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Optimized human clotting factor VIII gene expression cassettes and their use
Optimized human clotting factor VIII gene expression cassettes and their use
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机译:优化的人类凝血因子VIII基因表达盒及其应用
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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.
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