FACTOR IX POLYPEPTIDE MUTANT, ITS USES AND METHOD FOR ITS PRODUCTION
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Accused Products
Abstract
Disclosed are a modified FIX (factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.
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30 Claims
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1-10. -10. (canceled)
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11. A method of treating a coagulopathy in a human patient, comprising administering a vector to the human patient, wherein:
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a. the vector is an adeno-associated virus; b. the vector comprises a nucleic acid encoding a modified FIX polypeptide, the modified FIX polypeptide comprising at least 70% identity to SEQ ID NO;
2 and a leucine in position 338 of SEQ ID NO;
2; andc. the vector comprises promoter sequences and transcription termination and control elements; thereby treating the coagulopathy. - View Dependent Claims (12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, 27, 28, 29, 30)
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Specification