Claims (21)
1. A pharmaceutical composition comprising: a modified recombinant FIX (factor IX) polypeptide comprising at least 70% identity to SEQ ID NO: 2 and a leucine in position 338 of SEQ ID NO: 2; the modified recombinant FIX polypeptide in an amount suitable to provide a daily dosage comprised between 0.1 μg/kg and 400 μg/kg body weight, and at least one pharmaceutically acceptable excipient.
6. A sequence of nucleotides encoding a modified FIX (Factor IX) polypeptide comprising at least 70% identity to SEQ ID NO: 2 and a leucine at position 338 of SEQ ID NO: 2.
8. A nucleotide sequence encoding a modified FIX (Factor IV) polypeptide comprising 100% identity to SEQ ID NO: 2 except for an alanine at position 148 and a leucine at position 338 of SEQ ID NO: 2 in combination with positions corresponding to positions 34098, 34099 and 34100 of a sequence having accession number K02402 (GenBank) having a triplet selected from the group consisting of: TTA, UUA, TTG, UUG, CTT, CUU, CTC, CUC, CTA, CUA, CTG, and CUG , GAT, GAU and GAC .
Show 18 dependent claims
2. The pharmaceutical composition according to claim 1 , wherein the polypetide comprises at least 90% identity to SEQ ID NO: 2.
3. The pharmaceutical composition according to claim 1 formulated for oral, parenteral or rectal administration or suited to administration by inhalation or insufflation.
4. A method of treatment, the method comprising administering to an individual in need thereof the pharmaceutical composition according to claim 1 .
5. The method according to claim 4 wherein the administering is performed to treat at least one coagulopathy in the individual.
7. The sequence of nucleotides according to claim 6 , wherein the nucleotide sequence has at least 90% homology with the sequence having accession number K02402 (GenBank).
9. A nucleic acid comprising the nucleotide sequence according to claim 6 .
10. The nucleic acid according to claim 9 , the nucleic acid comprising a promoter in operational linkage with said nucleotide sequence.
11. A vector comprising the nucleic acid according to claim 9 .
12. A pharmaceutical composition comprising the sequence of nucleotide according to claim 6 .
13. A method for producing a modified FIX (Factor IX) polypeptide, the method comprising expressing the modified FIX polypeptide by means of a nucleic acid according to claim 9 .
14. A method to perform gene therapy, the method comprising administering to an individual in need thereof the nucleotide sequence according to claim 6 via a vector configured for gene therapy.
15. The method according to claim 14 , wherein the administering is performed to treat at least one coagulopathy in the individual.
16. The nucleotide sequence of claim 8 , wherein the triplet is selected from the group consisting of CTC and CTG.
17. The nucleotide sequence of claim 8 , wherein the triplet is selected from the group consisting of CUC and CUG.
18. The nucleotide sequence of claim 8 , wherein the triplet is CTC.
19. The nucleotide sequence of claim 8 , wherein the triplet is CTG.
20. The nucleotide sequence of claim 8 , wherein the triplet is CUC.
21. The nucleotide sequence of claim 8 , wherein the triplet is CUG.
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Citations
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