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Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
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机译:编码表达增加的重组fviii变体的病毒载体用于血友病a基因治疗
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摘要
The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.
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