Molecular Umbrella Conjugate for the Ocular Delivery of siRNA | Zendy

Václav Janout | Zendy; Lauren L. Cline | Zendy; Bradley P. Feuston | Zendy; Lee J. Klein | Zendy; Ann O’Brien | Zendy; Tom Tucker | Zendy; Yu Yuan | Zendy; Lynn O’Neill-Davis | Zendy; Robert L. Peiffer | Zendy; Sandhya S. Nerurkar | Zendy; Vasant Jadhav | Zendy; David M. Tellers | Zendy; Steven L. Regen | Zendy

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Molecular Umbrella Conjugate for the Ocular Delivery of siRNA

Author(s) -

Václav Janout,

Lauren L. Cline,

Bradley P. Feuston,

Lee J. Klein,

Ann O’Brien,

Tom Tucker,

Yu Yuan,

Lynn O’Neill-Davis,

Robert L. Peiffer,

Sandhya S. Nerurkar,

Vasant Jadhav,

David M. Tellers,

Steven L. Regen

Publication year - 2014

Publication title -

bioconjugate chemistry

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.279

H-Index - 172

eISSN - 1520-4812

pISSN - 1043-1802

DOI - 10.1021/bc400506m

Subject(s) - conjugate , chemistry , gene knockdown , in vivo , small interfering rna , transfection , messenger rna , interfacing , microbiology and biotechnology , biochemistry , apoptosis , biology , gene , genetics , mathematical analysis , mathematics , political science , law

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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