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

被引：236

作者：

Deng, Yan ^{[1
,2
]}

Wang, Chi Chiu ^{[1
]}

Choy, Kwong Wai ^{[1
,2
]}

Du, Quan ^{[3
]}

Chen, Jiao ^{[1
]}

Wang, Qin ^{[4
]}

Li, Lu ^{[1
]}

Chung, Tony Kwok Hung ^{[1
]}

Tang, Tao ^{[1
,2
]}

机构：

[1] Chinese Univ Hong Kong, Fac Med, Dept Obstet & Gynaecol, Shatin, Hong Kong, Peoples R China

[2] Chinese Univ Hong Kong, Shenzhen Res Inst, Hong Kong, Hong Kong, Peoples R China

[3] Peking Univ, Sch Pharmaceut Sci, State Key Lab Nat & Biomimet Drugs, Beijing 100871, Peoples R China

[4] Linyi City Peoples Hosp, Div Med, Dist Nursing Off, Linyi, Peoples R China

来源：

GENE | 2014年 / 538卷 / 02期

关键词：

RNA interference; Gene-silencing; RNAi delivery; Therapeutics; CATIONIC LIPOSOME REAGENT; LIPID-LIKE MATERIALS; DOUBLE-STRANDED-RNA; SIRNA DELIVERY; IN-VITRO; HEPATITIS-C; MULTIDRUG-RESISTANCE; NONVIRAL DELIVERY; DRUG-DELIVERY; CANCER-CELLS;

D O I：

10.1016/j.gene.2013.12.019

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

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.

引用

页码：217 / 227

页数：11

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