FUS-mutation carrying amyotrophic lateral sclerosis patient-derived motoneurons display lower survival, accumulate more DNA damage, and show elevated integrated stress response
Christine Roemer, Katarzyna Ludwik, Nicolai von Kuegelgen, David Peran Hayes, Samantha Mendosa, Marina Chekulaeva · FENS Forum 2024
Amyotrophic lateral sclerosis (ALS) is an incurable disease of the motoric nervous system characterized by loss of function and degeneration of upper and lower motoneurons. Models resembling clinics are required to study pathomechanisms and validate therapeutic options in ALS. Here, we reprogrammed fibroblasts from ALS patient carrying a mutation in Fused in Sarcoma (FUS) gene into induced pluripotent stem cells (iPSCs). An isogenic iPSC line was generated to serve as a control. Doxycycline-inducible NIL-cassette was introduced for motoneuron differentiation. Motoneurons were analyzed for
ePoster · Neuroscience