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AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

25 Jun 2025

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) involves motor neuron death due to mislocalized TDP-43. Pathologic TDP-43 associates with stress granules (SGs), and lowering the SG-associated protein ataxin-2 (ATXN2) using Atxn2-targeting antisense oligonucleotides prolongs survival in TAR4/4 sporadic ALS mice but failed in clinical trials likely due to poor target engagement. Here we show that an AAV with potent motor...

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PMID: 40562771
DOI: 10.1038/s41467-025-60497-8

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