CPC C12N 15/1137 (2013.01) [A61P 25/28 (2018.01); C12N 2310/11 (2013.01); C12N 2310/32 (2013.01); C12N 2310/3515 (2013.01)] | 30 Claims |
1. An RNAi agent for inhibiting expression of a Superoxide Dismutase 1 (SOD1) gene, comprising:
an antisense strand comprising the nucleotide sequence cPrpusGfsaGfaucacagAfaUfcUfucasasc (SEQ ID NO: 646); and
a sense strand comprising the nucleotide sequence guugaagaUfuCfuGfugaucuca (SEQ ID NO: 771)
wherein a, c, g, and u represent 2′-O-methyl adenosine, 2′-O-methyl cytidine, 2′-O-methyl guanosine, and 2′-O-methyl uridine, respectively; Af, Cf, Gf, and Uf represent 2′-fluoro adenosine, 2′-fluoro cytidine, 2′-fluoro guanosine, and 2′-fluoro uridine, respectively; cPrpu represents a 5′-cyclopropyl phosphonate-2′-O-methyl uridine, and s represents a phosphorothioate linkage.
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