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Pre-clinical evaluation of a gene therapy candidate for <i>SOD1</i> -ALS shows improved survival and signs of inflammation in the CNS of treated mice

2025-10-16

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder characterized by motor neurons loss (MN). In 15–20% of familial ALS cases, mutations in the superoxide dismutase 1 ( SOD1 ) gene are the underlying cause. Targeting human SOD1 (hSOD1) toxicity has emerged as a promising approach to treat SOD1-ALS. We previously demonstrated the efficacy of an exon-skipping strategy using a self-comple...

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Literature Corpus work
222b8f95-64cb-58ff-8c45-7e5c8da69b20
DOI
10.1101/2025.10.16.682744
Open publication

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Pre-clinical evaluation of a gene therapy candidate for <i>SOD1</i> -ALS shows improved survival and signs of inflammation in the CNS of treated miceDOI 10.1101/2025.10.16.682744
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