Therapeutic potentials of gene silencing by RNA interference: Principles, challenges, and new strategies
Publication in refereed journal


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摘要During recent decades there have been remarkable advances in biology, in which one of the most important discoveries is RNA interference (RNAi). RNAi is a specific post-transcriptional regulatory pathway that can result in silencing gene functions. Efforts have been done to translate this new discovery into clinical applications for disease treatment. However, technical difficulties restrict the development of RNAi, including stability, off-target effects, immunostimulation and delivery problems. Researchers have attempted to surmount these barriers and improve the bioavailability and safety of RNAi-based therapeutics by optimizing the chemistry and structure of these molecules. This paper aimed to describe the principles of RNA interference, review the therapeutic potential in various diseases and discuss the new strategies for in vivo delivery of RNAi to overcome the challenges. (C) 2013 Elsevier B.V. All rights reserved.
著者Deng Y, Wang CC, Choy KW, Du Q, Chen J, Wang Q, Li L, Chung TKH, Tang T
期刊名稱Gene
出版年份2014
月份4
日期1
卷號538
期次2
出版社Elsevier
頁次217 - 227
國際標準期刊號0378-1119
電子國際標準期刊號1879-0038
語言英式英語
關鍵詞Gene-silencing; RNA interference; RNAi delivery; Therapeutics
Web of Science 學科類別Genetics & Heredity; GENETICS & HEREDITY

上次更新時間 2021-12-01 於 00:21