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This research aims to evaluate the efficacy of an AAV9-based gene silencing vector in suppressing SOD1 and extending survival in an ALS mouse model.

June 27, 2026Nature CommunicationsOpen Access

Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model

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Randomized trial demonstrates improved survival and muscle function in ALS mice using AAV9 gene therapy, indicating potential for human treatment.

This research aims to evaluate the efficacy of an AAV9-based gene silencing vector in suppressing SOD1 and extending survival in an ALS mouse model.
AAV9 delivered artificial microRNAs targeting SOD1 in SOD1 G93A ALS mouse model.
Single intravenous injection led to sustained suppression of SOD1.
Efficacy assessed pre-symptomatically and during symptomatic stages.
Achieved significant improvement in respiratory function and motor performance.
Preserved α-motor neurons and maintained neuromuscular junctions.
Survival benefit unprecedented compared to previous AAV-based interventions.

Wan et al. (2026) studied this question.

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Randomized trial demonstrates improved survival and muscle function in ALS mice using AAV9 gene therapy, indicating potential for human treatment.