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Molecular Umbrella Conjugate for the Ocular Delivery of siRNA

Authors :
Thomas J. Tucker
Lauren L. Cline
Bradley P. Feuston
David M. Tellers
Vasant Jadhav
Robert L. Peiffer
Sandhya S. Nerurkar
Lynn A. O’Neill-Davis
Steven L. Regen
Vaclav Janout
Yu Yuan
Ann O’Brien
Lee J. Klein
Source :
Bioconjugate Chemistry
Publication Year :
2014
Publisher :
American Chemical Society (ACS), 2014.

Abstract

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.

Details

ISSN :
15204812 and 10431802
Volume :
25
Database :
OpenAIRE
Journal :
Bioconjugate Chemistry
Accession number :
edsair.doi.dedup.....eed0788df98a80639c2be2827b3f43b0
Full Text :
https://doi.org/10.1021/bc400506m