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首页> 外文期刊>Polymer Preprints >Effects of polymer topology upon transfection efficiency: synthesis, characterization, and transfection of poly-2-(dimethylamlno)ethyl methacrylate and poly-2-(dimethylamino)ehtyl-co-polyethylene glycol methacrylate
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Effects of polymer topology upon transfection efficiency: synthesis, characterization, and transfection of poly-2-(dimethylamlno)ethyl methacrylate and poly-2-(dimethylamino)ehtyl-co-polyethylene glycol methacrylate

机译:聚合物拓扑结构对转染效率的影响:甲基丙烯酸聚-2-(二甲基氨)乙酯和聚甲基丙烯酸-2-(二甲氨基)乙-共-聚乙二醇的合成,表征和转染

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摘要

The promise of gene therapy involves the treatment of inherited or acquired genetic disorders through the transfection of an encoding DNA sequence into a target cell, which allows for the synthesis of a therapeutic protein. Due to the negatively charged phosphate backbone of DNA, direct injection of naked DNA hardly passes through cellular membranes and has the increased vulnerability of degradation by the host cell immune response. Gene transfer agents, carrier molecules, transport genetic material (DNA or RNA) and allows for more efficient Iran section. Significant research has been devoted to the design of viral gene transfer agents, which have remarkable transfection efficiencies.
机译:基因治疗的前景涉及通过将编码的DNA序列转染至靶细胞来治疗遗传性或获得性遗传疾病,从而合成治疗性蛋白质。由于DNA的磷酸骨架带负电荷,裸DNA的直接注射几乎不穿过细胞膜,并且因宿主细胞免疫反应而具有更高的降解易损性。基因转移剂,载体分子可运输遗传物质(DNA或RNA),并能更有效地进行伊朗切片。已经进行了重要的研究以设计具有显着转染效率的病毒基因转移剂。

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