BiophysicsAdvanced SciencePeer-reviewed
Allosteric Modulation of Pathological Ataxin-3 Aggregation: A Path to Spinocerebellar Ataxia Type-3 Therapies
Alexandra Silva, Sara Duarte‐Silva, Pedro Miguel Martins…·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.
DOI 10.1002/advs.202502216