Article

High-Content Small Molecule Screen Identifies a Novel Compound That Restores AP-4-Dependent Protein Trafficking in Neuronal Models of AP-4-Associated Hereditary Spastic Paraplegia

2023-06-12

Abstract excerpt

<title>Abstract</title> <p>Unbiased phenotypic screens in patient-relevant disease models offer the potential to detect novel therapeutic targets for rare diseases. In this study, we developed a high-throughput screening assay to identify molecules that correct aberrant protein trafficking in adaptor protein complex 4 (AP-4) deficiency, a rare but prototypical form of childhood-onset hereditary spastic paraplegia...

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Literature Corpus work: 7e566cc1-53f1-5a11-b679-83843ee89fda
DOI: 10.21203/rs.3.rs-3036166/v1

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High-Content Small Molecule Screen Identifies a Novel Compound That Restores AP-4-Dependent Protein Trafficking in Neuronal Models of AP-4-Associated Hereditary Spastic ParaplegiaDOI 10.21203/rs.3.rs-3036166/v1Read

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