LINE OF BIOLOGICALLY ACTIVE GENE-THERAPY SUBSTANCES BASED ON TGFB1 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 A61P25/28 

Abstract RU 2652312 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 TGFB1 gene, associated with a quantitative decrease in the protein of the transforming growth factor beta-1, 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 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 gen therapy substances, each being a genetic construct based on a vector plasmid, including the cDNA of the TGFB1 gene, with the beta-1 transforming growth factor protein coding sequence, with deletions of 5' and 3' non-translated regions, and also containing regulatory elements that ensure transcription of this sequence in eukaryotic cells of human organs and tissues, 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 TGFB1 gene, associated with a quantitative decrease in the protein of the transforming growth factor beta-1.

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

5 cl, 20 dwg, 18 ex

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RU 2 652 312 C2

Authors

Savelieva Natalia

Gamolski Anton

Dates

2018-04-25Published

2016-01-20Filed