Neuromuscular dysfunction in patient-derived FUSR244RR-ALS iPSC model via axonal downregulation of neuromuscular junction proteins
Nicolai von Kügelgen, Katarzyna Anna Ludwik, Samantha Mendonsa, Christine Römer, Erik Becher, Laura Breimann, Mara Strauch, Tommaso Mari, Sandrine Mongellaz, Binyamin Zuckerman, Fatima Efendic, Nina Grexa, Anna Oliveras, Andrew Woehler, Matthias Selbach, , Marina Chekulaeva
The paper establishes an ALS hiPSC model showing axonal downregulation of neuromuscular junction proteins and identifies FUS binding to their mRNAs.
NAR Molecular Medicine · Mar 11, 2025Peer-reviewedCell Biology