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Development of Pre-Clinical Models for Evaluating the Therapeutic Potential of Candidate siRNA Targeting STAT6 /

PLoS ONE, Volume: 9, Issue: 2, Start page: e90338

Swansea University Author: Walker, William

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DOI (Published version): 10.1371/journal.pone.0090338

Abstract

As the development of siRNA therapeutics is technically challenging, this study evaluates various pre-clinical models in their ability to suitably test candidate siRNA for respiratory administration. This study shows that use of chemically-modified siRNA was not associated with cellular toxicity, ye...

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Published in: PLoS ONE
Published: 2014
Online Access: http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0090338
URI: https://cronfa.swan.ac.uk/Record/cronfa17428
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Abstract: As the development of siRNA therapeutics is technically challenging, this study evaluates various pre-clinical models in their ability to suitably test candidate siRNA for respiratory administration. This study shows that use of chemically-modified siRNA was not associated with cellular toxicity, yet enhanced bio-availability without evidence of reduced efficacy. Furthermore, we established a robust, sensitive method for determining siRNA bio-distribution in vivo, and co-developed a novel nasal administration model to further aid evaluation.
College: Swansea University Medical School
Issue: 2
Start Page: e90338