Optimized human clotting factor IX gene expression cassettes and their use

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专利权人:

The University of North Carolina at Chapel Hill

发明人:

Xiao, Xiao,Li, Juan

申请号：

AU2018275549

公开号：

AU2018275549A1

申请日:

2018.05.31

申请国别(地区):

AU

年份:

2019

代理人:

摘要：

The invention relates to synthetic liver-specific promoters and expression constructs for producing polypeptides and functional nucleic acids in the liver of a subject. The invention further relates to optimized polynucleotide sequences encoding Factor IX proteins, vector comprising the same, and methods of using these compositions to treat a bleeding disorder.

来源网站：

中国工程科技知识中心

来源网址：

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