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

• Published in: Journal of biomolecular screening Vol. 20; no. 9; pp. 1101 - 1111
• Main Authors: Kaufmann, Markus, Schuffenhauer, Ansgar, Fruh, Isabelle, Klein, Jessica, Thiemeyer, Anke, Rigo, Pierre, Gomez-Mancilla, Baltazar, Heidinger-Millot, Valerie, Bouwmeester, Tewis, Schopfer, Ulrich, Mueller, Matthias, Fodor, Barna D., Cobos-Correa, Amanda
• Format: Journal Article
• Language: English
• Published: United States 01.10.2015
• Subjects: Cells, Cultured; Drug Evaluation, Preclinical; Fragile X Mental Retardation Protein - genetics; Fragile X Mental Retardation Protein - metabolism; Fragile X Syndrome - drug therapy; Fragile X Syndrome - genetics; Gene Silencing - drug effects; High-Throughput Screening Assays; Humans; Induced Pluripotent Stem Cells - drug effects; Induced Pluripotent Stem Cells - physiology; Neural Stem Cells - drug effects; Neural Stem Cells - physiology; Trinucleotide Repeats; induced pluripotent stem cells; fragile X syndrome; high-content screening

Fragile X syndrome (FXS) is the most common form of inherited mental retardation, and it is caused in most of cases by epigenetic silencing of the Fmr1 gene. Today, no specific therapy exists for FXS, and current treatments are only directed to improve behavioral symptoms. Neuronal progenitors deriv...