RNAi therapeutic and its innovative biotechnological evolution
Weng, Yuhua; Xiao, Haihua; Zhang, Jinchao; Liang, Xing-Jie; Huang, Yuanyu
AbstractRecently, 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.
Indexed BySCI
WOS IDWOS:000477917000012
EI Accession Number1873-1899
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Cited Times:3[WOS]   [WOS Record]     [Related Records in WOS]
Document Type期刊论文
AffiliationChinese Acad Sci, Inst Chem, Beijing 100190, Peoples R China
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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.
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