RNA干扰的治疗潜力。
The therapeutic potential of RNA interference.
作者信息
Uprichard Susan L
机构信息
Department of Molecular and Experimental Medicine, The Scripps Research Institute, 10550 N. Torrey Pines Road, SBR10, La Jolla, CA 92037, USA.
出版信息
FEBS Lett. 2005 Oct 31;579(26):5996-6007. doi: 10.1016/j.febslet.2005.08.004. Epub 2005 Aug 15.
Abstract
In recent years, we have witnessed the discovery of a new mechanism of gene regulation called RNA interference (RNAi), which has revitalized interest in the development of nucleic acid-based technologies for therapeutic gene suppression. This review focuses on the potential therapeutic use of RNAi, discussing the theoretical advantages of RNAi-based therapeutics over previous technologies as well as the challenges involved in developing RNAi for clinical use. Also reviewed, are the in vivo proof-of principle experiments that provide the preclinical justification for the continued development of RNAi-based therapeutics.
摘要
近年来，我们见证了一种名为RNA干扰（RNAi）的基因调控新机制的发现，这重新激发了人们对开发基于核酸的治疗性基因抑制技术的兴趣。本综述聚焦于RNAi的潜在治疗用途，讨论基于RNAi的疗法相对于先前技术的理论优势以及开发临床用RNAi所涉及的挑战。此外，还回顾了体内原理验证实验，这些实验为基于RNAi的疗法的持续开发提供了临床前依据。
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本文引用的文献
1
Targeting angiogenesis, the underlying disorder in neovascular age-related macular degeneration.
Can J Ophthalmol. 2005 Jun;40(3):352-68. doi: 10.1016/S0008-4182(05)80078-X.
2
c-Myc-regulated microRNAs modulate E2F1 expression.
Nature. 2005 Jun 9;435(7043):839-43. doi: 10.1038/nature03677.
3
MicroRNA expression profiles classify human cancers.
Nature. 2005 Jun 9;435(7043):834-8. doi: 10.1038/nature03702.
4
A microRNA polycistron as a potential human oncogene.
Nature. 2005 Jun 9;435(7043):828-33. doi: 10.1038/nature03552.
5
The benefit/risk profile of TNF-blocking agents: findings of a consensus panel.
Semin Arthritis Rheum. 2005 Jun;34(6):819-36. doi: 10.1016/j.semarthrit.2004.11.006.
6
A virus-encoded inhibitor that blocks RNA interference in mammalian cells.
J Virol. 2005 Jun;79(12):7371-9. doi: 10.1128/JVI.79.12.7371-7379.2005.
7
Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors.
Nat Biotechnol. 2005 Jun;23(6):709-17. doi: 10.1038/nbt1101. Epub 2005 May 22.
8
Survivin-directed RNA interference cocktail is a potent suppressor of tumour growth in vivo.
J Med Genet. 2006 Feb;43(2):119-28. doi: 10.1136/jmg.2005.034686. Epub 2005 May 20.
9
Evidence that HIV-1 encodes an siRNA and a suppressor of RNA silencing.
Immunity. 2005 May;22(5):607-19. doi: 10.1016/j.immuni.2005.03.010.
10
Hepatitis C virus replicons escape RNA interference induced by a short interfering RNA directed against the NS5b coding region.
J Virol. 2005 Jun;79(11):7050-8. doi: 10.1128/JVI.79.11.7050-7058.2005.
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