新型抗补体药物：指日可待。
New anti-complement drugs: not so far away.
机构信息
UNIVERSITY OF COLORADO DENVER.
出版信息
Blood. 2014 Mar 27;123(13):1975-6. doi: 10.1182/blood-2014-02-555805.
Abstract
In this issue of Blood, Risitano et al demonstrate that small-molecule inhibitors of C3 cleavage prevent complement activation on erythrocytes from patients with paroxysmal nocturnal hemoglobinuria (PNH); the authors demonstrate that these agents reach therapeutic concentrations after subcutaneous injection in nonhuman primates.
摘要
在本期《Blood》中，Risitano 等人证明了 C3 裂解的小分子抑制剂可防止阵发性睡眠性血红蛋白尿症 (PNH) 患者红细胞上的补体激活；作者证明了这些药物在非人类灵长类动物中皮下注射后可达到治疗浓度。
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本文引用的文献
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Peptide inhibitors of C3 activation as a novel strategy of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria.作为补体抑制的一种新策略，C3 激活肽抑制剂可用于阵发性睡眠性血红蛋白尿症的治疗。
Blood. 2014 Mar 27;123(13):2094-101. doi: 10.1182/blood-2013-11-536573. Epub 2014 Feb 4.
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Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival.依库珠单抗长期治疗阵发性睡眠性血红蛋白尿症：疗效持续且生存改善。
Blood. 2011 Jun 23;117(25):6786-92. doi: 10.1182/blood-2011-02-333997. Epub 2011 Apr 1.
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Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria.补体抑制剂依库珠单抗治疗阵发性夜间血红蛋白尿症患者的多中心3期研究。
Blood. 2008 Feb 15;111(4):1840-7. doi: 10.1182/blood-2007-06-094136. Epub 2007 Nov 30.
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Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria.用于治疗阵发性夜间血红蛋白尿的补体抑制剂依库珠单抗的发现与研发。
Nat Biotechnol. 2007 Nov;25(11):1256-64. doi: 10.1038/nbt1344.
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Relation of putative thioester bond in C3 to activation of the alternative pathway and the binding of C3b to biological targets of complement.补体C3中假定硫酯键与替代途径激活及C3b与补体生物学靶点结合的关系。
J Exp Med. 1980 Oct 1;152(4):1102-14. doi: 10.1084/jem.152.4.1102.
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The membrane attack complex.膜攻击复合物
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