首页> 外国专利> LINE OF BIOLOGICALLY ACTIVE GENE-THERAPY SUBSTANCES BASED ON CLCA2 GENE FOR CORRECTION OF PATHOLOGICAL STATES OF CELLS OF ORGANS AND TISSUES AND HUMAN ORGANS AND TISSUES, METHOD OF OBTAINING AND USING

LINE OF BIOLOGICALLY ACTIVE GENE-THERAPY SUBSTANCES BASED ON CLCA2 GENE FOR CORRECTION OF PATHOLOGICAL STATES OF CELLS OF ORGANS AND TISSUES AND HUMAN ORGANS AND TISSUES, METHOD OF OBTAINING AND USING

机译:一种基于CLCA2基因的生物活性基因治疗物质线,用于纠正器官和组织细胞以及人类器官和组织的病理状态,获得和使用的方法

摘要

FIELD: medicine.;SUBSTANCE: group of inventions relates to medicine and concerns a means for correcting the pathological conditions of cells of organs and tissues and/or human organs and tissues based on the CLCA2 gene associated with a quantitative decrease in the calcium chloride-activated channel-2 protein, where the cells of organs and tissues are selected from cells of fibroblasts, keratocytes and epithelial cells of the eye, cells of the epithelium of the oral mucosa; organs and tissues are selected from the skin, mucous membrane of the oral cavity or human muscle tissue, which is a set of biologically active gene therapy substances, each of which is a gene therapy substance, selected from the group of gene therapy substances, each being a genetic construct based on a vector plasmid, comprising the cDNA of the CLCA2 gene with the coding sequence of the calcium chloride-activated channel-2 protein, with deletions of 5' and 3' non-translated regions, in combination with a transport molecule or without it. Group of inventions also relates to a method for producing said agent; a method of using said means for correcting the pathological conditions of cells of organs and tissues and/or human organs and tissues based on the CLCA2 gene associated with a quantitative decrease in the CLCA2 protein.;EFFECT: group of inventions provides a high and stable level of the target protein in the cells.;5 cl, 20 dwg, 18 ex
机译:发明领域本发明涉及医学,并且涉及一种基于CLCA2基因校正器官和组织和/或人体器官和组织的细胞的病理状况的手段,所述CLCA2基因与氯化钙的定量减少有关。活化通道2蛋白,其中器官和组织的细胞选自成纤维细胞,角膜细胞和眼上皮细胞,口腔粘膜上皮细胞。器官和组织选自皮肤,口腔粘膜或人的肌肉组织,它们是一组生物活性基因治疗物质,每种是基因治疗物质,选自基因治疗物质,每种是基于载体质粒的遗传构建体,包含CLCA2基因的cDNA和氯化钙激活的channel-2蛋白的编码序列,带有5'和3'非翻译区的缺失,并与转运蛋白结合分子或没有它。本发明组还涉及生产所述试剂的方法。一种使用所述手段基于与CLCA2蛋白的定量减少有关的CLCA2基因校正器官和组织和/或人体器官和组织的细胞的病理状况的方法。效果:一组发明提供了高而稳定的细胞中靶蛋白的水平; 5 cl,20 dwg,18 ex

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