THERAPEUTIC TARGETING OF METABOLIC microRNAs AS A NEW TREATMENT PARADIGM FOR NASH

NASH is a progressive subtype of nonalcoholic fatty liver disease characterized by abnormal fat accumulation in the liver, inflammation, and fibrosis. NASH is strongly associated with obesity and the metabolic syndrome and is rapidly becoming the leading cause of end-stage liver disease, liver transplantation, and hepatocellular carcinoma, underscoring the need for new therapies for treatment of NASH. This project focuses on developing miRNA-targeted therapeutics as a new treatment paradigm for NASH. Our goal is to discover drugs for effective and safe inhibition of metabolic miRNAs and assess their therapeutic potential to treat NASH in highly relevant preclinical models of NASH. Furthermore, we are evaluating hepatic and circulating miRNAs in human samples as biomarkers for NASH. We envision that the development of combined diagnostics and miRNA-targeted therapeutics has the potential to guide new treatment options for patients with NASH.