Compositions for modulating C9ORF72 expression
First Claim
1. A compound comprising a modified oligonucleotide consisting of 15 to 30 linked nucleosides and having a nucleobase sequence that is at least 90% complementary to an equal length portion of nucleobases 8213-8325 of SEQ ID NO:
- 2.
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Accused Products
Abstract
Disclosed herein are compositions and methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C90RF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon 1B of a C90RF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds.
51 Citations
34 Claims
- 1. A compound comprising a modified oligonucleotide consisting of 15 to 30 linked nucleosides and having a nucleobase sequence that is at least 90% complementary to an equal length portion of nucleobases 8213-8325 of SEQ ID NO:
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32. A compound comprising a modified oligonucleotide consisting of 14 to 30 linked nucleosides, wherein the modified oligonucleotide comprises at least 14 consecutive nucleobases of a sequence selected from SEQ ID NOs:
- 555 to 590, 1054, 1319 to 1365, and 1489 to 1491.
- View Dependent Claims (33, 34)
Specification