RNAi therapeutic and its innovative biotechnological evolution

doi:10.1016/j.biotechadv.2019.04.012

CORC > 化学研究所 > 中国科学院化学研究所

	RNAi therapeutic and its innovative biotechnological evolution
	Weng, Yuhua; Xiao, Haihua; Zhang, Jinchao; Liang, Xing-Jie; Huang, Yuanyu
刊名	BIOTECHNOLOGY ADVANCES
	2019-09-01
卷号	37 期号:5 页码:801-825
ISSN号	0734-9750
DOI	10.1016/j.biotechadv.2019.04.012
英文摘要	Recently, United States Food and Drug Administration (FDA) and European Commission (EC) approved Alnylam Pharmaceuticals' RNA interference (RNAi) therapeutic, ONPATTRO (TM) (Patisiran), for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hAITR) amyloidosis in adults. This is the first RNAi therapeutic all over the world, as well as the first FDA-approved treatment for this indication. As a milestone event in RNAi pharmaceutical industry, it means, for the first time, people have broken through all development processes for RNAi drugs from research to clinic. With this achievement, RNAi approval may soar in the coming years. In this paper, we introduce the basic information of ONPATTRO and the properties of RNAi and nucleic acid therapeutics, update the clinical and preclinical development activities, review its complicated development history, summarize the key technologies of RNAi at early stage, and discuss the latest advances in delivery and modification technologies. It provides a comprehensive view and biotechnological insights of RNAi therapy for the broader audiences.
语种	英语
WOS记录号	WOS:000477917000012
内容类型	期刊论文
源URL	[http://ir.iccas.ac.cn/handle/121111/82619]
专题	中国科学院化学研究所
作者单位	Chinese Acad Sci, Inst Chem, Beijing 100190, Peoples R China
推荐引用方式 GB/T 7714	Weng, Yuhua,Xiao, Haihua,Zhang, Jinchao,et al. RNAi therapeutic and its innovative biotechnological evolution[J]. BIOTECHNOLOGY ADVANCES,2019,37(5):801-825.
APA	Weng, Yuhua,Xiao, Haihua,Zhang, Jinchao,Liang, Xing-Jie,&Huang, Yuanyu.(2019).RNAi therapeutic and its innovative biotechnological evolution.BIOTECHNOLOGY ADVANCES,37(5),801-825.
MLA	Weng, Yuhua,et al."RNAi therapeutic and its innovative biotechnological evolution".BIOTECHNOLOGY ADVANCES 37.5(2019):801-825.