小分子干扰RNA用于基因治疗的研究进展

温爱萍;徐小薇;李大魁

中国药学杂志 ›› 2006, Vol. 41 ›› Issue (23) : 1763-1767.

中国药学杂志 ›› 2006, Vol. 41 ›› Issue (23) : 1763-1767.
综述

小分子干扰RNA用于基因治疗的研究进展

  • 温爱萍;徐小薇;李大魁
作者信息 +
文章历史 +

摘要

目的介绍小分子干扰RNA(siRNA)在基因治疗领域的研究进展。方法查阅国内外相关文献,进行分析、归纳和综述。结果siRNA用于基因治疗具有独特的优势,虽然还处于起步阶段,仍存在一些实际应用方面的问题,但随着研究的深入,这些问题正在或将被认识和解决。结论siRNA为基因治疗开辟了新的道路,具有广阔的应用前景。

关键词

小分子干扰RNA(siRNA) / RNA干扰

引用本文

导出引用
温爱萍;徐小薇;李大魁. 小分子干扰RNA用于基因治疗的研究进展[J]. 中国药学杂志, 2006, 41(23): 1763-1767

参考文献

[1] FIRE A,XU S,MONTGOMERY M K,et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans[J] .Nature,1998,391(6669): 806-811. [2] CAPLEN N J. Gene Therapy Progress and Prospects. Downregulating gene expression: the impact of RNA interference[J] .Gene Therapy,2004,11(16):1241-1248. [3] MA P P,XUE S P,HAN D S. Principles and applications of RNA interfering technology[J] .Chin J Histochem Cytochem (中国组织化学与细胞化学杂志),2003,12(2): 201-207. [4] SIDDHESH D,DAVID G,DIANE J.DNA-based therapeutics and DNA delivery systems: A comprehensive review[J] .The AAPS J,2005,7(1): E61-E77. [5] DORSETT Y,TUSCHL T. siRNAs: applications in functional genomics and potential as therapeutics[J] .Nat Rev Drug Discov,2004,3(4): 318-329. [6] RAY K M,PAUL A. RNA interference: from gene silencing to gene-specific therapeutics[J] .Pharmacology Therapeutics,2005,107(2): 222-239. [7] RYTHER R C,FLYNT A S,Phillips J A,et al. siRNA therapeutics: big potential from small RNAs[J] .Gene Therapy,2005,12(1): 5-11. [8] NOVINA C D,SHARP P A.The RNAi revolution[J] .Nature,2004,430(6996): 161-164. [9] REN X,SONG Y G,CHEN X Q. Progress in research of RNAi[J] . World Clin J Digestol(世界华人消化杂志),2004,12(3): 748-750. [10] BARIK S. Development of gene-specific double-stranded RNA drugs[J] .Ann Med,2004,36: 540-551. [11] GONG H,LIU C M,LIU D P,et al. The role of small RNAs in human diseases: potential troublemaker and therapeutic tools[J] .Med Res Rev,2005,25(3): 361-381. [12] SHLOMAI A,SHAUL Y. RNA interference--small RNAs effectively fight viral hepatitis[J] .Liver Int,2004,24(6): 526-531. [13] BERKHOUT B. RNA interference as an antiviral approach: targeting HIV-1[J] .Curr Opin Mol Ther,2004,6(2): 141-145. [14] SUN J. Progress in using RNA interference against HIV infection[J] . Clin Pharm J(中国药学杂志),2005,40(2): 84-86. [15] KRONKE J,KITTLER R,BUCHHOLZ F,et al. Alternative approaches for efficient inhibition of hepatitis C virus RNA replication by small interfering RNAs[J] .J Virol,2004,78(7): 3436-3446. [16] YUAN J,CHEUNG P K,ZHANG H M,et al. Inhibition of coxsackievirus B3 replication by small interfering RNAs requires perfect sequence match in the central region of the viral positive strand[J] .J Virol,2005,79(4): 2151-2159. [17] ZHENG B J,GUAN Y,TANG Q,et al. Prophylactic and therapeutic effects of small interfering RNA targeting SARS-coronavirus[J] . Antivir Ther,2004,9(3): 365-374. [18] MOHAPATRA J K,SANYAL A,HEMADRI D,et al.Evaluation of in vitro inhibitory potential of small interfering RNAs directed against various regions of foot-and-mouth disease virus genome[J] .Biochem Biophys Res Commun,2005,329(3): 1133-1138. [19] KUMAR R,ADAMS B,OLDENBURG A,et al. Characterisation and expression of a PP1 serine/threonine protein phosphatase (PfPP1) from the malaria parasite,Plasmodium falciparum:demonstration of its essential role using RNA interference[J] .Malar J,2002,1(1):5. [20] VAYSSIE L,VARGAS M,WEBER C,et al. Double-stranded RNA mediates homology-dependant gene silencing of gamma-tubulin in the human parasite Entamoeba histolytica[J] .Mol Biochem Parasitol,2004,138(1):21-28. [21] YANG Z,CLOUD A,HUGHES D,et al. Stable inhibition of human thymidylate synthase expression following retroviral introduction of an siRNA gene[J] .Cancer Gene Ther,2006,13(1): 107-114. [22] WANNENES F,CIAFRE S A,NIOLA F,et al. Vector-based RNA interference against vascular endothelial growth factor-A significantly limits vascularization and growth of prostate cancer in vivo[J] .Cancer Gene Ther,2005,12(12): 926-934. [23] CHEN Z J,KREN B T,WONG P Y,et al. Sleeping beauty-mediated down-regulation of huntingtin expression by RNA interference[J] . Biochem Biophys Res Commun,2005,329(2): 646-652. [24] XIA X G,ZHOU H,ZHOU S,et al. An RNAi strategy for treatment of amyotrophic lateral sclerosis caused by mutant Cu,Zn superoxide dismutase[J] .J Neurochem,2005,92(2): 362-367. [25] MILLER T M,KASPAR B K,KOPS G J,et al. Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis[J] .Ann Neurol,2005,57(5): 773-776. [26] ZHANG X Q. The concept,character,and implication of gene therapy[J] .Nature Magazine(自然杂志),2003,25(3): 153-156. [27] SOUTSCHEK J,AKINC A,BRAMLAGE B,et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs[J] .Nature,2004,432(7014): 173-178. [28] JOHN J A. Cholesterol connection in RNAi[J] .Nature,2004,432(7014): 155-156. [29] CROMBIE C,FRASER A. Treating genetic disease through RNA interference[J] .Lancet,2005,365(9467): 1288-1290. [30] GUO X H,HAN X X,ZHOU X Z.The gene delivery technology in gene therapy[J] .Letters Biotechnol(生物技术通讯),2003,14(3): 235-237. [31] ELMEN J,THONBERG H,LJUNGBERG K,et al. Locked nucleic acid (LNA)mediated improvements in siRNA stability and functionality[J] .Nucleic Acids Res,2005,33(1):439-447.

30

Accesses

0

Citation

Detail

段落导航
相关文章

/