CPC A61K 35/76 (2013.01) [A61P 25/14 (2018.01); C12N 7/00 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2310/141 (2013.01); C12N 2320/32 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14123 (2013.01); C12N 2750/14132 (2013.01); C12N 2750/14143 (2013.01)] | 21 Claims |
1. An adeno-associated viral (AAV) genome comprising a first inverted terminal repeat (ITR), a promoter operably linked to a nucleic acid comprising a modulatory polynucleotide encoding a sense strand sequence and an antisense strand sequence, and a second ITR; wherein the encoded antisense strand sequence comprises the nucleotide sequence of SEQ ID NO: 918 and the sense strand sequence comprises the nucleotide sequence of SEQ ID NO: 1079; and wherein the AAV viral genome comprises one or more of:
(i) a first ITR sequence comprising the nucleotide sequence of any one of SEQ ID NOs: 1380 or 1381-1383;
(ii) a promoter comprising a chicken β-actin (CBA) promoter, a cytomegalovirus (CMV) promoter, a phosphoglycerate kinase 1 (PGK) promoter, an H1 promoter, a T7 promoter, a ubiquitin c (UBC) promoter, a β-glucuronidase (GUSB) promoter, a neuron-specific enolase (NSE) promoter, a synapsin promoter, a methyl-CpG binding protein 2 (MeCP2) promoter, or a glial fibrillary acidic protein (GFAP) promoter;
(iii) a promoter comprising the nucleotide sequence of any one of SEQ ID NOs: 1410, 1411-1414; or
(iv) a second ITR sequence comprising the nucleotide sequence of any one of SEQ ID NOs: 1382, 1380, 1381, or 1383.
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