AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE

1 . An AAV vector genome comprising, in order:

a) a 5′ inverted terminal repeat (ITR), wherein said 5′ ITR is 141 nucleotides in length;

b) an AADC sequence region, said AADC sequence region comprising a nucleotide sequence encoding SEQ ID NO: 1,

c) a 3′ ITR, wherein said 3′ ITR is 141 nucleotides in length.

2 . The AAV vector genome of claim 1 , wherein the 5′ ITR and the 3′ ITR are derived from AAV2.

3 . The AAV vector genome of claim 2 , wherein the AAV vector genome comprises a cytomegalovirus (CMV) sequence region derived from a CMV gene; wherein the CMV sequence region comprises an enhancer region and promoter region.

4 . The AAV vector genome of claim 3 , wherein the CMV sequence region is 507 nucleotides in length.

5 . The AAV vector genome of claim 2 , wherein the AAV vector genome comprises an immediate early 1 (IE1) sequence region derived from an IE1 gene.

6 . The AAV vector genome of claim 5 , wherein the IE1 sequence region comprises a nucleotide sequence from IE1 exon1.

7 . The AAV vector genome of claim 6 , wherein the IE1 sequence region comprises a nucleotide sequence from IE1 intron 1 or a fragment thereof.

8 . The AAV vector genome of claim 7 , wherein the IE1 sequence region is 166 nucleotides in length.

9 . The AAV vector genome of claim 2 , wherein the AAV vector genome comprises a human beta globin (BB) sequence region derived from a HB gene.

10 . The AAV vector genome of claim 9 , wherein the HB sequence region comprises a nucleotide sequence from HB intron 2.

11 . The AAV vector genome of claim 10 , wherein the HB sequence region comprises a nucleotide sequence from HB exon 3.

12 . The AAV vector genome of claim 11 , wherein the HB sequence region is 400 nucleotides in length.

13 . The AAV vector genome of claim 2 , wherein the AAV vector genome comprises a poly(A) signal sequence region derived from the human growth hormone (hGH) gene.

14 . The AAV vector genome of claim 13 , wherein the poly(A) signal sequence region is 477 nucleotides in length.

15 . The AAV vector genome of claim 1 , wherein the AAV vector genome comprises a sequence selected from the group consisting of SEQ ID NO: 10-13, 15, 17-20 and 22 and variants having at least 95% identity thereto.

16 . The AAV vector genome of claim 2 , wherein, the AADC region comprises a codon optimized nucleotide sequence encoding SEQ ID NO: 1.

17 . The AAV vector genome of claim 16 , wherein the AAV vector genome comprises a sequence selected from the group consisting of SEQ ID NO: 7 and 12 or variants having at least 95% identity thereto.

18 . A recombinant AAV virus comprising the AAV vector genome of claim 1 .

19 . The recombinant AAV virus of claim 18 , comprising a capsid serotype selected from the group consisting of AAV2, AAV1, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV9.47, AAV9(hu14), AAV10, AAV11, AAV12, AAVrh8, AAVrh10, AAV-DJ, and AAV-DJ8.

20 . The recombinant AAV virus of claim 19 , wherein the capsid serotype is AAV2.

21 . A pharmaceutical composition comprising the recombinant AAV virus of claim 20 .

22 . The pharmaceutical composition of claim 21 , wherein at least 70% of the recombinant AAV vectors contain an AAV vector genome.