FIELD: medicine.
SUBSTANCE: group of inventions refers to medicine and concerns a means for correcting the pathological conditions of cells of organs and tissues and/or organs and tissues of human based on the SOD2 gene associated with oxidative stress, where the cells of organs and tissues are selected from cells of fibroblasts, keratocytes and epithelial cells of the eye, chondroblasts; organs and tissues are selected from the skin, the mucous membrane of the human mouth or the human muscle tissue, which is a collection of biologically active gene therapy substances, each of which is a gene therapeutic substance selected from the group of gene therapeutic substances, each being a genetic construct based on a vector plasmid comprising the cDNA of the SOD2 gene, with the coding sequence of the superoxide dismutase-1 protein, with deletions of 5' and 3' non-translated regions, namely the SOD2 gene derived from the site of the native unmodified cDNA, SEQ ID No: 1, or a modified cDNA of the SOD2 gene, with the modified cDNA of the SOD2 gene using 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 genetic constructs, each of which also contains regulatory elements that ensure transcription of this sequence in eukaryotic cells, human organs and tissues, in combination with or without a transport molecule. Group of inventions also relates to a method for producing said agent; a method of using said agent for correcting the pathological conditions of cells of organs and tissues and/or human organs and tissues based on the SOD2 gene associated with a quantitative reduction in the SOD2 protein.
EFFECT: group of inventions provides a high and stable level of the target protein in the cells.
5 cl, 18 ex, 20 dwg
Authors
Dates
2018-04-23—Published
2016-01-19—Filed