Design, synthesis and evaluation of novel, branched trident small interfering RNA nanostructures for sequence-specific RNAi activity

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Design, synthesis and evaluation of novel, branched trident small interfering RNA nanostructures for sequence-specific RNAi activity

机译：新型的设计，合成和评价，分支三叉戟小干扰RNA纳米序列术术术级rNAi活性

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Small interfering RNAs (siRNAs) are potential candidates for gene regulation with efficient activity, but off-target effects and limited systemic delivery. Herein, we report the design and synthesis of the branched siRNA nanostructures with highly improved resistance against exonucleases. Also, these branched siRNAs showed suppression of off-target gene silencing through selection of the passenger strand as the branching unit. The physical characterization of branched siRNAs showed that they form a compact assembly with a hydrodynamic diameter of 6.9 nm against 2.8 nm of the duplex. We demonstrated that a branched siRNA synthesized with a trebling solid-support selectively exhibits RNAi activity and suppresses the off-target effect.

机译：小干扰RNA（siRNA）是具有高效活动的基因调节的潜在候选者，但偏离目标效果和有限的系统性交付。在此，我们报告了支化的siRNA纳米结构的设计和合成具有高度改善的对外切核酸酶的抗性。此外，这些支链SiRNA通过选择乘客链作为分支单元而显示出脱靶基因沉默。分支SiRNA的物理表征显示，它们形成紧凑的组件，其具有6.9nm的流体动力直径，其对2.8nm的双链体。我们证明，用倾斜固体支持合成的分枝siRNA选择性地表现出RNAi活性并抑制脱靶效果。

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《RSC Advances》 |2019年第59期|共6页
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1. Design, synthesis and evaluation of novel, branched trident small interfering RNA nanostructures for sequence-specific RNAi activity [J] . RSC Advances . 2019,第59期

机译：新型的设计，合成和评价，分支三叉戟小干扰RNA纳米序列术术术级rNAi活性
2. Solid-phase synthesis, characterization and RNAi activity of branch and hyperbranch siRNAs [J] . MainaA., BlackmanB.A., ParronchiC.J., Bioorganic and Medicinal Chemistry Letters . 2013,第19期

机译：分支和超分支siRNA的固相合成，表征和RNAi活性
3. Synthesis of a stabilized Lu-177-siRNA complex and evaluation of its stability and RNAi activity [J] . Fathi Mojtaba, Yavari Kamal, Taghikhani Mohammad, Nuclear Medicine Communications . 2015,第6期

机译：稳定的Lu-177-siRNA复合物的合成及其稳定性和RNAi活性的评估
4. Enhanced RNAi Activity of Polyion Complex Micelles Composed of Poly(L-lysine) and LactosylatedPoly(ethylene glycol)-siRNA Conjugate Containing Acid-Labile Linkage [C] . Motoi Oishi, Yukio Nagasaki, Keiji Itaka, Annual meeting exposition of the Controlled Release Society . 2005

机译：包含酸不稳定键的聚（L-赖氨酸）和乳糖基化聚（乙二醇）-siRNA缀合物组成的聚离子复合物胶束的RNAi活性增强
5. Characterization of branched histidine-lysine polypeptides used for nucleic acid delivery and their complexes with DNA and small interfering RNA [D] . Tricoli, Lucas James 2013

机译：用于核酸递送的支链组氨酸赖氨酸多肽及其与DNA和小干扰RNA的复合物的表征
6. Design Synthesis Biochemical and Antiviral Evaluations of C6 Benzyl and C6 Biarylmethyl Substituted 2-Hydroxylisoquinoline-13-diones: Dual Inhibition against HIV Reverse Transcriptase-Associated RNase H and Polymerase with Antiviral Activities [O] . Sanjeev Kumar V. Vernekar, Zheng Liu, ∥, -1

机译：设计合成生化和抗病毒C6苄基和C6联芳甲基取代的2-羟基异喹啉-13-二酮的评估：对HIV逆转录酶相关的RNase H和聚合酶具有抗病毒活性的双重抑制
7. Design, Synthesis, Biochemical, and Antiviral Evaluations of C6 Benzyl and C6 Biarylmethyl Substituted 2-Hydroxylisoquinoline-1,3-diones: Dual Inhibition against HIV Reverse Transcriptase-Associated RNase H and Polymerase with Antiviral Activities [O] . Sanjeev Kumar V. Vernekar, Zheng Liu, Eva Nagy, 2014

机译：C6苄基和C6甲基甲基取代的2-羟基异喹啉-1,3-型抗体的设计，合成，生物化学和抗病毒评价：对HIV逆转录酶相关的RNase H和具有抗病毒活性的聚合酶的双重抑制作用

Design, synthesis and evaluation of novel, branched trident small interfering RNA nanostructures for sequence-specific RNAi activity

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