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Models of cationic liposome mediated transfection

机译：阳离子脂质体介导的转染模型

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Synthetic gene delivery vectors are being developed for in vivo gene transfer applications, and these systems may circumvent the risks inherent in the use of recombinant virus vectors. The majority of synthetic delivery systems are based on the use of cationic amphiphiles to coat and condense polynucleotides, and to facilitate the uptake and release of the polynucleotide payload into somatic cells. Cationic amphiphile-based vector technology has benefited from over a decade of mechanistic and structure-function analyses, but many aspects of the pharmacology, toxicology, and cell biology of these systems remain unresolved. Important outstanding issues include the structure and characteristics of active and inactive particles, in vivo distribution and clearance of particles, and the cellular events involved in binding, cytoplasmic delivery, and nuclear uptake. These processes are interdependent, and therefore difficult to isolate and examine experimentally. The wide range of compounds, methodologies, and polynucleotides used to study these phenomena further complicate development of general principles. This article focuses on the molecular and cellular processes involved in cationic amphiphile-mediated transfection. Both primary data and current literature will be used to illuminate the complexity that impacts on the development and application of this class of synthetic gene delivery vectors.
机译：正在开发用于体内基因转移应用的合成基因递送载体，并且这些系统可以规避使用重组病毒载体固有的风险。大多数合成递送系统基于使用阳离子两亲物来包被和浓缩多核苷酸，并促进多核苷酸有效载荷的摄取和释放到体细胞中。基于阳离子两亲物的载体技术得益于十多年来的机理和结构功能分析，但这些系统的药理学，毒理学和细胞生物学的许多方面仍未解决。重要的重要问题包括活性和非活性颗粒的结构和特性，颗粒在体内的分布和清除以及与结合，胞质传递和核吸收有关的细胞事件。这些过程是相互依赖的，因此很难进行分离和实验检验。用于研究这些现象的各种各样的化合物，方法学和多核苷酸进一步使一般原理的开发复杂化。本文重点介绍阳离子两亲物介导的转染涉及的分子和细胞过程。主要数据和当前文献都将用于阐明影响此类合成基因传递载体的开发和应用的复杂性。

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