Article

Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice

2025-01-29

Abstract excerpt

<h4>Background and aims</h4> Severe gastrointestinal (GI) symptoms occur in people with CLN3 disease, a neurodegenerative disorder. If left untreated these GI symptoms compromise life quality and may contribute to death. We hypothesized GI symptoms in CLN3 disease are at least partially due to neurodegeneration in the enteric nervous system (ENS), the master regulator of bowel function. <h4>Methods</h4> We exami...

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Literature Corpus work: e2b12275-1e82-5bb4-aae3-70811e4b3aac
DOI: 10.1101/2025.01.29.635518

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