首页> 外国专利> MEANS FOR TREATMENT OF HUMAN BODY STATES RELATED TO DECREASE OF THE CAT GENE EXPRESSION LEVEL AND/OR REDUCTION OF THE CATALYSIS PROTEIN ACTIVITY BASED ON GENE-THERAPEUTIC SUBSTANCES WITH THE CAT GENE, METHOD FOR PRODUCTION AND USE

MEANS FOR TREATMENT OF HUMAN BODY STATES RELATED TO DECREASE OF THE CAT GENE EXPRESSION LEVEL AND/OR REDUCTION OF THE CATALYSIS PROTEIN ACTIVITY BASED ON GENE-THERAPEUTIC SUBSTANCES WITH THE CAT GENE, METHOD FOR PRODUCTION AND USE

机译:基于降低了CAT基因表达水平和/或降低基于CAT基因的治疗物质的催化蛋白活性的人体状态的治疗方法,生产和使用方法

摘要

FIELD: medicine.;SUBSTANCE: group of inventions related to medicine and refers to a means for correcting the cells states of various organs and tissues, as well as the human organs and tissues, related to a decrease in the expression of the CAT gene and/or a decrease in the activity of the catalase protein, based on gene-therapeutic substances with the CAT gene, where the organs and tissues cells are selected from fibroblasts, epithelial cells of the cornea, the organs and tissues are selected from the skin, the oral mucosa or muscle tissue, which represents at least one gene-therapeutic substance selected from the group of gene-therapeutic substances, each of which represents a gene construct based on a vector plasmid comprising the cDNA of the CAT gene, with the coding sequence of the catalase protein, with deletions of 5'- and 3'-untranslated regions, namely the obtained on the basis of the site of a native unmodified cDNA of the CAT gene SEQ ID No: 1 or a modified cDNA of the CAT gene, while the modified cDNA of the CAT gene is used 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, or a combination of these gene constructs. Group of inventions also relates to a method for the preparation of said agent for treating states of the human body associated with a decrease in the CAT gene expression and/or a decrease in activity of the catalase protein; method of using said agent for treating states of the human body associated with a decrease in the CAT gene expression and/or a decrease in activity of the catalase protein.;EFFECT: group of inventions provides a high and stable level of the target protein in the cells while minimizing the probability of embedding the elements of the gene construct in the patient's genome.;5 cl, 18 ex, 20 dwg
机译:技术领域:医学领域的发明组,是指用于纠正与CAT基因的表达减少有关的各种器官和组织以及人体器官和组织的细胞状态的手段。 /或基于CAT基因的基因治疗物质降低过氧化氢酶蛋白的活性,其中器官和组织细胞选自成纤维细胞,角膜上皮细胞,器官和组织选自皮肤,代表至少一种选自基因治疗物质的基因治疗物质的口腔粘膜或肌肉组织,其各自代表基于包含CAT基因的cDNA的载体质粒的基因构建体,其编码序列的过氧化氢酶蛋白,具有5'和3'非翻译区的缺失,即基于CAT基因SEQ ID No:1的天然未修饰的cDNA或CAT g的修饰的cDNA的位点获得的烯,而使用CAT基因的修饰cDNA 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,或这些基因构建体的组合。本发明还涉及一种制备所述用于治疗与CAT基因表达降低和/或过氧化氢酶蛋白活性降低有关的人体状态的药剂的方法。使用所述试剂治疗与CAT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:一组发明提供了高水平且稳定的靶蛋白。 5 cl,18 ex,20 dwg最小化将基因构建体的元素嵌入患者基因组中的可能性。

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