Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
AssigneeVrije Universiteit Brussel
InventorsThierry Vandendriessche, Marinee Chuah
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Abstract
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.
Claims21
A vector comprising a nucleic acid expression cassette comprising a liver-specific regulatory element consisting of the Serpin enhancer having the nucleic acid sequence of SEQ ID NO:8, a promoter, a minute virus of mice (MVM) intron, a codon-optimized transgene, and a transcriptional termination signal.
The vector according to 2, wherein the B domain of said FVII is replaced by a linker having the nucleic acid sequence of SEQ ID NO:
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Dependencies are inferred from the claim text.