AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE

1 . An aromatic L-amino acid decarboxylase (AADC) polynucleotide comprising an AADC sequence region which has at least 95% identity to SEQ ID NO: 979.

2 . The AADC polynucleotide of claim 1 , wherein the AADC sequence region comprises a promoter region, an enhancer region, a multiple cloning site (MCS) region and a polyadenylation (poly(A)) signal region.

3 . The AADC polynucleotide of claim 2 , wherein the AADC sequence region comprises at least one 5′ inverted terminal repeat (ITR) region and one 3′ ITR region.

4 . The AADC polynucleotide of claim 3 , wherein one or more of the 5′ ITRs are located 5′ to the MCS region and one or more of the 3′ ITRs are located 3′ to the poly(A) signal.

5 . The AADC polynucleotide of claim 4 , wherein the AADC sequence region comprises a first exon region, a first intron region, a second intron region and a second exon region.

6 . The AADC polynucleotide of claim 5 , wherein the enhancer region and the promoter region are derived from CMV.

7 . The AADC polynucleotide of claim 6 , wherein the first exon region is immediate-early 1 (ie1) exon 1 or fragments thereof, the first intron region is ie1 intron 1 or fragments thereof, the second intron region is human beta-globin (hBglobin) intron 2 or fragments thereof and the second exon region is hBglobin exon 3 or fragments thereof.

8 . The AADC polynucleotide of claim 7 , wherein the poly(A) signal is derived from human growth hormone.

9 . The AADC polynucleotide of claim 8 , wherein the AADC sequence region consists of 3520-3530 nucleotides from the 5′ end of the 5′ ITR to the 3′ end of the 3′ ITR.

10 . The AADC polynucleotide of claim 8 , wherein the AADC sequence region consists of 3526 nucleotides from the 5′ end of the 5′ ITR to the 3′ end of the 3′ ITR.

11 . A recombinant adeno-associated virus (rAAV) comprising the AADC polynucleotide of claim 8 .

12 . The rAAV virus of claim 11 , wherein the capsid serotype of the rAAV is AAV2.

13 . The AADC polynucleotide of claim 3 , wherein the AADC sequence region has at least 99% identity to SEQ ID NO: 979.

14 . The AADC polynucleotide of claim 13 , wherein the AADC sequence region consists of 3526 nucleotides from the 5′ end of the 5′ ITR to the 3′ end of the 3′ ITR.

15 . A recombinant adeno-associated virus (rAAV) comprising the AADC polynucleotide of claim 13 .

16 . The rAAV virus of claim 15 , wherein the capsid serotype of the rAAV is AAV2.

17 . A pharmaceutical composition comprising an adeno-associated virus (AAV) particle, wherein the AAV particle comprises an AAV capsid and a vector genome, and wherein the vector genome comprises at least one AADC sequence region with at least 95% identity to SEQ ID NO: 979.

18 . The pharmaceutical composition of claim 17 , wherein the AADC sequence region has at least 99% identity SEQ ID NO: 979.

19 . The pharmaceutical composition of claim 18 , wherein the AAV capsid serotype is AAV2.

20 . The pharmaceutical composition of claim 19 , wherein at least 70% of the AAV particles contain a vector genome.