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

The goal of this study is to identify transcriptomic changes in AP4B1-WT vs. AP4B1-KO SH-SY5Y cells following treatment with compound C-01. This lead compound was identified in a cell based high-throughput screen by Saffari et al. The title of the study is: "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"