The Design and Exogenous Delivery of siRNA for Post-transcriptional Gene Silencing
- 1 July 2004
- journal article
- review article
- Published by Taylor & Francis in Journal of Drug Targeting
- Vol. 12 (6) , 315-340
- https://doi.org/10.1080/10611860400006257
Abstract
RNA interference (RNAi) is a natural cellular process that effects post-transcriptional gene silencing in eukaryotic systems. Small interfering RNA (siRNA) molecules are the key intermediaries in this process which when exogenously administered can inhibit or “silence” the expression of any given target gene. Thus, siRNA molecules hold great promise as biological tools and as potential therapeutic agents for targeted inhibition of disease-causing genes. However, key challenges to the effective and widespread use of these polyanionic, macromolecular duplexes of RNA are their appropriate design and efficient delivery to cells in vitro and in vivo. This review highlights the current strategies used in the design of effective siRNA molecules and also summarises the main strategies being considered for the exogenous delivery of siRNA for both in vitro and in vivo applications.Keywords
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