CAG repeat-selective compounds reduce abundance of expanded CAG RNAs in patient cell and murine models of SCAs
Hannah K. Shorrock, Asmer Aliyeva, Jesus A. Frias, Victoria A. DeMeo, Claudia D. Lennon, Cristina C. DeMeo, Amy K. Mascorro, Sharon Shaughnessy, Hormoz Mazdiyasni, John D. Cleary, Kaalak Reddy, Sweta Vangaveti, Damian Seung-Ho Shin, J. Andrew Berglund
The paper identifies small molecules that reduce expanded CAG RNA abundance in SCA patient and mouse models, showing repeat-selective effects across multiple SCAs.
bioRxiv · Aug 17, 2024 · PreprintMolecular Biology