COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION
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Abstract
Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal. Such methods are useful to treat, prevent, ameliorate, or slow progression of neurodegenerative diseases in an individual in need thereof.
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Citations
40 Claims
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1-20. -20. (canceled)
- 21. A modified oligonucleotide according to the following formula:
- 29. A modified oligonucleotide according to the following formula:
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33. A compound comprising a modified oligonucleotide, wherein the modified oligonucleotide is a gapmer consisting of a 5′
- wing segment, a central gap segment, and a 3′
wing segment, wherein;the 5′
wing segment consists of six 2′
-O-methoxyethyl nucleosides,the central gap segment consists of ten β
-D-deoxyribonucleosides, andthe 3′
wing segment consists of four 2′
-O-methoxyethyl nucleosides;wherein the modified oligonucleotide has the nucleobase sequence 5′
-GGTTAATCTTTATCAGGTCT-3′
(SEQ ID NO;
49), wherein each cytosine is a 5-methylcytosine; and
wherein the internucleoside linkages of the modified oligonucleotide are, from 5′
to 3′
, sooooossssssssssoss, wherein each s is a phosphorothioate linkage and each o is a phosphodiester linkage.- View Dependent Claims (34, 35, 36, 37, 38, 39, 40)
- wing segment, a central gap segment, and a 3′
Specification