MicroRNA-based therapeutics in experimental diseased animal models and human patients. (a) MiRNA-based therapeutic applications in animal models of human diseases. In animal models, various miRNA-based therapeutics possess promising treatment potential against different human diseases, including cardiovascular diseases, obesity-associated disorders, atherosclerosis, cancers, liver diseases, neurodegenerative diseases and central nervous system (CNS) injuries. (b) MiRNA-based therapeutic applications in patients. To our knowledge, five miRNA-based drugs have been tested in clinical trials. Miravirsen and RG-101 are miR-122 inhibitors and are designed for the treatment of HCV-induced liver disease. RG-125/AZD4076 is miR-103/107 inhibitor and is designed for the treatment of Type 2 Diabetes and non-alcoholic fatty liver diseases. MRX34 is miR-34 mimic and is designed for the treatment of advanced hepatocellular carcinoma. TargomiRs is miR-16 mimic and is designed for the treatment of recurrent thoracic cancer. “↑”, upregulate miRNA levels by various miRNA mimics; “↓”, downregulate miRNA levels by various miRNA inhibitors.