Sang Ryong Kim

1 Introduction Cerebellar ataxia (CA) comprises a clinically and genetically heterogeneous group of neurological disorders unified by progressive cerebellar degeneration. Cardinal features—gait instability, appendicular dysmetria, dysarthria, and oculomotor abnormalities—arise from Purkinje cell loss and disruption of cerebellar circuitry regardless of the underlying cause [1, 2].

Keywords spinocerebellar ataxia gene therapy adeno-associated viral vectors RNA-targeting therapeutics genome editing Emerging importance of gene therapy in spinocerebellar ataxias A key development in spinocerebellar ataxias (SCAs) (see Glossary) is the shift to mechanistic characterization and therapeutic platforms matched to defined mutational classes.

In a recent publication in Cell, Liebana and colleagues demonstrate that dopamine acts as a circuit-specific teaching signal, shaping individual learning trajectories over time.