Resolving the fibrotic niche of human liver cirrhosis at single-cell level

Nature. 2019 Nov;575(7783):512-518. doi: 10.1038/s41586-019-1631-3. Epub 2019 Oct 9.

Abstract

Liver cirrhosis is a major cause of death worldwide and is characterized by extensive fibrosis. There are currently no effective antifibrotic therapies available. To obtain a better understanding of the cellular and molecular mechanisms involved in disease pathogenesis and enable the discovery of therapeutic targets, here we profile the transcriptomes of more than 100,000 single human cells, yielding molecular definitions for non-parenchymal cell types that are found in healthy and cirrhotic human liver. We identify a scar-associated TREM2+CD9+ subpopulation of macrophages, which expands in liver fibrosis, differentiates from circulating monocytes and is pro-fibrogenic. We also define ACKR1+ and PLVAP+ endothelial cells that expand in cirrhosis, are topographically restricted to the fibrotic niche and enhance the transmigration of leucocytes. Multi-lineage modelling of ligand and receptor interactions between the scar-associated macrophages, endothelial cells and PDGFRα+ collagen-producing mesenchymal cells reveals intra-scar activity of several pro-fibrogenic pathways including TNFRSF12A, PDGFR and NOTCH signalling. Our work dissects unanticipated aspects of the cellular and molecular basis of human organ fibrosis at a single-cell level, and provides a conceptual framework for the discovery of rational therapeutic targets in liver cirrhosis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Case-Control Studies
  • Cell Lineage
  • Duffy Blood-Group System / metabolism
  • Endothelial Cells / metabolism
  • Endothelial Cells / pathology*
  • Female
  • Hepatic Stellate Cells / cytology
  • Hepatic Stellate Cells / metabolism
  • Hepatic Stellate Cells / pathology
  • Hepatocytes / cytology
  • Hepatocytes / metabolism
  • Hepatocytes / pathology
  • Humans
  • Liver / cytology
  • Liver / pathology*
  • Liver Cirrhosis / genetics
  • Liver Cirrhosis / pathology*
  • Macrophages / metabolism
  • Macrophages / pathology*
  • Male
  • Membrane Glycoproteins / metabolism
  • Membrane Proteins / metabolism
  • Mice
  • Phenotype
  • Receptor, Platelet-Derived Growth Factor alpha / metabolism
  • Receptors, Cell Surface / metabolism
  • Receptors, Immunologic / metabolism
  • Single-Cell Analysis*
  • Tetraspanin 29 / metabolism
  • Transcriptome
  • Transendothelial and Transepithelial Migration

Substances

  • ACKR1 protein, human
  • Duffy Blood-Group System
  • Membrane Glycoproteins
  • Membrane Proteins
  • PLVAP protein, human
  • Receptors, Cell Surface
  • Receptors, Immunologic
  • TREM2 protein, human
  • Tetraspanin 29
  • Receptor, Platelet-Derived Growth Factor alpha