Commentary: Loss of FOCAD, Operating via the SKI Messenger RNA Surveillance Pathway, Causes a Pediatric Syndrome with Liver Cirrhosis [1]

Abstract

Liver disease is becoming a major health concern and is estimated to be the fifth most common cause of death worldwide [2]. A systematic review from the Global Burden of Disease Study identified 1.32 million deaths due to liver cirrhosis in 2017, accounting for more than two percent of the total global deaths [3]. Liver cirrhosis is usually diagnosed late in life; it is irreversible, staying chronic for the rest of the patient ́s life; and it can be life-threatening, as it may evolve into hepatocellular carcinoma or lead to liver failure. Despite extensive research, to date none of the tested compounds for treating this disorder have proven to be safe and effective in human patients. Current available therapies are generally palliative and focused on slowing down the disease, with liver transplant remaining as the gold standard strategy to treat patients with decompensated cirrhosis. Therefore, there is a patent need for the identification and validation of new targets to treat, reverse and, ultimately, cure this hepatic disorder.