Wilson's disease gene therapy
There is described a new gene therapy approach for treating Wilson's disease in which a nucleic acid molecule is used which comprises a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO: 1. Also described are vectors comprising the nucleotide sequence and methods and uses thereof.
1. A nucleic acid molecule comprising a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has the sequence of SEQ ID NO.1.
2. A vector for expressing ATP7B protein, the vector comprising the nucleic acid molecule of claim 1 .
3. The vector of claim 2 , further comprising a liver specific promoter.
4. The vector of claim 2 , wherein the vector is an AAV vector.
5. The vector of claim 2 , wherein the vector is a single stranded vector.
6. A host cell comprising the nucleic acid molecule of claim 1 .
7. A pharmaceutical composition comprising the nucleic acid molecule of claim 1 , and one or more pharmaceutically acceptable excipients.
8. A pharmaceutical composition comprising the vector of claim 2 , and one or more pharmaceutically acceptable excipients.
9. A method of treating Wilson's disease comprising administering a therapeutically effective amount of the vector of claim 2 to a patient suffering from Wilson's disease.
10. A method for delivery of a nucleotide sequence encoding ATP7B protein to a subject, which method comprises administering to the said subject the vector of claim 2 .