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Enhanced gene transfection performance and biocompatibility of polyethylenimine through pseudopolyrotaxane formation with alpha-cyclodextrin

机译:通过用α-环糊精形成通过假亚吡甲烷形成增强基因转染性能和聚乙胺的生物相容性

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摘要

Polyethylenimine (PEI), a commercially available gene transfection reagent, is a promising nonviral vector due to its inherent ability to efficiently condense genetic materials and its successful transfection performance in vitro. However, its low transfection efficiency in vivo, along with its high cytotoxicity, limit any further applications in gene therapy. To enhance the gene transfection performance and reduce the cytotoxicity of linear polyethylenimine, pseudopolyrotaxane PEI25k/CD and the polyrotaxanes PEI25k/CD-PA and PEI25k/CD-PB were prepared and their transfection efficiencies were then evaluated. The pseudopolyrotaxane PEI25k/CD exhibited better transfection efficiency and lower cytotoxicity than the transfection reagent linear PEI25k, even in the presence of serum. It also showed a remarkably higher cell viability, similar DNA protecting capability, and better DNA decondensation and release ability, and could be useful for the development of novel and safe nonviral gene delivery vectors for gene therapy.
机译:多乙基亚胺(PEI)是一种商业上可获得的基因转染试剂,是一种有前途的非血载体,因为其具有有效凝结材料的固有能力及其在体外的成功转染性能。然而,其体内的低转染效率以及其高细胞毒性,限制了基因治疗中的任何进一步应用。为了增强基因转染性能并减少线性聚乙烯亚胺的细胞毒性,制备伪亚吡烷PEI25K / CD和聚划线PEI25K / CD-PA和PEI25K / CD-PB,然后评价它们的转染效率。即使在血清存在下,伪唑烷PEI25K / CD也表现出比转染试剂线性PEI25K更好的转染效率和低细胞毒性。它还表现出具有显着更高的细胞活力,类似的DNA保护能力和更好的DNA裂缝和释放能力,并且可用于开发用于基因治疗的新颖和安全的非血流基因递送载体。

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