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Vectors and methods and uses for liver-directed gene therapy for hemophilia
Vectors and methods and uses for liver-directed gene therapy for hemophilia
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机译：血友病肝相关基因治疗的载体，方法和用途
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摘要
The present invention relates to vectors containing liver-specific regulatory sequences and codon-optimized factor IX or factor VIII genes, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and codon-optimized factor IX or factor VIII genes are also disclosed. The present invention is particularly useful for applications using gene therapy, in particular for the treatment of hemophilia A and B.
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