Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that has no effective therapeutic means at present. ALS is characterized by the progressive paralysis of the limbs and muscles involved in speech, swallowing, and respiration due to the progressive degeneration of spontaneous motor neurons. In 2011, the repeated amplification of a six-nucleotide motif (GGGGCC) in the protein-coding gene C9ORF72 (chromosome 9 ORF 72) became the first identified causative mutation in ALS and front temporal dementia (FTD).55, 56 Non-coding transcripts have now also been identified at the C9ORF72 locus. The transcription of the C9ORF72 repeats is bidirectional, producing both sense and antisense RNAs,57 both of which are elevated in the brains of ALS patients, and these transcripts are localized predominantly in the nucleus.58 C9ORF72 antisense lncRNA can inhibit C9ORF72 mRNA, thereby inhibiting gene expression. However, even if the expression of a disease-related gene is corrected in fibroblasts derived from a patient, the disease still cannot be treated.58