1.北京理工大学前沿交叉科学研究院、生命学院,分子医学与生物诊疗重点实验室,北京 100081;2.湖南中医药大学药学院,长沙 410208
国家自然科学基金(31871003),北京理工大学科技创新计划创新人才科技资助专项,北京理工大学青年教师研究基金资助,以及湖南省自然科学基金(2018JJ1019)和湖湘青年人才(2018RS3094)资助项目
1.Advanced Research Institute of Multidisciplinary Science, School of Life Science, Key Laboratory of Molecular Medicine and Biotherapy;2.Beijing Institute of Technology, Beijing 100081, China;3.School of Pharmacy, Hunan University of Chinese Medicine, Changsha 410208, China
This work was supported by grants from The National Natural Science Foundation of China(31871003),the Beijing Institute of Technology Research Fund Program for Young Scholars and the Fundamental Research Funds for the Central Universities of China, the Hunan Provincial Natural Science Foundation of China (2018JJ1019) and the Hu-Xiang Young Talent Program (2018RS3094)
近期,RNA干扰(RNAi)制药公司Alnylam开发的小干扰RNA(siRNA)药物ONPATTRO?(Patisiran)相继获得美国食品与药物管理局和欧盟委员会批准上市,用于治疗成人患者的遗传性转甲状腺素蛋白淀粉样变性引起的多发性神经病变. 这是全球第一款RNAi药物,该事件标志着人类继小分子化合物、单克隆抗体蛋白类药物后,在前沿生物制药领域实现了新的突破,意味着RNAi疗法从基础研究到临床治疗的开发全过程首次贯通,具有里程碑式的意义. 本文概述了该药物及适应症的基本情况,介绍了RNAi发现历史、作用机理与药物特征,RNAi药物的研发历程,以及该领域关键技术的最新研究进展.
Recently, the United States Food and Drug Administration (FDA) and the European Commission (EC) approved ONPATTRO? (Patisiran) lipid complex injection, an RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals, Inc., for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. ONPATTRO is the first approved RNAi therapeutics all over the world, indicating the whole development processes have been well established. It also means a novel form of drug molecule comes to beside from bench, following the small molecules and monoclonal antibodies. Based on this milestone achievement, the basic information of the drug and indication, the mechanism and properties of RNAi therapeutics, its complicated development history, as well as the latest advances of delivery and modification technologies, were thoroughly reviewed and discussed in this paper, which provides a rough picture for ONPATTRO and RNAi therapy.
黄渊余.首例RNA干扰药物问世及该领域技术演化历程[J].生物化学与生物物理进展,2019,46(3):313-322
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