August 12, 2025Brain

In vivo self-assembled SOD1-siRNAs mitigate muscle atrophy and denervation in amyotrophic lateral sclerosis

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The study demonstrates that self-assembled SOD1-siRNAs significantly reduce mutant SOD1 levels in the spinal cord.
Intravenous administration of a synthetic construct facilitated effective delivery of SOD1-siRNAs to spinal neurons.
Treatment with sEV-encapsulated SOD1-siRNAs resulted in marked reduction of ALS symptoms in Tg(SOD1G93A) transgenic mice.
AAV-based strategies offer a long-term solution, achieving sustained inhibition of SOD1 with a single injection.

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Synthetic biology strategy reduces SOD1 levels and mitigates symptoms in transgenic mice, suggesting new ALS therapies.

Guo et al. (2025) studied this question.

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