Gene therapy and pharmaceutical composition for prevention and treatment of lung cancer
    2.
    发明申请
    Gene therapy and pharmaceutical composition for prevention and treatment of lung cancer 审中-公开
    用于预防和治疗肺癌的基因治疗和药物组合物

    公开(公告)号:US20090169485A1

    公开(公告)日:2009-07-02

    申请号:US12285867

    申请日:2008-10-15

    摘要: Disclosed herein is a gene therapeutic agent and pharmaceutical composition for the prevention and treatment of lung cancer. For aerosol delivery, chemically synthesized polyester amine is used as a carrier in the gene therapeutic agent. The polyester amine/Akt1 siRNA complex is found to be effectively delivered to the lungs of K-ras null mice through a nose-only inhalation system and to significantly suppress lung cancer progression as denoted by gene delivery efficiency and inhibition of Akt-related signals and cell cycle. Thus, the aerosol delivery of polyester amine-mediated Akt1 siRNA is provided as an effective model for noninvasive gene therapy.

    摘要翻译: 本文公开了用于预防和治疗肺癌的基因治疗剂和药物组合物。 对于气溶胶输送,化学合成的聚酯胺用作基因治疗剂中的载体。 发现聚酯胺/ Akt1 siRNA复合物通过仅鼻吸入系统被有效地递送到K-ras无效小鼠的肺中,并显着抑制肺癌进展,如基因递送效率和Akt相关信号的抑制以及 细胞周期。 因此,提供聚酯胺介导的Akt1 siRNA的气溶胶递送作为无创基因治疗的有效模型。

    Polysorbitol-based osmotically active transporter and gene therapy using same
    3.
    发明授权
    Polysorbitol-based osmotically active transporter and gene therapy using same 有权
    基于聚山梨糖醇的渗透活性转运蛋白和使用其的基因治疗

    公开(公告)号:US09138488B2

    公开(公告)日:2015-09-22

    申请号:US14007064

    申请日:2011-08-12

    摘要: The present invention relates to a biodegradable polysorbitol-based osmotically active transporter (PSOAT) and gene therapy using the same as a gene carrier. The biodegradable polysorbitol-based osmotically active transporter (PSOAT) of the present invention includes a sorbitol skeleton, which imparts an osmotic pressure to the cell membrane to improve membrane permeability, thereby exhibiting remarkably improved transfection efficiency. Further, the polysorbitol-based osmotically active transporter of the present invention exhibits high DNA binding ability, effectively protects DNA from nuclease, exhibits physicochemical properties suitable for use as a gene carrier, and has very low cytotoxicity in vitro and in vivo, thereby capable of being used as a gene carrier for gene therapy.

    摘要翻译: 本发明涉及可生物降解的聚山梨糖醇基渗透活性转运蛋白(PSOAT)和使用其作为基因载体的基因治疗。 本发明的可生物降解性聚山梨糖醇类渗透活性转运体(PSOAT)包括赋予细胞膜渗透压以改善膜通透性的山梨糖醇骨架,从而显着提高转染效率。 此外,本发明的基于聚山梨醇醇的渗透性活性转运体显示出高的DNA结合能力,有效地保护DNA免受核酸酶的影响,表现出适合用作基因载体的物理化学性质,并且在体外和体内具有非常低的细胞毒性, 被用作基因治疗的基因载体。

    Biopolymer and gene complex
    5.
    发明授权
    Biopolymer and gene complex 有权
    生物聚合物和基因复合物

    公开(公告)号:US08066978B2

    公开(公告)日:2011-11-29

    申请号:US11571890

    申请日:2005-03-23

    IPC分类号: A61K31/74 C12N15/00 C07H21/02

    摘要: Disclosed herein is a biopolymer/gene complex for the aerosol delivery of a gene. The biopolymer/gene complex comprises a polyethyleneimine (PEI) having a substitution of glucose for at least a portion of the primary amino groups of the polyethyleneimine, and the content of the polyethyleneimine is 2-4 times the content of the gene. Also, disclosed is a lung cancer therapeutic agent comprising a biopolymer/gene complex including a polyethyleneimine (PEI) having a substitution of glucose for 30-40 mol % of the primary amino groups of the polyethyleneimine, and a gene encoding PTEN, in which the content of the polyethyleneimine is 2-4 times the content of the gene.

    摘要翻译: 本文公开了用于基因的气溶胶递送的生物聚合物/基因复合物。 生物聚合物/基因复合物包含在聚乙烯亚胺的至少一部分伯氨基取代葡萄糖的聚乙烯亚胺(PEI),聚乙烯亚胺的含量为基因含量的2-4倍。 此外,公开了包含生物聚合物/基因复合物的肺癌治疗剂,所述生物聚合物/基因复合物包括具有替代聚乙烯亚胺的30-40摩尔%的伯氨基的葡萄糖的聚乙烯亚胺(PEI)和编码PTEN的基因,其中 聚乙烯亚胺的含量是基因含量的2-4倍。

    Polysorbitol-Based Osmotically Active Transporter and Gene Therapy Using Same
    6.
    发明申请
    Polysorbitol-Based Osmotically Active Transporter and Gene Therapy Using Same 有权
    基于聚山梨糖醇的渗透活性转运体和使用相同的基因治疗

    公开(公告)号:US20140348777A1

    公开(公告)日:2014-11-27

    申请号:US14007064

    申请日:2011-08-12

    摘要: The present invention relates to a biodegradable polysorbitol-based osmotically active transporter (PSOAT) and gene therapy using the same as a gene carrier. The biodegradable polysorbitol-based osmotically active transporter (PSOAT) of the present invention includes a sorbitol skeleton, which imparts an osmotic pressure to the cell membrane to improve membrane permeability, thereby exhibiting remarkably improved transfection efficiency. Further, the polysorbitol-based osmotically active transporter of the present invention exhibits high DNA binding ability, effectively protects DNA from nuclease, exhibits physicochemical properties suitable for use as a gene carrier, and has very low cytotoxicity in vitro and in vivo, thereby capable of being used as a gene carrier for gene therapy.

    摘要翻译: 本发明涉及可生物降解的聚山梨糖醇基渗透活性转运蛋白(PSOAT)和使用其作为基因载体的基因治疗。 本发明的可生物降解性聚山梨糖醇类渗透活性转运体(PSOAT)包括赋予细胞膜渗透压以改善膜通透性的山梨糖醇骨架,从而显着提高转染效率。 此外,本发明的基于聚山梨醇醇的渗透性活性转运体显示出高的DNA结合能力,有效地保护DNA免受核酸酶的影响,表现出适合用作基因载体的物理化学性质,并且在体外和体内具有非常低的细胞毒性, 被用作基因治疗的基因载体。