对这一原发事件的下游后遗症。很少有人注意到可以抑制纤维形成的多种方式的研究。在这篇文章中，我们描述了5种不同的方法来抑制聚合的分子原理，并讨论了目前在临床试验中的几种抗聚合药物的进展。
Although the root cause of sickle cell disease is the polymerization of hemoglobin S (HbS) to form fibers that make red cells less flexible, most drugs currently beingassessedin clinical trials are targeting the downstream sequelae of this primary event. Less attention has been devoted to investigation of the multiple ways in which fiber formation can be inhibited. In this article, we describe the molecular rationale for 5 distinct approaches to inhibiting polymerization and also discuss progress with the few antipolymerization drugs currently in clinical trials.