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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 using these vectors, and uses of these vectors. Also disclosed are expression cassettes and vectors comprising these liver-specific regulatory elements and codon optimized Factor IX or Factor VIII genes. The present invention is particularly useful for applications using gene therapy, particularly for the treatment of hemophilia A and B.
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