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Selective gene silencing by viral delivery of short hairpin RNA

RNA interference (RNAi) technology has not only become a powerful tool for functional genomics, but also allows rapid drug target discovery and in vitro validation of these targets in cell culture. Furthermore, RNAi represents a promising novel therapeutic option for treating human diseases, in part...

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Detalles Bibliográficos
Autores principales: Sliva, Katja, Schnierle, Barbara S
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2010
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2949849/
https://www.ncbi.nlm.nih.gov/pubmed/20858246
http://dx.doi.org/10.1186/1743-422X-7-248
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author Sliva, Katja
Schnierle, Barbara S
author_facet Sliva, Katja
Schnierle, Barbara S
author_sort Sliva, Katja
collection PubMed
description RNA interference (RNAi) technology has not only become a powerful tool for functional genomics, but also allows rapid drug target discovery and in vitro validation of these targets in cell culture. Furthermore, RNAi represents a promising novel therapeutic option for treating human diseases, in particular cancer. Selective gene silencing by RNAi can be achieved essentially by two nucleic acid based methods: i) cytoplasmic delivery of short double-stranded (ds) interfering RNA oligonucleotides (siRNA), where the gene silencing effect is only transient in nature, and possibly not suitable for all applications; or ii) nuclear delivery of gene expression cassettes that express short hairpin RNA (shRNA), which are processed like endogenous interfering RNA and lead to stable gene down-regulation. Both processes involve the use of nucleic acid based drugs, which are highly charged and do not cross cell membranes by free diffusion. Therefore, in vivo delivery of RNAi therapeutics must use technology that enables the RNAi therapeutic to traverse biological membrane barriers in vivo. Viruses and the vectors derived from them carry out precisely this task and have become a major delivery system for shRNA. Here, we summarize and compare different currently used viral delivery systems, give examples of in vivo applications, and indicate trends for new developments, such as replicating viruses for shRNA delivery to cancer cells.
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spelling pubmed-29498492010-10-06 Selective gene silencing by viral delivery of short hairpin RNA Sliva, Katja Schnierle, Barbara S Virol J Review RNA interference (RNAi) technology has not only become a powerful tool for functional genomics, but also allows rapid drug target discovery and in vitro validation of these targets in cell culture. Furthermore, RNAi represents a promising novel therapeutic option for treating human diseases, in particular cancer. Selective gene silencing by RNAi can be achieved essentially by two nucleic acid based methods: i) cytoplasmic delivery of short double-stranded (ds) interfering RNA oligonucleotides (siRNA), where the gene silencing effect is only transient in nature, and possibly not suitable for all applications; or ii) nuclear delivery of gene expression cassettes that express short hairpin RNA (shRNA), which are processed like endogenous interfering RNA and lead to stable gene down-regulation. Both processes involve the use of nucleic acid based drugs, which are highly charged and do not cross cell membranes by free diffusion. Therefore, in vivo delivery of RNAi therapeutics must use technology that enables the RNAi therapeutic to traverse biological membrane barriers in vivo. Viruses and the vectors derived from them carry out precisely this task and have become a major delivery system for shRNA. Here, we summarize and compare different currently used viral delivery systems, give examples of in vivo applications, and indicate trends for new developments, such as replicating viruses for shRNA delivery to cancer cells. BioMed Central 2010-09-21 /pmc/articles/PMC2949849/ /pubmed/20858246 http://dx.doi.org/10.1186/1743-422X-7-248 Text en Copyright ©2010 Sliva and Schnierle; licensee BioMed Central Ltd. http://creativecommons.org/licenses/by/2.0 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Sliva, Katja
Schnierle, Barbara S
Selective gene silencing by viral delivery of short hairpin RNA
title Selective gene silencing by viral delivery of short hairpin RNA
title_full Selective gene silencing by viral delivery of short hairpin RNA
title_fullStr Selective gene silencing by viral delivery of short hairpin RNA
title_full_unstemmed Selective gene silencing by viral delivery of short hairpin RNA
title_short Selective gene silencing by viral delivery of short hairpin RNA
title_sort selective gene silencing by viral delivery of short hairpin rna
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2949849/
https://www.ncbi.nlm.nih.gov/pubmed/20858246
http://dx.doi.org/10.1186/1743-422X-7-248
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