Importantly, our system could be easily expanded to screen potential therapeutics for other RAN translation- and aggregate-related diseases, most of which are also late-onset, such as myotonic dystrophy, C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia, spinocerebellar ataxias or Huntington disease (Green et al., 2016; Cleary and Ranum, 2017; Zhang and Ashizawa, 2017). Here, RAN is linked to amyotrophic lateral sclerosis.