MicroRNA-targeting therapeutics for hepatitis C
MicroRNA-targeting therapeutics for hepatitis C
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DOI：
10.1007/s12272-013-0318-9
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发表时间：
2014-03-01
影响因子：
6.7
通讯作者：
Min, Hyeyoung
中科院分区：
文献类型：
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作者：
Baek, Jihae;Kang, Soowon;Min, Hyeyoung
MiR-122是一种肝脏特异性microRNA (miRNA)，在调节肝功能如脂质代谢和应激反应中起关键作用。观察到丙型肝炎病毒（HCV）只能在miR-122阳性的肝细胞中复制，导致发现miR-122对HCV复制至关重要，miR-122现在是抗HCV治疗的关键宿主因子之一。目前，最先进的miR-122靶向治疗是SPC3649 (miravirsen)，这是一种锁定的核酸修饰的寡核苷酸，可拮抗miR-122。本综述旨在提供关于SPC3649的发现和开发的信息，SPC3649是第一个进入人体临床试验的mirna靶向药物，并介绍其他正在开发的针对mir -122的丙型肝炎治疗方法。
MiR-122 is a liver-specific microRNA (miRNA) that plays a pivotal role in regulating hepatic functions such as lipid metabolism and stress response. The observation that hepatitis C virus (HCV) could only replicate in miR-122-positive hepatocytes led to the discovery that miR-122 is essential for HCV replication, and miR-122 is now one of the crucial host factors for anti-HCV therapy. Currently, the most advanced miR-122 targeting therapy is SPC3649 (miravirsen), a locked nucleic acid-modified oligonucleotide antagonizing miR-122. This review serves to provide information on the discovery and development of SPC3649, the first miRNA-targeted drug to enter human clinical trials, and introduce other miR-122-targeting therapeutics being developed for hepatitis C.