SCA3/MJD-iPSCs own the same mutant ATXN3 as parental somatic cells and have the potential to differentiate into neural stem cells (NSCs) and neuronal cells, which provides a good cell model for the study of pathological mechanisms and drug screening of SCA3 [35, 36]. Here, ATXN3 is linked to Spinocerebellar ataxia type 3.