Fabry disease gene therapy
There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional α-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.
1. A nucleic acid molecule comprising a nucleotide sequence encoding a polypeptide having α-galactosidase A activity wherein the nucleotide sequence has at least 91% identity to the nucleotide sequence of SEQ ID NO: 1.
2. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 95% identity to the nucleotide sequence of SEQ ID NO: 1.
3. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has the nucleotide sequence of SEQ ID NO: 1.
4. A vector, a host cell, a transgenic animal, or a pharmaceutical composition comprising the nucleic acid molecule of claim 1 .
5. The vector of claim 4 , wherein the nucleotide sequence is operably linked to a liver specific promoter.
6. The vector of claim 4 , wherein the vector is an AAV vector.
7. The vector of claim 4 , wherein the vector is a single stranded vector.
8. The vector of claim 4 , wherein the vector comprises a nucleotide sequence having at least 90% identity to the nucleotide sequence of SEQ ID NO: 3.
9. The vector of claim 4 , wherein the vector comprises a nucleotide sequence having the nucleotide sequence of SEQ ID NO: 3.
10. The vector of claim 4 , wherein the vector comprises a nucleotide sequence having at least 95% identity to the nucleotide sequence of SEQ ID NO: 3.
11. The vector of claim 4 , wherein the vector comprises a nucleotide sequence having at least 98% identity to the nucleotide sequence of SEQ ID NO: 3.
12. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 98% identity to the nucleotide sequence of SEQ ID NO: 1.
13. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 99% identity to the nucleotide sequence of SEQ ID NO: 1.
14. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 92% identity to the nucleotide sequence of SEQ ID NO: 1.
15. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 93% identity to the nucleotide sequence of SEQ ID NO: 1.
16. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 94% identity to the nucleotide sequence of SEQ ID NO: 1.
17. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 96% identity to the nucleotide sequence of SEQ ID NO: 1.
18. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence has at least 97% identity to the nucleotide sequence of SEQ ID NO: 1.