C9orf72 and amyotrophic lateral sclerosis: In C9orf72 ALS/FTD, ADAR2 was found to be mislocalized in postmortem spinal cord tissue from C9orf72 patients, hiPSC MNs derived from C9orf72 patients, and in 6-month-old mice expressing the hexanucleotide repeat expansion via P0 intracerebroventricular (ICV) injection with AAV-(G4C2)149 [56].