首页> 外国专利> MEANS FOR CORRECTION OF PATHOLOGICAL CONDITIONS OF CELLS OF AUTHORITIES AND TISSUES AND / OR HUMAN ORGANS AND TISSUES BASED ON THE COL1A2 GENE ASSOCIATED WITH A QUANTITATIVE REDUCTION OF CHAIN ALPHA-2 PROTEIN

MEANS FOR CORRECTION OF PATHOLOGICAL CONDITIONS OF CELLS OF AUTHORITIES AND TISSUES AND / OR HUMAN ORGANS AND TISSUES BASED ON THE COL1A2 GENE ASSOCIATED WITH A QUANTITATIVE REDUCTION OF CHAIN ALPHA-2 PROTEIN

机译:基于链蛋白ALPHA-2定量还原的COL1A2基因的权威,组织和/或人体组织和细胞的病理学状况校正方法

摘要

FIELD: medicine.SUBSTANCE: group of inventions relates to medicine and concerns a means for correction of states of cells, different organs and tissues, as well as human organs and tissues, related to quantitative decrease of protein of collagen type i alpha 2 chain, based on genetic-therapeutic substances with the COL1A2 gene, where the cells of organs and tissues are selected from fibroblasts, keratocytes, chondroblasts and epithelial cells of the eye cornea, organs and tissues are selected from skin, cartilage tissue or muscle tissue of a person, representing at least one genetic-therapeutic substance, selected from the group of genetic therapeutic substances, each of which is a genetic construct based on a vector plasmid, containing the native complementary DNA of the COL1A2 gene SEQ ID №: 1 or a modified complementary DNA of the COL1A2 gene, wherein the modified complementary DNA of the COL1A2 gene is SEQ ID No: 2, or SEQ ID No: 3, or SEQ ID No: 4, or SEQ ID No: 5, or SEQ ID No: 6, or SEQ ID No: 7, combination of these genetic constructs, each of which also contains regulatory elements in combination with or without a transport molecule. Group of inventions also relates to a method for producing said agent for treating human body states, related to quantitative decrease of protein of collagen type i alpha 2 chain, based on genetic-therapeutic substances with the COL1A2 gene; method for using said agent for treating human body states related to decreased expression of the COL1A2 gene and/or decreased activity of the catalase protein.EFFECT: group of inventions provides a high and stable level of the target protein in the cells.5 cl, 18 ex, 20 dwg
机译:技术领域本发明的组涉及医学,并且涉及一种校正细胞,不同器官和组织以及人体器官和组织的状态的方法,该方法与i型胶原2型胶原蛋白的定量减少有关,基于具有COL1A2基因的遗传治疗物质,其中器官和组织的细胞选自人眼角膜的成纤维细胞,角化细胞,软骨母细胞和上皮细胞,器官和组织选自人的皮肤,软骨组织或肌肉组织代表至少一种遗传治疗物质,选自遗传治疗物质,每种物质都是基于载体质粒的遗传构建体,包含COL1A2基因SEQ ID№:1的天然互补DNA或修饰的互补物所述COL1A2基因的DNA,其中所述COL1A2基因的修饰的互补DNA是SEQ ID No:2,或SEQ ID No:3,或SEQ ID No:4,或SEQ ID No:5,或SEQ ID No:6或SEQ ID No:7,这些基因构建体的组合,每个遗传构建体还包含与或不与转运分子结合的调节元件。本发明还涉及基于具有COL1A2基因的遗传治疗性物质生产所述治疗人体状态的试剂的方法,所述方法涉及定量减少i型胶原2α胶原蛋白。使用所述试剂治疗与COL1A2基因表达降低和/或过氧化氢酶蛋白活性降低相关的人体状态的方法。效果:一组发明在细胞中提供了高水平且稳定的靶蛋白。5cl, 18 ex,20 dwg

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