Excessive EFHD1-dependent ER-mitochondrial contacts drive a maladaptive antiviral response in metabolic liver disease

Metabolic-associated steatohepatitis (MASH) involves hepatocyte damage that cannot be explained solely by lipid accumulation. Here, to discover injury-specific pathways, we focused on a gene of uncertain function, EF-Hand Domain Family Member D1 (EFHD1), identified in human genome-wide association studies of liver injury but not liver fat. We show that EFHD1, a Ca2+-dependent actin crosslinker, stabilizes endoplasmic reticulum-mitochondria contact sites (ERMCS), detecting spatiotemporal coincidence of inter-organellar proximity and ER Ca2+ release. During MASH, EFHD1 upregulation drives pathological mitochondrial fragmentation via excessive contact persistence. This structural failure promotes mitochondrial double-stranded RNA escape and activation of a maladaptive antiviral PKR-associated stress response, a causal relationship also supported by Mendelian randomization in humans. Consequently, inhibiting EFHD1 in human and mouse models blunts hepatocyte damage. These findings identify EFHD1 as a Ca2+-dependent ERMCS stabilizer, reveal a hepatocyte-intrinsic injury pathway, and suggest EFHD1 inhibition as a therapeutic strategy.

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Publication Details

Journal
Journal of Clinical Investigation
Published
2026-08-25
DOI
https://doi.org/10.1172/jci204023
Primary Topic
Liver Disease Diagnosis and Treatment
Type
article
Field-Weighted Citation Impact
0.00

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article

Excessive EFHD1-dependent ER-mitochondrial contacts drive a maladaptive antiviral response in metabolic liver disease

Stavros Drakos, Enrique Balderas, Dipayan Chaudhuri, Scott A. Summers et al.
Journal of Clinical Investigation
Liver Disease Diagnosis and Treatment
article

Excessive EFHD1-dependent ER-mitochondrial contacts drive a maladaptive antiviral response in metabolic liver disease

Stavros Drakos, Enrique Balderas, Dipayan Chaudhuri, Scott A. Summers, Robin M. Shaw, Anthony M. Balynas, William L. Holland, Chris Stubben, Daisuke Shimura, Sudipa Maity, Tara R. Price, Francisco Verdeguer, Sandra Lee, Jared Rutter, Ademuyiwa S. Aromolaran, Joel Zvick, Sihem Boudina, Emma C. Rekate, Vishaka Vinod, Dung M. Nguyen, Hannah E. Duron, Vu D Nguyen, Ashley R. Bratt, Sarah Franklin, Devorah Stucki, Adrian M Velarde, Yasmin B Masini, Neeraj K Rai, Nicolas Hartel, Vivek Garg, David R. Eberhardt, David Mollinedo, Marcus G. Pezzolesi, Kimberley J. Evason, Kamrul H. Chowdhury, Xue Yin, Anshu Kumari, Patrice N. Mimche, Ryan Bia, Andrea Corbin
article en

Abstract

Metabolic-associated steatohepatitis (MASH) involves hepatocyte damage that cannot be explained solely by lipid accumulation. Here, to discover injury-specific pathways, we focused on a gene of uncertain function, EF-Hand Domain Family Member D1 (EFHD1), identified in human genome-wide association studies of liver injury but not liver fat. We show that EFHD1, a Ca2+-dependent actin crosslinker, stabilizes endoplasmic reticulum-mitochondria contact sites (ERMCS), detecting spatiotemporal coincidence of inter-organellar proximity and ER Ca2+ release. During MASH, EFHD1 upregulation drives pathological mitochondrial fragmentation via excessive contact persistence. This structural failure promotes mitochondrial double-stranded RNA escape and activation of a maladaptive antiviral PKR-associated stress response, a causal relationship also supported by Mendelian randomization in humans. Consequently, inhibiting EFHD1 in human and mouse models blunts hepatocyte damage. These findings identify EFHD1 as a Ca2+-dependent ERMCS stabilizer, reveal a hepatocyte-intrinsic injury pathway, and suggest EFHD1 inhibition as a therapeutic strategy.

Journal of Clinical Investigation
University of Maryland, Baltimore (US), Howard Hughes Medical Institute (US), University of Utah (US), Huntsman Cancer Institute (US), Thermo Fisher Scientific (United States) (US), Inspire Institute (US), Indiana University – Purdue University Indianapolis (US)
American Heart Association, Barth Syndrome Foundation, Nora Eccles Treadwell Foundation, National Heart, Lung, and Blood Institute, National Institute of General Medical Sciences, National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Arthritis and Musculoskeletal and Skin Diseases
Good health and well-being
Openalex Percentile: Top 10%
Liver Disease Diagnosis and Treatment
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