Drug screen in iPSC-Neurons identifies nucleoside analogs as inhibitors of (G4C2)n expression in C9orf72 ALS/FTD
Mareike Czuppa, Ashutosh Dhingra, Qihui Zhou, Carina Schludi, Laura König, Elisabeth Scharf, Daniel Farny, Anupriya Dalmia, Joachim Täger, Melissa Castillo-Lizardo, Eszter Katona, Kohji Mori, Tina Aumer, Florian Schelter, Markus Müller, Thomas Carell, Tuomo Kalliokoski, Josef Messinger, Patrizia Rizzu, Peter Heutink, Dieter Edbauer
Abstract
foci and DPRs in C9orf72 BAC transgenic mice. Our findings suggest that nucleoside analogs are a promising compound class for therapeutic development in C9orf72 repeat-expansion-associated disorders.
Topics & Concepts
C9orf72DrugInduced pluripotent stem cellNucleosideChemistryBiologyPharmacologyTrinucleotide repeat expansionBiochemistryGeneEmbryonic stem cellAlleleAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders ResearchAdenosine and Purinergic Signaling