TREATMENT OF DISEASES BY MEANS OF EXPRESSION OF ENZYME WITH DEOXYRIBONUCLEASE (DNase) ACTIVITY IN LIVER Russian patent published in 2022 - IPC C12N15/86 C12N9/22 A61K35/761 A61P25/28 A61P35/00 A61P9/10 A61K48/00 A61P35/04 

FIELD: medicine.

SUBSTANCE: invention relates to the treatment of a disease or a condition in a subject accompanied by the accumulation of extracellular DNA (hereinafter – ecDNA) in the port-sinusoidal blood flow of the liver. A method for the treatment of the above-mentioned disease or condition in a subject is proposed, including the administration to the subject of therapeutically effective amount of a recombinant expression vector of an adeno-associated virus (rAAV), containing (i) capsid protein and (ii) nucleic acid containing a liver-specific promoter functionally connected to a nucleotide sequence encoding an enzyme with deoxyribonuclease (hereinafter – DNase) activity. A method for the treatment of the above-mentioned disease or condition in a subject is also proposed, including the administration to the subject of an expression vector containing nucleic acid containing a promoter functionally connected to a sequence encoding an enzyme with DNase activity, where the promoter is a liver-specific promoter and/or where the vector includes one or more molecules targeting nucleic acid into liver cells.

EFFECT: invention provides an increase in the clearance of ecDNA accumulated in the port-sinusoidal blood flow of the liver.

24 cl, 21 dwg, 13 tbl, 15 ex