ADENO-ASSOCIATED VIRAL VECTOR FOR EXON SKIPPING IN A GENE ENCODING A DISPENSIBLE-DOMAIN PROTEIN

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专利权人:

Luis Garcia

发明人:

Luis Garcia

申请号：

US13458037

公开号：

US20130072541A1

申请日:

2012.04.27

申请国别(地区):

US

年份:

2013

代理人:

摘要：

The invention concerns an adeno-associated viral vector comprising:a U7 type modified snRNA sequence；the native U7 promoter；at least one antisense sequence directed against at least one splice site of at least one exon, the said exon encoding a dispensable domain of dystrophin.

来源网站：

中国工程科技知识中心

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