High-Content Screening Identifies A Small Molecule 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 BCH-HSP-C01. This lead compound was identified in a phenotypic cell based high-throughput screen by Saffari et al. The title of the study is: "High-Content Screening Identifies A Small Molecule That Restores AP-4-dependent Protein Trafficking In Neuronal Models Of AP-4-Associated Hereditary Spastic Paraplegia