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OPTIMIZED HUMAN CLOTTING FACTOR IX GENE EXPRESSION CASSETTES AND THEIR USE
OPTIMIZED HUMAN CLOTTING FACTOR IX GENE EXPRESSION CASSETTES AND THEIR USE
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机译:优化人凝血因子IX基因表达盒及其使用
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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 optimized polynucleotide sequences encoding Factor IX proteins, vector comprising the same, and methods of using these compositions to treat a bleeding disorder.
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