COMPOSITION POUR LA PRÉVENTION OU LE TRAITEMENT DE L'ATAXIE SPINO-CÉRÉBELLEUSE DE TYPE 36

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专利权人:

Kyoto University

发明人:

申请号：

EP18802436.8

公开号：

EP3626821A1

申请日:

2018.05.17

申请国别(地区):

EP

年份:

2020

代理人:

摘要：

An object of the present invention is to provide ASO that can effectively suppress a formation of RNA foci spontaneously occurring in neurons having SCA36 mutation, without inducing the cleavage of NOP56 pre-mRNA. The present invention provides an oligonucleotide comprising a nucleotide sequence in which one or more nucleotide sequences represented by SEQ ID NO: 2 are consecutively connected and is complementary to pre-mRNA of NOP56 gene, wherein a structure formed by the hybridization of the oligonucleotide to the pre-mRNA is resistant to RNaseH.

来源网站：

中国工程科技知识中心

来源网址：

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