Factor IX gene therapy
The invention relates to a new, more potent, coagulation Factor IX (FIX) expression cassette for gene therapy of Haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein, and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.
1. A nucleic acid molecule comprising a promoter, wherein the promoter has a nucleotide sequence having at least 90% identity to the sequence of SEQ ID NO. 4.
2. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 91% identity to the sequence of SEQ ID NO. 4.
3. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 92% identity to the sequence of SEQ ID NO. 4.
4. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 93% identity to the sequence of SEQ ID NO. 4.
5. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 94% identity to the sequence of SEQ ID NO. 4.
6. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 95% identity to the sequence of SEQ ID NO. 4.
7. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 96% identity to the sequence of SEQ ID NO. 4.
8. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 97% identity to the sequence of SEQ ID NO. 4.
9. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 98% identity to the sequence of SEQ ID NO. 4.
10. The nucleic acid molecule of claim 1 , wherein the promoter has a nucleotide sequence having at least 99% identity to the sequence of SEQ ID NO. 4.
11. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence of the promoter is the sequence of SEQ ID NO. 4.
12. The nucleic acid molecule of claim 1 , wherein the promoter is:
(a) between 330 and 380 nucleotides in length;
(b) between 345 and 365 nucleotides in length; or
(c) between 350 and 360 nucleotides in length.
13. A host cell comprising the nucleic acid molecule of claim claim 1 .
14. A pharmaceutical composition comprising the nucleic acid molecule of claim 1 and one or more pharmaceutically acceptable excipients.