NUCLEIC ACIDS FOR INHIBITION OF TARGET GENE EXPRESSION CONTAINING PHOSPHODITHIOATE BONDS Russian patent published in 2024 - IPC C12N15/11 C12N15/113 A61K31/00 

FIELD: biotechnology.

SUBSTANCE: following is described: a group of inventions including a nucleic acid for inhibiting the expression of a target gene in a cell (options), a conjugate for inhibiting the expression of a target gene in a cell (options), a composition for use as a medicinal product for the prevention or treatment of a disease, disorder or syndrome, with the purpose of which is to reduce the expression of target gene products, a pharmaceutical composition for the prevention or treatment of a disease, disorder or syndrome, the use of a nucleic acid or conjugate or pharmaceutical composition in the prevention or treatment of a disease, disorder or syndrome with the target to reduce the expression of target gene products and a method of preventing or treating a disease, disorder or syndrome with the target to reduce the expression of target gene products.

EFFECT: invention expands the arsenal of agents for inhibiting the expression of transthyretin (TTR) in the cell.

26 cl, 20 dwg, 8 tbl, 12 ex