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RECOMBINANT VECTOR FOR GENE THERAPY FOR INSULIN-DEPENDENT DIABETES MELLITUS AND THERAPEUTIC COMPOSITIONS THEREOF

机译:胰岛素依赖性糖尿病基因治疗的重组载体及其治疗组合物

摘要

Disclosed are a recombinant vector for use in gene therapy for insulin-dependent diabetes mellitus and a therapeutic composition thereof. Following the injection of a beta -galactosidase expression vector having a K14 promoter gene, along with a Drosophola's P transposase expression helper vector, into murine skin in a liposome-mediated manner, the beta -galactosidase gene is expressed in the keratinocyte layer from 24 hours to 20 weeks after injection as measured by X-gal staining. With the enhancement effect and tissue specificity, the K14 promoter is applied for the expression of a human insulin gene in keratinocytes, thereby suggesting a new gene therapy method for treating insulin-dependent diabetes mellitus. When, in combination with the P-element expression helper vector, a human insulin expression vector with the K14 promoter is injected into the skin of diabetic mice, which lack insulin-producing beta -cells of the pancreas, their blood glucose levels are maintained in a normal range. IMAGE
机译:公开了用于基因治疗胰岛素依赖型糖尿病的重组载体及其治疗组合物。将具有K14启动子基因的β-半乳糖苷酶表达载体与果蝇的P转座酶表达辅助载体一起以脂质体介导的方式注射到鼠皮中后,从24小时开始,β-半乳糖苷酶基因在角质形成细胞层中表达。通过X-gal染色测量注射后20周内。 K14启动子具有增强作用和组织特异性,可用于角质形成细胞中人胰岛素基因的表达,从而为治疗胰岛素依赖型糖尿病提供了一种新的基因治疗方法。当与P元素表达辅助载体结合使用时,将具有K14启动子的人胰岛素表达载体注射到缺乏胰腺胰岛素生成β细胞的糖尿病小鼠皮肤中,即可维持其血糖水平。正常范围。 <图像>

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