FIELD: biotechnology.
SUBSTANCE: what is declared is a group of inventions comprising a modified oligonucleotide having the sequence SEQ ID NO: 33, or a salt thereof, the sodium salt of said oligonucleotide, a pharmaceutical composition, containing a therapeutically effective amount of said modified oligonucleotide for treating a disease associated with C9ORF72 (versions), a pharmaceutical composition, containing a therapeutically effective amount of a modified oligonucleotide sodium salt for treating a C9ORF72 associated disease. In one embodiment, the modified oligonucleotide is characterized by a nucleic acid sequence 5'-GCCCCTAGCGCGCGACTC-3' (SEQ ID NO: 33), wherein each cytosine is 5-methylcytosine; and where internucleoside linkages of the modified oligonucleotide, in direction 5'–3' are soosssssssssoooss, where each s is a thiophosphate bond, and each o is a phosphodiester linkage.
EFFECT: invention extends the range of products for treating diseases associated with C9ORF72.
20 cl, 17 tbl, 14 ex
| Title | Year | Author | Number |
|---|---|---|---|
| COMPOUND FOR MODULATION OF C9ORF72 GENE EXPRESSION AND USE THEREOF | 2013 |
|
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| COMPOSITION FOR MODULATION OF C90RF72 EXPRESSION | 2014 |
|
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|
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| COMPOSITIONS AND METHODS FOR MODULATING PKK EXPRESSION | 2015 |
|
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| COMPOSITIONS FOR MODULATION OF ATAXIN 2 EXPRESSION | 2015 |
|
RU2702838C2 |
| COMPOSITIONS AND METHODS FOR MODULATING EXPRESSION OF APOLIPOPROTEIN (A) | 2014 |
|
RU2699985C2 |
| MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPOPROTEIN LIPASE DEFICIENT (LPLD) POPULATIONS | 2014 |
|
RU2661781C2 |
