CPC C12N 15/113 (2013.01) [A61K 9/0019 (2013.01); A61K 9/0085 (2013.01); A61K 48/0075 (2013.01); A61P 25/02 (2018.01); A61P 25/16 (2018.01); A61P 25/28 (2018.01); C07K 14/005 (2013.01); C12N 7/00 (2013.01); C12N 9/2402 (2013.01); C12N 15/86 (2013.01); C12Y 302/01045 (2013.01); A61K 48/00 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2710/14043 (2013.01); C12N 2750/14133 (2013.01); C12N 2750/14143 (2013.01); C12N 2770/32022 (2013.01)] | 41 Claims |
1. An isolated nucleic acid comprising
(i) an expression construct comprising a transgene encoding a single gene product, wherein the gene product is an inhibitory nucleic acid targeting α-Synuclein, wherein the inhibitory nucleic acid is encoded by the sequence set forth in SEQ ID NO: 3; and
(ii) two adeno-associated virus (AAV) inverted terminal repeats (ITR) sequences flanking the expression construct, wherein the first ITR sequence is a 5′ ITR, and the second ITR sequence is a 3′ ITR.
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