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

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)...

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Detalles Bibliográficos
Autores principales: Saffari, Afshin, Brechmann, Barbara, Boeger, Cedric, Saber, Wardiya Afshar, jumo, Hellen, Whye, Dosh, Wood, Delaney, Wahlster, Lara, Alecu, Julian, Ziegler, Marvin, Scheffold, Marlene, Winden, Kellen, Hubbs, Jed, Buttermore, Elizabeth, Barrett, Lee, Borner, Georg, Davies, Alexandra, Sahin, Mustafa, Ebrahimi-Fakhari, Darius
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Journal Experts 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10312991/
https://www.ncbi.nlm.nih.gov/pubmed/37398196
http://dx.doi.org/10.21203/rs.3.rs-3036166/v1

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