OLIGOMERS TARGETING HEXANUCLEOTIDE REPEAT EXPANSION IN HUMAN C9ORF72 GENE

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专利权人:

PFIZER INC.

发明人:

JENSEN, Mads Aaboe,LINDOW, Morten

申请号：

WO2015IB56080

公开号：

WO2016024205(A8)

申请日:

2015.08.10

申请国别(地区):

世界知识产权组织国际局

年份:

2016

代理人:

摘要：

The disclosure relates to oligomers capable of targeting RNA expressed from the human C9ORF72 gene containing a pathogenic hexanucleotide repeat expansion. Such oligomers are useful for, among other things, reducing or eliminating C9ORF72 RNA and/or proteins translated therefrom, and treating or preventing diseases or disorders caused by, or associated with, hexanucleotide repeat expansion, including familial frontotemporal dementia (FTD) and familial amyotrophic lateral sclerosis (ALS).

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中国工程科技知识中心

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http://www.ckcest.cn/home/