Nowadays, hundreds of dysregulated splicing factors, including in Cacna1d (McKinney et al., 2009), Tanc2 (Han et al., 2010), Ndrg4 (Yamamoto et al., 2011), and GRIN1 (Shimizu et al., 2000) have been detected in the brain of Mbnl 2–/– mice, most of which were similarly dysregulated in DM1 patients, indicating a critical role of the Mbnl2 loss in DM1 brain pathology (Charizanis et al., 2012). The gene discussed is NDRG4; the disease is myotonic dystrophy type 1.