Therapeutic potentials of gene silencing by RNA interference: Principles, challenges, and new strategies
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AbstractDuring 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.
All Author(s) ListDeng Y, Wang CC, Choy KW, Du Q, Chen J, Wang Q, Li L, Chung TKH, Tang T
Journal nameGene
Year2014
Month4
Day1
Volume Number538
Issue Number2
PublisherElsevier
Pages217 - 227
ISSN0378-1119
eISSN1879-0038
LanguagesEnglish-United Kingdom
KeywordsGene-silencing; RNA interference; RNAi delivery; Therapeutics
Web of Science Subject CategoriesGenetics & Heredity; GENETICS & HEREDITY

Last updated on 2021-23-02 at 00:14