Factor IX polypeptide mutant, its uses and method for its production

  • US 9,982,248 B2
  • Filed: 07/14/2017
  • Issued: 05/29/2018
  • Est. Priority Date: 09/15/2008
  • Status: Active Grant
First Claim
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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.

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