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Gene. 2014 Apr 1;538(2):217-27. doi: 10.1016/j.gene.2013.12.019. Epub 2014 Jan 7.

Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies.

Author information

1
Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China; Shenzhen Research Institute, The Chinese University of Hong Kong, Hong Kong, China.
2
Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China.
3
State Key Laboratory of Natural and Biomimetic Drugs, School of Pharmaceutical Sciences, Peking University, Beijing, China.
4
Linyi City People's Hospital Division Medical District Nursing Office, Linyi, China.
5
Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China; Shenzhen Research Institute, The Chinese University of Hong Kong, Hong Kong, China. Electronic address: tangtao@cuhk.edu.hk.

Abstract

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.

KEYWORDS:

Gene-silencing; RNA interference; RNAi delivery; Therapeutics

PMID:
24406620
DOI:
10.1016/j.gene.2013.12.019
[Indexed for MEDLINE]
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