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October 18, 2025Open Access

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

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Pre-clinical evaluation reveals significant survival improvement and reduced inflammation in SOD1-ALS mice.

Mean survival extended by up to 27% in SOD1G93A mice treated with gene therapy.
The study found a dose-dependent reduction in mutant hSOD1 levels in brain and peripheral tissues.
Inflammation and microglial activation were observed, particularly at higher doses of treatment.
No major off-target effects were noted in human cell models, supporting the safety of this gene therapy approach.

Pezet et al. (2025) studied this question.

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