OPTIMIZED HUMAN CLOTTING FACTOR VIII GENE EXPRESSION CASSETTES AND THEIR USE
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United States of America
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Apr 3, 2025
app pub date -
Sep 13, 2024
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Sep 13, 2024
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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.
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