COMPOSITIONS AND METHODS FOR INHELIBITED EXACT GENESIS ALAS1 Russian patent published in 2019 - IPC C12N15/113 C07H21/04 

FIELD: biotechnologies.

SUBSTANCE: invention relates to the biotechnology. Described is a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of ALAS1. Above dsRNA comprises a semantic suture and an antisense suture. Anti-sense suture is complementary to at least nucleotides 871–889 of SEQ ID NO: 1 and includes SEQ ID NO: 1296. Semantic thread contains at least 15 consecutive nucleotides sequence SEQ ID NO: 1295. Each of sense and antisense threads has length from 19 to 24 nucleotides. dsRNA comprises one or more nucleotides with modification, selected from 2'-O-methyl-modified nucleotide, 2'-fluorine-modified nucleotide or both. Also disclosed is a pharmaceutical composition for inhibiting ALAS1 gene expression containing a therapeutically effective amount of dsRNA. Methods of inhibiting the expression of ALAS1 in a cell, treating the disorder and reducing the level of porphyrin or a porphyrin precursor in a cell associated with ALAS1 expression, including dsRNA introduction.

EFFECT: invention enables to inhibit the expression of the ALAS1 gene.

45 cl, 15 dwg, 12 ex, 20 tbl