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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...

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Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Chemical Society 2014
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.
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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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