FIELD: medicine; pharmaceuticals.
SUBSTANCE: group of inventions relates to compounds and methods for reducing expression of C9ORF72 gene and/or protein containing hexanucleotide repeat expansion, in animals by means of specific C9ORF72 inhibitors. Such specific inhibitors of C9ORF72 include antisense compounds containing a modified oligonucleotide, consisting of 16–20 bound nucleosides and having a sequence of nucleotide bases containing at least 12 consecutive nucleotide bases, which are 100 % complementary to part of equal length from nucleotide bases 1446–1479 of SEQ ID NO: 2. Modified oligonucleotide can contain at least one modification selected from modified sugar and modified internucleoside bond.
EFFECT: compound, conjugates and compositions based thereon are suitable for treating, preventing or alleviating a neurodegenerative disease, associated with nucleic acid C9ORF72, containing expansion of hexanucleotide repeat, in the individual in need thereof.
55 cl, 28 tbl, 6 ex
| Title | Year | Author | Number |
|---|---|---|---|
| NUCLEIC ACID MOLECULES FOR REDUCING THE LEVEL OF PAPD5 OR PAPD7 MRNA FOR TREATING INFECTIOUS HEPATITIS B | 2017 |
|
RU2768699C2 |
| COMPOSITIONS FOR MODULATING TAU PROTEIN EXPRESSION | 2014 |
|
RU2735551C2 |
| COMPOSITIONS FOR MODULATING EXPRESSION OF TAU PROTEIN | 2014 |
|
RU2823361C1 |
| MODULATION OF REP PROTEIN ACTIVITY WHEN PRODUCING CLOSED-END DNA (ceDNA) | 2020 |
|
RU2812850C2 |
| COMPOSITION FOR MODULATION OF C90RF72 EXPRESSION | 2014 |
|
RU2748426C2 |
| COMPOSITIONS AND METHODS FOR REDUCING TAU EXPRESSION | 2016 |
|
RU2747734C2 |
| COMPOSITIONS AND METHODS FOR REDUCING TAU EXPRESSION | 2016 |
|
RU2777570C2 |
| ANIMALS OTHER THAN HUMANS CHARACTERIZED IN EXPANSION OF HEXANUCLEOTIDE REPEATS AT LOCUS C9orf72 | 2017 |
|
RU2760877C2 |
| MYOSTATIN SIGNAL INHIBITOR | 2019 |
|
RU2820270C2 |
| COMPOSITIONS CONTAINING CURONES AND WAYS OF APPLICATION THEREOF | 2018 |
|
RU2822800C2 |
