发明名称 |
OPTIMIZED HUMAN CLOTTING FACTOR VIII GENE EXPRESSION CASSETTES AND THEIR USE |
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 Factor VIII proteins containing modifications in the amino acid sequence of the Factor VIII protein, as well as nucleic acid constructs encoding the Factor VIII proteins and methods of using these compositions to treat a bleeding disorder. |
申请公布号 |
WO2016127057(A1) |
申请公布日期 |
2016.08.11 |
申请号 |
WO2016US16777 |
申请日期 |
2016.02.05 |
申请人 |
THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL |
发明人 |
XIAO, XIAO;LI, JUAN;YUAN, ZHENHUA |
分类号 |
C07K14/755;A61K38/37;A61P7/04;C12N5/10;C12N15/12;C12N15/85;C12P21/02 |
主分类号 |
C07K14/755 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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