FIELD: medicine.
SUBSTANCE: group of inventions refers to medicine and concerns a means for correcting the pathological conditions of the cells of organs and tissues and/or organs and human tissues based on the SOD1 gene associated with oxidative stress, where the cells of organs and tissues are selected from fibroblast cells, keratocytes and epithelial cells of the eye, chondroblasts; organs and tissues are selected from the skin, mucous membrane of human mouth 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, including the cDNA of the SOD1 gene, with the coding sequence of the superoxide dismutase-1 protein, with deletions of 5' and 3' non-translated regions, namely obtained from the site of the native, unmodified cDNA of the SOD1 gene, SEQ ID No: 1, or a modified cDNA of the SOD1 gene, 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 agent for correcting the pathological conditions of cells of organs and tissues and/or human organs and tissues based on the SOD1 gene, associated with a quantitative reduction in the SOD1 protein.
EFFECT: group of inventions provides a high and stable level of the target protein in the cells.
5 cl, 18 ex, 20 dwg
