Wilner Samantha E, Levy Matthew
Department of Biochemistry, Michael F. Price Center for Genetic and Translational Medicine, Albert Einstein College of Medicine, Room 519, 1301 Morris Park Avenue, Bronx, NY, 10461, USA.
Methods Mol Biol. 2016;1380:211-24. doi: 10.1007/978-1-4939-3197-2_18.
Aptamers selected against cell surface receptors represent a unique set of ligands that can be used to target nanoparticles and other therapeutics to specific cell types. Here, we describe a method for using aptamers to deliver stable nucleic acid lipid particles (SNALPs) encapsulating small interfering RNA (siRNA) to cells in vitro. Using this method, we have demonstrated the ability of aptamer-conjugated SNALPs to achieve target-specific delivery and siRNA-mediated knockdown of a gene of interest. We also describe methods to characterize SNALP size, siRNA encapsulation efficiency, and aptamer conjugation efficiency.
筛选出的针对细胞表面受体的适配体代表了一组独特的配体,可用于将纳米颗粒和其他治疗药物靶向特定细胞类型。在此,我们描述了一种在体外使用适配体将包裹小干扰RNA(siRNA)的稳定核酸脂质颗粒(SNALP)递送至细胞的方法。使用该方法,我们证明了适配体偶联的SNALP能够实现靶向特异性递送以及siRNA介导的目的基因敲低。我们还描述了表征SNALP大小、siRNA包封效率和适配体偶联效率的方法。