Spinocerebellar ataxia (SCA) is a group of progressively aggravated neurodegenerative disease with autosomal dominant inheritance. Cerebellar ataxia is the core symptom, which may be accompanied by pyramidal tract signs, extrapyramidal signs, cognitive dysfunction and peripheral neuropathy. Although SCA can be accurately diagnosed by genetic testing, treatment is still difficult. We review the pathogenesis and therapeutic targets of SCA in recent years, in terms of genetic or gene regulation abnormalities, disruption of protein quality control (PQC) network, disruption of energy homeostasis, stability maintenance of PQC system, maintenance of cerebellar Purkinje cell function, and regulation of neuroinflammation, so as to promote the transformation of preclinical research into human therapy.

Spinocerebellar ataxia; Autophagy; Gene silencing; Neuroinflammation; Purkinje cell