Oligodendrocyte-specific promoter, miRNA specific to PLP1 gene, vector including said promoter and/or miRNA, and pharmaceutical composition including said vector

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专利权人:

National Center of Neurology and Psychiatry;Nippon Medical School Foundation;Daiichi Sankyo Company, Limited

发明人:

INOUE, Ken,LI, Heng,OKADA, Takashi,OHKI, Yu,KOIZUMI, Makoto

申请号：

AU2019219286

公开号：

AU2019219286A1

申请日:

2019.02.06

申请国别(地区):

AU

年份:

2020

代理人:

摘要：

The purpose of the present invention is to provide a vector, a promoter and miRNA for the same, and a pharmaceutical composition including the vector, whereby expression of the PLP1 gene can be suppressed specifically in oligodendrocytes in order to treat PMD caused by an abnormality in the PLP1 gene. This oligodendrocyte-specific promoter includes a nucleic acid having at least 90% sequence identity with the base sequence represented by SEQ ID NO: 1. This miRNA specific to the PLP1 gene has a pair of base sequences comprising a predetermined antisense sequence and sense sequence.

来源网站：

中国工程科技知识中心

来源网址：

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