Self-assembled triangular DNA nanoparticles are an efficient system for gene delivery.
Self-assembled triangular DNA nanoparticles are an efficient system for gene delivery.
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DOI：
10.1016/j.jconrel.2016.05.038
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发表时间：
2016-07
期刊：
影响因子：
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通讯作者：
Yingming Wang;Zaichun You;Juan Du;Hongli Li;Huaping Chen;Jingtong Li;Weijie Dong;Binfeng He;C. Mao;Guansong Wang
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文献类型：
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作者：
Yingming Wang;Zaichun You;Juan Du;Hongli Li;Huaping Chen;Jingtong Li;Weijie Dong;Binfeng He;C. Mao;Guansong Wang
开发先进的核酸药物传递系统对于实现基因的最佳传递具有重要意义。自组装核酸纳米颗粒是一种用于核酸和其他小分子药物递送的优良平台。在本研究中，我们开发了高效的三链RNA/DNA杂交三角形自组装纳米颗粒，即mTOR单链siRNA负载的三角形DNA纳米颗粒（ssRNA-TNP）。ssRNA-TNP由上述三种组分互补缔合形成，并且在完全培养基中比标准双链siRNA更稳定。转染NCI-H292细胞，转染效率高，且呈剂量和时间依赖性。此外，ssRNA-TNP摄取依赖于巨胞饮和网格蛋白介导的内吞途径。有趣的是，ssRNA-TNP更有效地抑制mTOR的表达。这种ssRNA-TNP具有结构简单、稳定性好、转染效率高等优点，有望成为一种新型的非病毒基因递送纳米系统。
Developing an advanced nucleic acid drug delivery system is of great significance in order to achieve optimal gene delivery. Self-assembled nucleic acid nanoparticles are an excellent platform for the delivery of nucleic acids and other small molecular drugs. In this study, we developed the efficient, three-stranded, RNA/DNA hybrid triangular self-assembled nanoparticles, namely, mTOR single-stranded siRNA-loaded triangular DNA nanoparticles (ssRNA-TNP). The ssRNA-TNP is formed by the complementary association of the above mentioned three components and is more stable in complete medium than standard duplex siRNA. It could be efficiently transfected into NCI-H292 cells in a dose- and time-dependent manner, resulting in high transfection efficiency. Furthermore, ssRNA-TNP uptake is dependent on macropinocytosis and clathrin-mediated endocytosis pathways. Interestingly, ssRNA-TNP is more efficient to inhibit the expression of mTOR. This ssRNA-TNP has a simpler structure, better stability, and higher transfection efficiency; therefore it may become a novel nonviral nanosystem for gene delivery.