Article

Sortilin inhibition treats multiple neurodegenerative lysosomal storage disorders

2023-09-22

Abstract excerpt

Lysosomal storage disorders (LSDs) are a genetically and clinically diverse group of diseases characterized by lysosomal dysfunction. Batten disease is a family of severe LSDs primarily impacting the central nervous system. Here we show that AF38469, a small molecule inhibitor of sortilin, improves lysosomal and glial pathology across multiple LSD models. Live-cell imaging and comparative transcriptomics demonstra...

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Literature Corpus work: 9eb1453b-d3c0-5ead-af22-74d3ce49f5c8
DOI: 10.1101/2023.09.22.559064

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