中国科学院机构知识库网格
Chinese Academy of Sciences Institutional Repositories Grid
RNAi therapeutic and its innovative biotechnological evolution

文献类型:期刊论文

作者Weng, Yuhua; Xiao, Haihua; Zhang, Jinchao; Liang, Xing-Jie; Huang, Yuanyu
刊名BIOTECHNOLOGY ADVANCES
出版日期2019-09-01
卷号37期号:5页码:801-825
ISSN号0734-9750
DOI10.1016/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
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.

入库方式: OAI收割

来源:化学研究所

浏览0
下载0
收藏0
其他版本

除非特别说明,本系统中所有内容都受版权保护,并保留所有权利。