ATXN3 and Machado-Joseph disease: In a study that employed CRISPR/Cas9 genome-editing therapy of SCA3, the primary objective of suppressing mATXN3 was effectively accomplished by eliminating the mutated CAG repeats in the ATXN3 gene through the CRISPR/Cas9 system, specifically by knocking out the mutant CAG expansions in exon 10.