Studies have showed that the dysregulation of A-to-I RNA editing in transcriptome or abnormal expression of ADAR is associated with various diseases, including neurological diseases, immune disease, cancer, viral infections and etc. For example, A-to-I RNA editing in a subunit of glutamate receptor 2 (GluR2) is essential for the survival of motor neurons, hence the inhibitors targeting non-A-to-I RNA editing receptors may serve as an additional tool for the treatment of amyotrophic lateral sclerosis (ALS) [63]. The gene discussed is ADAR; the disease is amyotrophic lateral sclerosis.