Vectors for Liver-Directed Gene Therapy of Hemophilia and Methods and Use Thereof

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专利权人:

VRIJE UNIVERSITEIT BRUSSEL

发明人:

Thierry Vandendriessche,Marinee Chuah

申请号：

US14438571

公开号：

US20150283267A1

申请日:

2012.10.25

申请国别(地区):

US

年份:

2015

代理人:

摘要：

The present invention relates to vectors containing liver-specific regulatory sequences and codon-optimized factor IX or factor VIII genes, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and codon-optimized factor IX or factor VIII genes are also disclosed. The present invention is particularly useful for applications using gene therapy, in particular for the treatment of hemophilia A and B.

来源网站：

中国工程科技知识中心

来源网址：

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