Optimized human clotting factor IX gene expression cassettes and their use
AssigneeThe University of North Carolina at Chapel Hill
InventorsXiao Xiao, Juan Li
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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 optimized polynucleotide sequences encoding Factor IX proteins, vector comprising the same, and methods of using these compositions to treat a bleeding disorder.
Claims26
A polynucleotide comprising a synthetic liver-specific promoter, wherein the promoter comprises the nucleotide sequence of SEQ ID NO:
- 1.
A polynucleotide encoding a human Factor IX that has been codon optimized for expression in humans, further comprising a synthetic intron, wherein the codon-optimized Factor IX encoding sequence and synthetic intron comprise the nucleotide sequence of one of SEQ ID NOS:
- 6-8.
Dependencies are inferred from the claim text.