Molecular Umbrella Conjugate for the Ocular Delivery of siRNA

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• 作者：Vaclav Janout ; Lauren L. Cline ; Bradley P. Feuston ; Lee Klein ; Ann O’Brien ; Tom Tucker ; Yu Yuan ; Lynn A. O’Neill-Davis ; Robert L. Peiffer ; Sandhya S. Nerurkar ; Vasant Jadhav ; David M. Tellers ; Steven L. Regen
• 刊名：Bioconjugate Chemistry
• 出版年：2014
• 出版时间：February 19, 2014
• 年：2014
• 卷：25
• 期：2
• 页码：197-201
• 全文大小：355K
• ISSN：1520-4812

文摘

The synthesis, computer modeling, and biological activity of an octawalled molecular umbrella short interfacing RNA (siRNA) conjugate is described. This molecular umbrella鈥搒iRNA 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.