Article

Symptomatic treatment by a BBB-permeable AAV engineered to restore TDP-43 function slows motor neuron disease and prevents paralysis

2025-08-20

Abstract excerpt

<h4>ABSTRACT</h4> TAR DNA-binding protein 43kDa (TDP-43) dysfunction is an early pathogenic mechanism that underlies amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disorder that lacks disease modifying therapies. We previously developed a mouse model in which TDP-43 is selectively deleted from motor neurons ( ChAT-Cre;Tardbp f/f ) that mimics the early stages of ALS. Here, we demonstrate t...

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Identifiers and source

Literature Corpus work: 7c6d3fb0-eb66-57c7-bc3c-c1376bb4e496
DOI: 10.1101/2025.08.14.670400

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