Nonalcoholic fatty liver disease (
NAFLD), especially in its inflammatory form (
steatohepatitis, NASH), is closely related to the pathogenesis of chronic
liver disease. Despite substantial advances in the management of
NAFLD/NASH in recent years, there are currently no efficacious
therapies for its treatment. The biogenesis and expansion of lipid droplets (LDs) are critical pathophysiological processes in the development of
NAFLD/NASH. In the past decade, increasing evidence has demonstrated that
lipid droplet-associated proteins may represent potential therapeutic targets for the treatment of
NAFLD/NASH given the critical role they play in regulating the biogenesis and metabolism of lipid droplets. Recently, HSD17B13, a newly identified liver-enriched, hepatocyte-specific, lipid droplet-associated
protein, has been reported to be strongly associated with the development and progression of
NAFLD/NASH in both mice and humans. Notably, human genetic studies have repeatedly reported a robust association of HSD17B13 single nucleotide polymorphisms (SNPs) with the occurrence and severity of
NAFLD/NASH and other chronic
liver diseases (
CLDs). Here we briefly overview the discovery, tissue distribution, and subcellular localization of HSD17B13 and highlight its important role in promoting the pathogenesis of
NAFLD/NASH in both experimental animal models and patients. We also discuss the potential of HSD17B13 as a promising target for the development of novel therapeutic agents for
NAFLD/NASH.