Repeat associated non-AUG translation as a common mechanism for the polyGln ataxias
Mónica Báñez-Coronel, Tao Zu, Madeline Aldridge, Shu Guo, Ramadan Ajredini, Deborah Morrison, Alexis B. Tays, Lisa A. Duvick, Olga Pletniková, Anthony T. Yachnis, Juan Carlos Troncoso, Henry Lauris Paulson, Hayley S. McLoughlin, Tetsuo Ashizawa, S. H. Subramony, , Laura P.W. Ranum
The paper identifies sense and antisense RAN proteins as a common mechanism in CAG-SCAs, showing their accumulation and toxicity in brain regions and demonstrating metformin's ability to reduce RAN protein levels.
Cell Reports · Dec 19, 2025Peer-reviewedNeuroscience