Article

Local administration of a novel siRNA modality into the CNS extends survival and improves motor function in the SOD1 <sup>G93A</sup> mouse model for ALS

2023-02-28

Abstract excerpt

<h4>ABSTRACT</h4> Antisense oligonucleotides (ASOs) were the first modality to pioneer targeted gene knockdown in the treatment of ALS caused by mutant superoxide dismutase 1 (SOD1). RNA interference (RNAi) is another mechanism of gene silencing with historically superior potency in which short interfering RNAs (siRNAs) guide the RNA-induced silencing complex (RISC) to cleave complementary transcripts. However, d...

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Literature Corpus work: 1b16b881-db83-57eb-a7a6-58ea115687fb
DOI: 10.1101/2023.02.27.530262

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