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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Detalles Bibliográficos
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
Descripción
Sumario:[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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