Since loss-of- function mutation of Ttbk2 leads to spinocerebellar ataxia 11 [26] and neuron-specific deletion of Ttbk2 induces loss of Purkinje cells [36], development of TTBK1-specific inhibitor such as ASO-Ttbk1, is necessary to avoid unwanted off-target effect. The gene discussed is TTBK1; the disease is cerebellar ataxia.