Factor IX polypeptide mutant, its uses and method for its production
CAFCFirst Claim
Patent Images
1. A method of treating a coagulopathy in a human patient, comprising administering a vector to the human patient, wherein:
- 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; and
c. the vector comprises promoter sequences and transcription termination and control elements;
thereby treating the coagulopathy.
1 Assignment
2 Petitions
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.
41 Citations
20 Claims
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1. 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 (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20)
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Specification