Factor ix polypeptide mutant, its uses and a method for its production

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.

What is claimed is: 
     
         1 . An adeno-associated virus vector comprising:
 (a) a nucleic acid encoding a modified FIX polypeptide, the modified FIX polypeptide comprising SEQ ID NO:1 having a leucine at position 384 of SEQ ID NO:1 and an alanine or threonine at position 194 of SEQ ID NO:1; and   (b) a promoter sequence.   
     
     
         2 . A pharmaceutical composition comprising the adeno-associated virus vector of  claim 1 . 
     
     
         3 . A method of treatment of at least one coagulopathy in a patient in need thereof comprising administering the adeno-associated virus vector of  claim 1  to the patient. 
     
     
         4 . The method of  claim 3 , wherein the at least one coagulopathy is haemophilia B.