REAGENTS FOR TREATMENT OF OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) AND THEIR APPLICATION Russian patent published in 2021 - IPC C12N15/113 A61K48/00 A61K31/7088 A61P21/00 A61P27/02 C12N15/861 C12N15/864 C12N15/867 

FIELD: biotechnology.

SUBSTANCE: group of inventions is described, including a construct of DNA-directed RNA interference (hereinafter – ddRNAi) for the inhibition of PABPN1 protein expression, which is the cause of oculopharyngeal muscular dystrophy (hereinafter – OPMD), a DNA construct for the treatment of oculopharyngeal muscular dystrophy (OPMD), an expression vector containing the ddRNAi construct, a composition for the inhibition of PABPN1 protein expression (options), a method for the inhibition of PABPN1 protein expression, a method for the treatment of oculopharyngeal muscular dystrophy (OPMD) in a subject suffering from it, and a kit for the inhibition of PABPN1 protein expression.

EFFECT: invention expands the arsenal of means for the inhibition of PABPN1 protein expression.

43 cl, 20 dwg, 4 tbl, 9 ex