Applications of RNA interference: current state and prospects for siRNA-based strategies in vivo |
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Authors: | Achim Aigner |
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Institution: | (1) Department Pharmacology and Toxicology, School of Medicine, Philipps-University Marburg, Karl-von-Frisch-Strasse 1, 35033 Marburg, Germany |
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Abstract: | Within the recent years, RNA interference (RNAi) has become an almost-standard method for in vitro knockdown of any target
gene of interest. Now, one major focus is to further explore its potential in vivo, including the development of novel therapeutic
strategies. From the mechanism, it becomes clear that small interfering RNAs (siRNAs) play a pivotal role in triggering RNAi.
Thus, the efficient delivery of target gene-specific siRNAs is one major challenge in the establishment of therapeutic RNAi.
Numerous studies, based on different modes of administration and various siRNA formulations and/or modifications, have already
accumulated promising results. This applies to various animal models covering viral infections, cancer and multiple other
diseases. Continuing efforts will lead to the development of efficient and “double-specific” drugs, comprising of siRNAs with
high target gene specificity and of nanoparticles enhancing siRNA delivery and target organ specificity. |
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Keywords: | RNA interference RNAi siRNA Gene-targeting Gene knockdown Nonviral siRNA delivery Nanoplexes |
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