Article

A novel porcine model of CLN3 Batten disease recapitulates clinical phenotypes

2022-10-07

Abstract excerpt

Mouse models of CLN3 Batten disease, a rare lysosomal storage disorder with no cure, have improved our understanding of CLN3 biology and therapeutics through their ease of use and a consistent display of cellular pathology. However, the translatability of murine models is limited by disparities in anatomy, body size, life span, and inconsistent, subtle behavior deficits that can be difficult to detect in CLN3 muta...

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Literature Corpus work: 89d1f168-2596-56a7-80c7-6ad4b33689a7
DOI: 10.1101/2022.10.07.511360

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