用于自动化纳米医学的 siRNA 功能化 RNA 纳米颗粒的设计和自组装。
Design and self-assembly of siRNA-functionalized RNA nanoparticles for use in automated nanomedicine.
机构信息
Department of Chemistry and Biochemistry, Biomolecular Science and Engineering Program, University of California, Santa Barbara, California, USA.
出版信息
Nat Protoc. 2011 Dec 1;6(12):2022-34. doi: 10.1038/nprot.2011.418.
Individual genes can be targeted with siRNAs. The use of nucleic acid nanoparticles (NPs) is a convenient method for delivering combinations of specific siRNAs in an organized and programmable manner. We present three assembly protocols to produce two different types of RNA self-assembling functional NPs using processes that are fully automatable. These NPs are engineered based on two complementary nanoscaffold designs (nanoring and nanocube), which serve as carriers of multiple siRNAs. The NPs are functionalized by the extension of up to six scaffold strands with siRNA duplexes. The assembly protocols yield functionalized RNA NPs, and we show that they interact in vitro with human recombinant Dicer to produce siRNAs. Our design strategies allow for fast, economical and easily controlled production of endotoxin-free therapeutic RNA NPs that are suitable for preclinical development.
可以使用 siRNA 靶向单个基因。核酸纳米颗粒 (NPs) 的使用是一种方便的方法，可以有组织、可编程地递呈特定 siRNA 的组合。我们提出了三种组装方案，使用完全自动化的过程来生产两种不同类型的 RNA 自组装功能 NPs。这些 NPs 是基于两种互补的纳米支架设计（纳米环和纳米立方）构建的，作为多个 siRNA 的载体。NPs 通过最多六个支架链的延伸来功能化，与 siRNA 双链体。组装方案产生功能化的 RNA NPs，我们表明它们在体外与人重组 Dicer 相互作用产生 siRNA。我们的设计策略允许快速、经济且易于控制地生产无内毒素的治疗性 RNA NPs，适用于临床前开发。
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