COMPOSITIONS BASED ON iRNA SERPINA1 AND THEIR APPLICATION METHODS Russian patent published in 2021 - IPC C12N15/113 C12N5/10 C12N15/85 A61K48/00 A61P35/00 

FIELD: biotechnology.

SUBSTANCE: group of inventions is described, including an agent based on double-stranded RNA (hereinafter – dsRNA) that inhibits the expression of Serpina1 gene (options), a pharmaceutical composition (options), an isolated human cell, a method for inhibiting the expression of Serpina1 in a cell in vitro, the use of the agent based on dsRNA or the pharmaceutical composition in the production of a drug for the treatment of a subject, the use of the agent based on dsRNA or the pharmaceutical composition in the production of a drug for inhibiting the development of hepatocellular carcinoma in a subject with a defective Serpina1 option, the use of the agent based on dsRNA or the pharmaceutical composition in the production of a drug for reducing the accumulation of improperly folded Serpina1 in the liver of a subject with the defective Serpina1 option, the use of the pharmaceutical composition containing the agent based on dsRNA that inhibits the expression of Serpina1 gene in the production of a drug for the treatment of a subject.

EFFECT: invention expands the arsenal of means for inhibiting the expression of the gene of serine peptidase inhibitor, clade A, representative 1 (Serpina1).

50 cl, 3 dwg, 5 tbl, 2 ex