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A viral vector encoding a recombinant FVIII variant with increased expression for gene therapy of type A haemophilia
A viral vector encoding a recombinant FVIII variant with increased expression for gene therapy of type A haemophilia
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机译:用于A型血友病基因治疗的,编码表达增加的重组FVIII变体的病毒载体
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摘要
In another aspect, the invention provides codon-modified polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the invention also provides mammalian gene therapy vectors and methods for treating type A hemophilia.
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