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 Russian patent published in 2018 - IPC A61K48/00 A61P39/06 

Abstract RU 2649814 C1

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

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RU 2 649 814 C1

Authors

Savelieva Natalia

Sadykova Galina Kadymovna

Savelieva Natalia

Dates

2018-04-04Published

2017-02-14Filed