Optimized human clotting factor VIII gene expression cassettes and their use
AssigneeThe University of North Carolina at Chapel Hill
InventorsXiao Xiao, Juan Li, Zhenhua Yuan
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Abstract
The invention relates to synthetic liver-specific promoters and expression constructs for producing polypeptides and functional nucleic acids in the liver of a subject. The invention further relates to Factor VIII proteins containing modifications in the amino acid sequence of the Factor VIII protein, as well as nucleic acid constructs encoding the Factor VIII proteins and methods of using these compositions to treat a bleeding disorder.
Claims16
An isolated polynucleotide comprising a synthetic liver specific promoter, wherein the promoter comprises the nucleotide sequence as set forth in SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.