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Molecular Umbrella Conjugate for the Ocular Delivery of siRNA
[Image: see text] The synthesis, computer modeling, and biological activity of an octawalled molecular umbrella short interfacing RNA (siRNA) conjugate is described. This molecular umbrella–siRNA conjugate exhibited mRNA knockdown activity in vitro in the absence of a transfection reagent. Evaluatio...
Autores principales: | , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Chemical
Society
2014
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4334295/ https://www.ncbi.nlm.nih.gov/pubmed/24433067 http://dx.doi.org/10.1021/bc400506m |
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author | Janout, Vaclav Cline, Lauren L. Feuston, Bradley P. Klein, Lee O’Brien, Ann Tucker, Tom Yuan, Yu O’Neill-Davis, Lynn A. Peiffer, Robert L. Nerurkar, Sandhya S. Jadhav, Vasant Tellers, David M. Regen, Steven L. |
author_facet | Janout, Vaclav Cline, Lauren L. Feuston, Bradley P. Klein, Lee O’Brien, Ann Tucker, Tom Yuan, Yu O’Neill-Davis, Lynn A. Peiffer, Robert L. Nerurkar, Sandhya S. Jadhav, Vasant Tellers, David M. Regen, Steven L. |
author_sort | Janout, Vaclav |
collection | PubMed |
description | [Image: see text] The synthesis, computer modeling, and biological activity of an octawalled molecular umbrella short interfacing RNA (siRNA) conjugate is described. This molecular umbrella–siRNA conjugate exhibited mRNA knockdown activity in vitro in the absence of a transfection reagent. Evaluation of this molecular umbrella conjugate in vivo, using the rat eye via intravitreal injection, resulted in sequence specific mRNA knockdown in the retina with no obvious signs of toxicity, as judged by ophthalmic examination. |
format | Online Article Text |
id | pubmed-4334295 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | American Chemical
Society |
record_format | MEDLINE/PubMed |
spelling | pubmed-43342952015-02-20 Molecular Umbrella Conjugate for the Ocular Delivery of siRNA Janout, Vaclav Cline, Lauren L. Feuston, Bradley P. Klein, Lee O’Brien, Ann Tucker, Tom Yuan, Yu O’Neill-Davis, Lynn A. Peiffer, Robert L. Nerurkar, Sandhya S. Jadhav, Vasant Tellers, David M. Regen, Steven L. Bioconjug Chem [Image: see text] The synthesis, computer modeling, and biological activity of an octawalled molecular umbrella short interfacing RNA (siRNA) conjugate is described. This molecular umbrella–siRNA conjugate exhibited mRNA knockdown activity in vitro in the absence of a transfection reagent. Evaluation of this molecular umbrella conjugate in vivo, using the rat eye via intravitreal injection, resulted in sequence specific mRNA knockdown in the retina with no obvious signs of toxicity, as judged by ophthalmic examination. American Chemical Society 2014-01-16 2014-02-19 /pmc/articles/PMC4334295/ /pubmed/24433067 http://dx.doi.org/10.1021/bc400506m Text en Copyright © 2014 American Chemical Society This is an open access article published under an ACS AuthorChoice License (http://pubs.acs.org/page/policy/authorchoice_termsofuse.html) , which permits copying and redistribution of the article or any adaptations for non-commercial purposes. |
spellingShingle | Janout, Vaclav Cline, Lauren L. Feuston, Bradley P. Klein, Lee O’Brien, Ann Tucker, Tom Yuan, Yu O’Neill-Davis, Lynn A. Peiffer, Robert L. Nerurkar, Sandhya S. Jadhav, Vasant Tellers, David M. Regen, Steven L. Molecular Umbrella Conjugate for the Ocular Delivery of siRNA |
title | Molecular Umbrella Conjugate for the Ocular Delivery
of siRNA |
title_full | Molecular Umbrella Conjugate for the Ocular Delivery
of siRNA |
title_fullStr | Molecular Umbrella Conjugate for the Ocular Delivery
of siRNA |
title_full_unstemmed | Molecular Umbrella Conjugate for the Ocular Delivery
of siRNA |
title_short | Molecular Umbrella Conjugate for the Ocular Delivery
of siRNA |
title_sort | molecular umbrella conjugate for the ocular delivery
of sirna |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4334295/ https://www.ncbi.nlm.nih.gov/pubmed/24433067 http://dx.doi.org/10.1021/bc400506m |
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