Allosteric Modulation of Pathological Ataxin-3 Aggregation: A Path to Spinocerebellar Ataxia Type-3 Therapies
Alexandra Silva, Sara Duarte‐Silva, Pedro Miguel Martins, Beatriz Rocha Ilidio Rodrigues, Débora Serrenho, Daniela Vilasboas‐Campos, Andreia Teixeira‐Castro, Julio Vieyto‐Nuñez, Joel Mieres‐Pérez, Francisco Figueiredo, Martyna Podlasiak, Tatiana Van‐der‐Kellen, Joana S. Fraga, James W. Noble, Carter Lantz, , Sandra Macedo‐Ribeiro
This study shows that CLR01, a supramolecular tweezer, reduces Atx3 aggregation by binding to a specific site, delaying fibril formation and improving symptoms in SCA3 models.
Advanced Science · Nov 26, 2025Peer-reviewedBiophysics