Compositions for modulating c9orf72 expression

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专利权人:

IONIS PHARMACEUTICALS INC.

发明人:

Rigo, Frank

申请号：

NZ73388216

公开号：

NZ733882A

申请日:

2016.04.15

申请国别(地区):

NZ

年份:

2020

代理人:

摘要：

Disclosed herein are compositions and methods for reducing expression of C90RF72 mRNA and protein in an animal. In particular, the invention relates to a modified antisense oligonucleotide against C90RF72, which has the nucleobases sequence of 5’-GCCCCTAGCGCGCGACTC-3’ and comprises a particular pattern of modification of the internucleoside linkages. Such nucleotide and methods are useful to treat, prevent, ameliorate, or slow progression of neurodegenerative diseases in an individual in need thereof.

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