High-Throughput Screening Using iPSC-Derived Neuronal Progenitors to Identify Compounds Counteracting Epigenetic Gene Silencing in Fragile X Syndrome

We describe an integrated drug discovery model comprising iPSC generation, culture scale-up, and quality control and screening with a very sensitive high-content imaging assay assisted by single-cell image analysis and multiparametric data analysis based on machine learning algorithms. The screening identified several compounds that induced a weak expression of fragile X mental retardation protein (FMRP) and thus sets the basis for further large-scale screens to find candidate drugs or targets tackling the underlying mechanism of FXS with potential for therapeutic intervention.

Source: Journal of Biomolecular Screening - September 18, 2015 Category: Molecular Biology Authors: Kaufmann, M., Schuffenhauer, A., Fruh, I., Klein, J., Thiemeyer, A., Rigo, P., Gomez-Mancilla, B., Heidinger-Millot, V., Bouwmeester, T., Schopfer, U., Mueller, M., Fodor, B. D., Cobos-Correa, A. Tags: Original Research Source Type: research