ASOs are an effective treatment for disease-associated oligodendrocyte signatures in premanifest and symptomatic SCA3 mice
Kristen H. Schuster, Annie J. Zalon, Danielle M. DiFranco, Alexandra F. Putka, Nicholas R. Stec, Sabrina I. Jarrah, Arsal Naeem, Zaid Haque, Hanrui Zhang, Yuanfang Guan, Hayley S. McLoughlin
This study shows that ASO treatment rescues oligodendrocyte dysfunction in SCA3 mice by targeting mutant ATXN3, suggesting potential for glial-targeted therapies in neurodegenerative diseases.
Molecular Therapy · Feb 29, 2024Peer-reviewedNeuroscience