DOUBLE-STRANDED RNA CAPABLE OF REDUCING EXPRESSION OF MUTANT ALLELE C.607G>A OF HUMAN GNAO1 GENE Russian patent published in 2024 - IPC C12N15/113 C12N15/63 

FIELD: biotechnology.

SUBSTANCE: invention relates to RNA molecules capable of reducing the expression of the mutant allele c.607G>A of the human GNAO1 gene, triggering the mechanism of RNA interference, and can be used in medicine in the development of gene therapy approaches for patients with GNAO1 encephalopathy who have a mutation c.607G>A (p.Gly203Arg). The following is proposed: a double-stranded RNA containing a sense RNA sequence and an antisense RNA sequence that are complementary, wherein the antisense RNA sequence contains at least 19–22 nucleotides and is complementary to the target mRNA sequence SEQ ID NO: 28, or the sense RNA sequence is selected from the group consisting of SEQ ID NO: 1–17: Expression vectors containing DNA encoding the said double-stranded RNA are also proposed.

EFFECT: invention allows allele-selective inhibition of the expression of the mutant allele c.607G>A of the human GNAO1 gene.

17 cl, 17 dwg, 1 tbl, 4 ex