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This is a Experimental Study study.

October 19, 2025Open Access

Preemptive SOD1 Silencing via Neonatal Intramuscular AAV Therapy Modifies Disease Trajectory in an ALS Mouse Model

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Gene therapy demonstrates delayed disease onset and preserved function in ALS mouse model, suggesting new treatment pathways.

Neonatal intramuscular therapy significantly delayed disease onset and prolonged survival in ALS mice, showcasing its potential efficacy.
A single injection of rAAV2-retro-miRNA led to widespread spinal motor neuron transduction and a notable reduction in mutant SOD1 protein levels.
This development employs innovative gene therapy using an artificial microRNA, improving upon traditional viral delivery systems and ensuring safety.
The findings are pivotal for establishing rAAV2-retro-miRNA therapy as a viable preemptive intervention for SOD1-related ALS and similar motor neuron diseases.

Gong et al. (2025) studied this question.

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