Neuromuscular dysfunction in patient-derived FUSR244RR-ALS iPSC model via axonal downregulation of neuromuscular junction proteins
Nicolai von Kügelgen, Katarzyna Anna Ludwik, Samantha Mendonsa…·Marina Chekulaeva
The paper establishes an ALS hiPSC model showing axonal downregulation of neuromuscular junction proteins and identifies FUS binding to their mRNAs.