Gene therapy for spinocerebellar ataxias
Spinocerebellar ataxias reframe neurodegeneration as a mutation-stratified therapeutic landscape.
The failure of conventional therapies reflects a mismatch with mutation driven pathogenesis.
Gene- and RNA-based therapies enable direct intervention at the level of the causative lesion.
Transcript-lowering approaches represent the leading clinically tractable strategy in dominant spinocerebellar ataxias.
Clinical success for gene therapy will require optimizing delivery, specificity, durability, and biomarker readiness.
https://www.cell.com/trends/pharmacological-sciences/fulltext/S0165-6147(26)00162-8
