CPC A61K 31/713 (2013.01) [A61K 9/0085 (2013.01); C12N 15/113 (2013.01); C12N 15/63 (2013.01); C12N 15/85 (2013.01); C12N 15/86 (2013.01); A61P 25/28 (2018.01); C12N 2310/11 (2013.01); C12N 2310/14 (2013.01); C12N 2320/30 (2013.01)] | 24 Claims |
1. A method for inhibiting the expression of the superoxide dismutase 1 (SOD1) gene in a cell in a subject comprising administering to the subject an effective amount of an adeno-associated viral (AAV) particle comprising a viral genome and a capsid, wherein said viral genome comprises a modulatory polynucleotide sequence positioned between two inverted terminal repeats (ITRs), wherein said modulatory polynucleotide sequence comprises a nucleic acid sequence encoding a sense strand sequence and an antisense strand sequence of a siRNA duplex, and wherein the sense strand sequence comprises at least 20 consecutive nucleotides from the nucleotide sequence of SEQ ID NO: 2, and the antisense strand sequence comprises at least 20 consecutive nucleotides from the nucleotide sequence of SEQ ID NO: 1, thereby inhibiting expression of the SOD1 gene in the cell in the subject.
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