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Molecular umbrella conjugate for the ocular delivery of siRNA.

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.

SUBMITTER: Janout V

PROVIDER: S-EPMC4334295 | biostudies-literature | 2014 Feb

REPOSITORIES: biostudies-literature

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Molecular umbrella conjugate for the ocular delivery of siRNA.

Janout Vaclav V Cline Lauren L LL Feuston Bradley P BP Klein Lee L O'Brien Ann A Tucker Tom T Yuan Yu Y O'Neill-Davis Lynn A LA Peiffer Robert L RL Nerurkar Sandhya S SS Jadhav Vasant V Tellers David M DM Regen Steven L SL

Bioconjugate chemistry 20140123 2

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 examin ...[more]

PMID: 24433067

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