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 human organs and tissues based on the GPX1 gene, where the cells of organs and tissues are selected from fibroblasts, keratocytes and epithelial cells of the eye, chondroblasts; organs and tissues are selected from the skin, mucous membrane of the mouth or muscle tissue, which is a combination of gene therapy substances, each of which represents a gene therapy substance selected from a group of genetic therapeutic substances, each being a genetic construct based on a vector plasmid comprising the cDNA of the GPX1 gene, with the glutathione peroxidase-1 protein coding sequence, with deletions of 5' and 3' non-translated regions, namely obtained from the site of the native unmodified cDNA of the GPX1 gene SEQ ID No: 1, or a modified cDNA of the GPX1 gene, wherein as the modified cDNA of the GPX1 gene, 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 human cells 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 GPX1 gene.
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
2018-05-08—Published
2016-01-20—Filed