Article

RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice

2024-06-25

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative condition, with 20% of familial and 2-3% of sporadic cases linked to mutations in the cytosolic superoxide dismutase (SOD1) gene. Mutant SOD1 protein is toxic to motor neurons, making SOD1 gene lowering a promising approach, supported by preclinical data and the 2023 FDA approval of the GapmeR ASO targeting SOD1, tofersen. Despite the approval of an...

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Literature Corpus work: 762dfa0d-8a6c-5059-becf-25d58a842544
DOI: 10.1101/2024.06.20.599943

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