OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION Russian patent published in 2021 - IPC C12N15/11 C12N15/113 

FIELD: biotechnology.

SUBSTANCE: invention relates to biotechnology. Disclosed is a set of inventions including an antisense oligonucleotide inducing UBE3A expression in a target cell where the paternal UBE3A expression is suppressed, a pharmaceutical composition containing the above-mentioned antisense oligonucleotide, a method for inducing UBE3A expression in a target cell where the paternal UBE3A expression is suppressed, the use of an antisense oligonucleotide or pharmaceutical composition in the treatment or prevention of Angelman syndrome and the use of an antisense oligonucleotide or pharmaceutical composition for the preparation of a drug for use in the treatment or prevention of Angelman syndrome. The antisense oligonucleotide according to the present invention is an oligonucleotide compound TTAcActtaattatactTCC where the capital letters represent beta-D-oxy LNA nucleosides, the lowercase letters represent DNA nucleosides, all C LNA represent 5-methylcytosine and all the inter-nucleoside bonds represent phosphorothioate inter-nucleoside bonds.

EFFECT: invention expands the arsenal of methods for inducing the UBE3A expression in the target cell.

12 cl, 2 dwg, 10 tbl, 7 ex