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本文引用的文献

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Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity.具有可预测体内siRNA递送活性的可降解脂质纳米颗粒。
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Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.用于在啮齿动物和非人类灵长类动物中进行有效和选择性 siRNA 递送的脂肽纳米颗粒。
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Delivery of therapeutic siRNA to the lung endothelium via novel Lipoplex formulation DACC.通过新型脂质体复合物配方DACC将治疗性小干扰RNA递送至肺内皮细胞。
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Influence of polyethylene glycol density and surface lipid on pharmacokinetics and biodistribution of lipid-calcium-phosphate nanoparticles.聚乙二醇密度和表面脂质对脂质-钙-磷纳米颗粒的药代动力学和生物分布的影响。
Biomaterials. 2014 Mar;35(9):3027-34. doi: 10.1016/j.biomaterials.2013.12.022. Epub 2014 Jan 2.
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Advances in Lipid Nanoparticles for siRNA Delivery.脂质纳米颗粒在 siRNA 递送上的进展。
Pharmaceutics. 2013 Sep 18;5(3):498-507. doi: 10.3390/pharmaceutics5030498.
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Gene-silencing potency of symmetric and asymmetric lipid-conjugated siRNAs and its correlation with dicer recognition.对称和非对称脂质缀合 siRNA 的基因沉默效力及其与 Dicer 的识别相关性。
Bioconjug Chem. 2013 Dec 18;24(12):2045-57. doi: 10.1021/bc400391n. Epub 2013 Dec 9.
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Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial.在健康志愿者中,一种 RNA 干扰药物对前蛋白转化酶枯草溶菌素/柯萨奇蛋白酶 9(PCSK9)合成和血清 LDL 胆固醇浓度的影响:一项随机、单盲、安慰剂对照、1 期临床试验。
Lancet. 2014 Jan 4;383(9911):60-68. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3.
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Multiparametric approach for the evaluation of lipid nanoparticles for siRNA delivery.用于评估用于 siRNA 递送的脂质纳米颗粒的多参数方法。
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Surfactant-free, lipo-polymersomes stabilized by iron oxide nanoparticles/polymer interlayer for synergistically targeted and magnetically guided gene delivery.无表面活性剂,由氧化铁纳米粒子/聚合物夹层稳定的脂多聚物囊泡用于协同靶向和磁导向基因传递。
Adv Healthc Mater. 2014 Feb;3(2):273-82. doi: 10.1002/adhm.201300122. Epub 2013 Jul 18.

用于基因递送的脂质纳米颗粒。

Lipid nanoparticles for gene delivery.

作者信息

Zhao Yi, Huang Leaf

机构信息

Division of Molecular Pharmaceutics and Center for Nanotechnology in Drug Delivery, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

出版信息

Adv Genet. 2014;88:13-36. doi: 10.1016/B978-0-12-800148-6.00002-X.

DOI:10.1016/B978-0-12-800148-6.00002-X
PMID:25409602
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5006671/
Abstract

Nonviral vectors which offer a safer and versatile alternative to viral vectors have been developed to overcome problems caused by viral carriers. However, their transfection efficacy or level of expression is substantially lower than viral vectors. Among various nonviral gene vectors, lipid nanoparticles are an ideal platform for the incorporation of safety and efficacy into a single delivery system. In this chapter, we highlight current lipidic vectors that have been developed for gene therapy of tumors and other diseases. The pharmacokinetic, toxic behaviors and clinic trials of some successful lipids particles are also presented.

摘要

为克服病毒载体所带来的问题,已开发出比病毒载体更安全、用途更广泛的非病毒载体。然而,它们的转染效率或表达水平远低于病毒载体。在各种非病毒基因载体中,脂质纳米颗粒是将安全性和有效性整合到单一递送系统中的理想平台。在本章中,我们重点介绍了目前已开发用于肿瘤和其他疾病基因治疗的脂质载体。还介绍了一些成功的脂质颗粒的药代动力学、毒性行为和临床试验情况。