COMPOSITIONS FOR MODULATION OF ATAXIN 2 EXPRESSION Russian patent published in 2019 - IPC C12N15/113 

FIELD: biotechnology.

SUBSTANCE: described is a compound containing a modified oligonucleotide consisting of 12–30 bound nucleosides and having a nucleotide base sequence containing a part of at least 8, at least 9, at least 10, at least 11, at least 12, at least at least 13, at least 14, at least 15, at least 16, at least 17, at least 18, at least 19 or at least 20 consecutive nucleotide bases, complementary: (a) parts of equal length of nucleotide bases 1053–1109 of SEQ ID NO: 1; (b) parts of equal length of nucleotide bases 606–656 of SEQ ID NO: 1; (c) parts of equal length of nucleotide bases 1693–1767 of SEQ ID NO: 1; (d) parts of equal length of nucleotide bases 1789–1826 of SEQ ID NO: 1; (e) parts of equal length of nucleotide bases 1844–1877 of SEQ ID NO: 1; (f) parts of equal length of nucleotide bases 1957–1988 SEQ ID NO: 1; (g) parts of equal length of nucleotide bases 2291–2350 of SEQ ID NO: 1; (h) parts of equal length of nucleotide bases 3082–3141 SEQ ID NO: 1; (i) parts of equal length of nucleotide bases 3903–3946 of SEQ ID NO: 1; (j) parts of equal length of nucleotide bases 4005–4054 of SEQ ID NO: 1 or (k) parts of equal length of nucleotide bases 4429–4454 of SEQ ID NO: 1; wherein the modified oligonucleotide comprises at least one modification selected from a modified saccharide and a modified internucleoside bond, and wherein the modified oligonucleotide is capable of reducing ataxin 2 mRNA levels. Also described is a compound containing a modified oligonucleotide consisting of 12–30 bound nucleosides and having a nucleotide base sequence comprising at least 8, at least 9, at least 10, at least 11, at least 12, at least 13, at least 14, at least 15, at least 16, at least 17, at least 18, at least 19 or at least 20 consecutive nucleotide bases on any of sequences of nucleotide bases of SEQ ID NO: 18, 11–17 or 19–165, wherein the modified oligonucleotide comprises at least one modification selected from a modified saccharide and a modified internucleoside bond, and wherein the modified oligonucleotide is capable of reducing ataxin 2 mRNA levels.

EFFECT: described compounds are suitable for treating, preventing or relieving ataxin 2 related diseases, disorders and pathological conditions; such ataxin-2 related diseases include spinal-cerebellar ataxia of type 2 (SCA2), amyotrophic lateral sclerosis (ALS) and Parkinson's disease.

18 cl, 13 tbl, 9 ex