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Optimized Human Coagulation Factor IX Gene Expression Cassette and Use thereof
Optimized Human Coagulation Factor IX Gene Expression Cassette and Use thereof
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机译:优化的人凝血因子IX基因表达盒及其用途
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摘要
The present invention relates to synthesized 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, vectors comprising them, and methods of using these compositions to treat bleeding disorders.
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