VITAMIN B6-COUPLED POLY(ESTER AMINE) AS GENE CARRIER AND APPLICATION IN CANCER GENE THERAPY
    13.
    发明申请
    VITAMIN B6-COUPLED POLY(ESTER AMINE) AS GENE CARRIER AND APPLICATION IN CANCER GENE THERAPY 有权
    维生素B6联合聚(ESTER AMINE)作为基因载体和在癌基因治疗中的应用

    公开(公告)号:US20150030854A1

    公开(公告)日:2015-01-29

    申请号:US14182031

    申请日:2014-02-17

    Abstract: 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.

    Abstract translation: 本发明涉及作为基因载体的维生素B6-偶联聚(酯胺)(VBPEA)和制备该基因载体的方法。 此外,本发明涉及包含与基因载体偶联的治疗基因和用于基因治疗的药物制剂的基因递送复合物,其包含基因递送复合物作为活性成分。 此外,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。 与现有基因载体相比,本发明的VBPEA具有显着高的基因传递速率,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,并且可以诱导高细胞死亡率和癌细胞中细胞增殖的抑制,这表明其可用于抗癌基因治疗。 因此,本发明的基因载体VBPEA可以用作实验基因载体,也可以根据治疗基因的种类广泛用于各种疾病的基因治疗。

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