2015 Clinical trials update in sickle cell anemia.
2015 Clinical trials update in sickle cell anemia.
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DOI：
10.1002/ajh.24116
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发表时间：
2015-10
影响因子：
12.8
通讯作者：
Brugnara C
中科院分区：
文献类型：
--
作者：
Archer N;Galacteros F;Brugnara C
HBs聚合和细胞镰状是镰状细胞病的主要病理生理事件。在过去的30年里，人们在分子水平上对血红蛋白β链结构中单一氨基酸的变化如何导致HBS聚合物的爆炸性增长以及与之相关的红细胞形态变化有了实质性的了解。氧分压和细胞内HBS浓度是这一过程的主要分子驱动因素，也是开发新疗法的明显目标。然而，聚合和镰刀状正在驱动红细胞内外相关的细胞变化的复杂网络，这些变化成为炎症性血管病变的重要组成部分，并导致大量潜在的急性和慢性器官损害。在这些领域，出现了许多治疗发展的新目标，并有几个正在进行或计划中的新治疗干预措施。这篇综述概述了SCD病理生理学的关键点，因为它们与临床前和临床水平的新疗法的发展有关。
Polymerization of HbS and cell sickling are the prime pathophysiological events in sickle cell disease (SCD). Over the last 30 years, a substantial understanding at the molecular level has been acquired on how a single amino acid change in the structure of the beta chain of hemoglobin leads to the explosive growth of the HbS polymer and the associated changes in red cell morphology. O2 tension and intracellular HbS concentration are the primary molecular drivers of this process, and are obvious targets for developing new therapies. However, polymerization and sickling are driving a complex network of associated cellular changes inside and outside of the erythrocyte, which become essential components of the inflammatory vasculopathy and result in a large range of potential acute and chronic organ damages. In these areas, a multitude of new targets for therapeutic developments have emerged, with several ongoing or planned new therapeutic interventions. This review outlines the key points of SCD pathophysiology as they relate to the development of new therapies, both at the pre-clinical and clinical levels.
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