Leveraging Human Genetics to Identify Potential New Treatments for Fatty Liver Disease

Stefano Romeo, Arun Sanyal, Luca Valenti

Research output: Contribution to journalReview articlepeer-review

Abstract

Fatty liver disease (FLD), including its more severe pathologies, namely steatohepatitis, hepatocarcinoma, and cirrhosis, is the most common cause of chronic liver disease worldwide and is projected to become the leading cause of hepatocellular carcinoma and end-stage liver disease. FLD is heterogeneous with multiple etiologies and diverse histological phenotypes, so therapies will ultimately need to be individualized for relevant targets. Inherited factors contribute to FLD, and most of the genetic variation influencing liver disease development and progression is derived from genes involved in lipid biology, including PNPLA3, TM6SF2, GCKR, MBOAT7, and HSD17B13. From this point of view, we focus in this perspective on how human molecular genetics of FLD have highlighted defects in hepatic lipid handling as a major common mechanism of its pathology and how this insight could be leveraged to treat and prevent its more serious complications. Fatty liver is primarily a metabolic disease with a strong genetic susceptibility. Understanding human and molecular genetics of this disease will allow us to improve the design of clinical intervention studies and discover novel drug pathways.

Original languageEnglish
Pages (from-to)35-45
Number of pages11
JournalCell Metabolism
Volume31
Issue number1
DOIs
Publication statusPublished - Jan 7 2020

ASJC Scopus subject areas

  • Physiology
  • Molecular Biology
  • Cell Biology

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