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Vitamin B6-coupled poly(ester amine) as gene carrier and application in cancer gene therapy

机译:维生素B6偶联聚(酯胺)作为基因载体及其在癌症基因治疗中的应用

摘要

The present invention relates to a vitamin B6-coupled poly(ester amine) (VBPEA) as a gene carrier and a method for preparing the gene carrier. Moreover, the present invention relates to a gene delivery complex comprising a therapeutic gene coupled to the gene carrier and a pharmaceutical formulation for gene therapy, which comprises the gene delivery complex as an active ingredient. In addition, the present invention relates to gene therapy utilizing the gene carrier, the gene delivery complex or the pharmaceutical formulation. The VBPEA of the invention has a significantly high gene delivery rate compared to existing gene carriers and a complex of the VBPEA with DNA has little or no cytotoxicity and shows a very high in vivo transfection efficiency. In addition, a complex of the VBPEA with siRNA shows high gene silencing efficiency and can induce a high rate of cell death and the inhibition of cell proliferation in cancer cells, suggesting that it can be used for anticancer gene therapy. Thus, the gene carrier VBPEA of the invention can be used as an experimental gene carrier and can also be widely used in gene therapy against various diseases depending on the kind of therapeutic gene.
机译:本发明涉及作为基因载体的维生素B6偶联的聚(酯胺)(VBPEA)及其制备方法。此外,本发明涉及一种基因递送复合物,其包含与基因载体偶联的治疗性基因和用于基因治疗的药物制剂,其包含所述基因递送复合物作为活性成分。另外,本发明涉及利用基因载体,基因递送复合物或药物制剂的基因治疗。与现有的基因载体相比,本发明的VBPEA具有显着高的基因递送速率,并且VBPEA与DNA的复合物几乎没有或没有细胞毒性,并且显示出非常高的体内转染效率。此外,VBPEA与siRNA的复合物显示出高的基因沉默效率,并且可以诱导癌细胞中的高细胞死亡率和细胞增殖抑制作用,表明它可以用于抗癌基因治疗。因此,根据治疗基因的种类,本发明的基因载体VBPEA可以用作实验性基因载体,并且还可以广泛用于针对各种疾病的基因治疗。

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