LINE OF BIOLOGICALLY ACTIVE GENE-THERAPY SUBSTANCES BASED ON TGFBR2 GENE FOR CORRECTION OF PATHOLOGICAL STATES OF CELLS OF ORGANS AND TISSUES AND HUMAN ORGANS AND TISSUES, METHOD OF OBTAINING AND USING Russian patent published in 2018 - IPC C12N15/00 A61P35/00 

Abstract RU 2652351 C2

FIELD: medicine.

SUBSTANCE: group of inventions relates to medicine and concerns a means for correcting the pathological conditions of the cells of organs and tissues and/or human organs and tissues based on the TGFBR2 gene, associated with a quantitative decrease in the TGFBR2 protein, where the organs and tissues cells are selected from fibroblasts, keratocytes and epithelial cells of the eye, chondroblasts; organs and tissues are selected from the skin, cartilage tissue or 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 TGFBR2 gene, with the TGFBR2 protein coding sequence, with deletions of 5' and 3' non-translated regions, 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 gene TGFBR2, associated with a quantitative decrease in the protein TGFBR2.

EFFECT: group of inventions provides a high and stable level of the target protein in the cells.

5 cl, 18 ex, 20 dwg

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RU 2 652 351 C2

Authors

Savelieva Natalia

Gamolski Anton

Dates

2018-04-25Published

2016-01-20Filed