Gene manipulation through the use of small interfering RNA (siRNA): From in vitro to in vivo applications

Kumar, Lekha Dinesh and Clarke, Alan Richard ORCID: https://orcid.org/0000-0002-4281-426X 2007. Gene manipulation through the use of small interfering RNA (siRNA): From in vitro to in vivo applications. Advanced Drug Delivery Reviews 59 (2-3) , pp. 87-100. 10.1016/j.addr.2007.03.009

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Abstract

The conventional approach to investigate genotype–phenotype relationships has been the generation of gene targeted murine strains. However, the emergence of RNAi technologies has opened the possibility of much more rapid (and indeed more cost effective) genetic manipulation in vivo at the level of the transcriptome. Successful application of RNAi in vivo depends on intracellular targeted delivery of siRNA/shRNA molecules for efficient knockdown of the desired gene. In this review, we discuss the rationale and different strategies of using siRNA/shRNA for accomplishing the silencing of targeted genes in a spatial and /or temporally regulated manner. We also summarise the steps involved in extending these approaches to in vivo applications, with a specific focus upon the development of silencing in the mouse.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Biosciences
Subjects:	Q Science > Q Science (General)
Additional Information:	siRNA; shRNA; transgenic; transfection; target validation; lentiviral; adenoviral; therapeutic; siRNA design; methods of delivery; mammalian system.
Publisher:	Elsevier
ISSN:	0169-409X
Last Modified:	25 Oct 2022 10:10
URI:	https://orca.cardiff.ac.uk/id/eprint/61343

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