用于 siRNA 眼内递药的分子伞状缀合物。
Molecular umbrella conjugate for the ocular delivery of siRNA.
机构信息
Department of Chemistry, Lehigh University , Bethlehem, Pennsylvania 18015, United States.
出版信息
Bioconjug Chem. 2014 Feb 19;25(2):197-201. doi: 10.1021/bc400506m. Epub 2014 Jan 23.
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.
描述了一种八面体分子伞短界面 RNA（siRNA）缀合物的合成、计算机建模和生物活性。这种分子伞-siRNA 缀合物在没有转染试剂的情况下表现出体外 mRNA 敲低活性。通过玻璃体内注射在大鼠眼中评估这种分子伞缀合物，导致视网膜中序列特异性 mRNA 敲低，且通过眼科检查判断没有明显的毒性迹象。
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