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首页> 外文期刊>Angewandte Chemie >Self-Assembled Fluorodendrimers Combine the Features of Lipid and Polymeric Vectors in Gene Delivery
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Self-Assembled Fluorodendrimers Combine the Features of Lipid and Polymeric Vectors in Gene Delivery

机译:自组装的氟树枝形分子在基因传递中结合了脂质和高分子载体的特征

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摘要

An ideal vector in gene therapy should exhibit high serum stability, excellent biocompatibility, a desired transfection efficacy and permeability into targeted tissues. Here, we describe a class of low-molecular-weight fluorodendrimers for efficient gene delivery. These materials self-assemble into uniform nanospheres and allow for efficient transfection at low charge ratios and very low DNA doses with minimal cytotoxicity. Our results demonstrate that these vectors combine the features of synthetic gene vectors such as liposomes and cationic polymers and present promising potential for clinical gene therapy.
机译:基因治疗中理想的载体应表现出较高的血清稳定性,出色的生物相容性,所需的转染功效和对靶组织的通透性。在这里,我们描述了一类用于有效基因传递的低分子量氟树状聚合物。这些材料自组装成均匀的纳米球,并允许以低电荷比和非常低的DNA剂量进行有效的转染,同时将细胞毒性降至最低。我们的结果表明,这些载体结合了合成基因载体(如脂质体和阳离子聚合物)的功能,并为临床基因治疗提供了有希望的潜力。

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