Molecular Umbrella Conjugate for the Ocular Delivery of siRNA

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 molecula...

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Veröffentlicht in:	Bioconjugate chemistry 2014-02, Vol.25 (2), p.197-201
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Biochemistry Chemical synthesis Communication Drug Administration Routes Drug Carriers Eye Eyes & eyesight HEK293 Cells Humans Molecular Dynamics Simulation Ribonucleic acid RNA RNA, Small Interfering - administration & dosage Rodents Toxicity
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Beschreibung
Zusammenfassung:	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.
ISSN:	1043-1802 1520-4812
DOI:	10.1021/bc400506m