siRNA-mediated gene silencing in vitro and in vivo - PubMed (original) (raw)
. 2002 Oct;20(10):1006-10.
doi: 10.1038/nbt739. Epub 2002 Sep 16.
Affiliations
- PMID: 12244328
- DOI: 10.1038/nbt739
siRNA-mediated gene silencing in vitro and in vivo
Haibin Xia et al. Nat Biotechnol. 2002 Oct.
Abstract
RNA interference is now established as an important biological strategy for gene silencing, but its application to mammalian cells has been limited by nonspecific inhibitory effects of long dsRNA on translation. Here, we describe a viral-mediated delivery mechanism that results in specific silencing of targeted genes through expression of small interfering RNA (siRNA). We establish proof of principle by markedly diminishing expression of exogenous and endogenous genes in vitro and in vivo in brain and liver, and further apply this strategy to a model system of a major class of neurodegenerative disorders, the polyglutamine diseases, to show reduced polyglutamine aggregation in cells. This viral-mediated strategy should prove generally useful in reducing expression of target genes to model biological processes or to provide therapy for dominant human diseases.
Similar articles
- Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model.
Reich SJ, Fosnot J, Kuroki A, Tang W, Yang X, Maguire AM, Bennett J, Tolentino MJ. Reich SJ, et al. Mol Vis. 2003 May 30;9:210-6. Mol Vis. 2003. PMID: 12789138 - Sequence-specific gene silencing in murine muscle induced by electroporation-mediated transfer of short interfering RNA.
Kishida T, Asada H, Gojo S, Ohashi S, Shin-Ya M, Yasutomi K, Terauchi R, Takahashi KA, Kubo T, Imanishi J, Mazda O. Kishida T, et al. J Gene Med. 2004 Jan;6(1):105-10. doi: 10.1002/jgm.456. J Gene Med. 2004. PMID: 14716682 - Effects of treatment with small interfering RNA on joint inflammation in mice with collagen-induced arthritis.
Schiffelers RM, Xu J, Storm G, Woodle MC, Scaria PV. Schiffelers RM, et al. Arthritis Rheum. 2005 Apr;52(4):1314-8. doi: 10.1002/art.20975. Arthritis Rheum. 2005. PMID: 15818667 - Toxicogenomics of non-viral drug delivery systems for RNAi: potential impact on siRNA-mediated gene silencing activity and specificity.
Akhtar S, Benter I. Akhtar S, et al. Adv Drug Deliv Rev. 2007 Mar 30;59(2-3):164-82. doi: 10.1016/j.addr.2007.03.010. Epub 2007 Mar 21. Adv Drug Deliv Rev. 2007. PMID: 17481774 Review. - RNAi in functional genomics.
Scherr M, Eder M. Scherr M, et al. Curr Opin Mol Ther. 2004 Apr;6(2):129-35. Curr Opin Mol Ther. 2004. PMID: 15195923 Review.
Cited by
- Ferroptosis: A double-edged sword.
Wang S, Guo Q, Zhou L, Xia X. Wang S, et al. Cell Death Discov. 2024 May 30;10(1):265. doi: 10.1038/s41420-024-02037-9. Cell Death Discov. 2024. PMID: 38816377 Free PMC article. Review. - A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition.
Chinnapaiyan S, Santiago MJ, Panda K, Rahman MS, Alluin J, Rossi J, Unwalla HJ. Chinnapaiyan S, et al. Mol Ther Nucleic Acids. 2023 Apr 19;32:553-565. doi: 10.1016/j.omtn.2023.04.011. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37215150 Free PMC article. - Molecular Characterization of U6 Promoters from Orange-Spotted Grouper (Epinephelus coioides) and Its Application in DNA Vector-Based RNAi Technology.
Yan F, Xiao X, Long C, Tang L, Wang C, Zhang M, Zhang J, Lin H, Huang H, Zhang Y, Li S. Yan F, et al. Mar Biotechnol (NY). 2023 Jun;25(3):388-402. doi: 10.1007/s10126-023-10212-9. Epub 2023 May 8. Mar Biotechnol (NY). 2023. PMID: 37154998 - Biomolecular condensates: Formation mechanisms, biological functions, and therapeutic targets.
Niu X, Zhang L, Wu Y, Zong Z, Wang B, Liu J, Zhang L, Zhou F. Niu X, et al. MedComm (2020). 2023 Feb 28;4(2):e223. doi: 10.1002/mco2.223. eCollection 2023 Apr. MedComm (2020). 2023. PMID: 36875159 Free PMC article. Review. - MicroRNAs in T Cell-Immunotherapy.
Dosil SG, Rodríguez-Galán A, Sánchez-Madrid F, Fernández-Messina L. Dosil SG, et al. Int J Mol Sci. 2022 Dec 23;24(1):250. doi: 10.3390/ijms24010250. Int J Mol Sci. 2022. PMID: 36613706 Free PMC article. Review.
Publication types
MeSH terms
Substances
LinkOut - more resources
Full Text Sources
Other Literature Sources