Loss of the BMP antagonist USAG-1 ameliorates disease in a mouse model of the progressive hereditary kidney disease Alport syndrome

J Clin Invest. 2010 Mar;120(3):768-77. doi: 10.1172/JCI39569. Epub 2010 Feb 8.

Abstract

The glomerular basement membrane (GBM) is a key component of the filtering unit in the kidney. Mutations involving any of the collagen IV genes (COL4A3, COL4A4, and COL4A5) affect GBM assembly and cause Alport syndrome, a progressive hereditary kidney disease with no definitive therapy. Previously, we have demonstrated that the bone morphogenetic protein (BMP) antagonist uterine sensitization-associated gene-1 (USAG-1) negatively regulates the renoprotective action of BMP-7 in a mouse model of tubular injury during acute renal failure. Here, we investigated the role of USAG-1 in renal function in Col4a3-/- mice, which model Alport syndrome. Ablation of Usag1 in Col4a3-/- mice led to substantial attenuation of disease progression, normalization of GBM ultrastructure, preservation of renal function, and extension of life span. Immunohistochemical analysis revealed that USAG-1 and BMP-7 colocalized in the macula densa in the distal tubules, lying in direct contact with glomerular mesangial cells. Furthermore, in cultured mesangial cells, BMP-7 attenuated and USAG-1 enhanced the expression of MMP-12, a protease that may contribute to GBM degradation. These data suggest that the pathogenetic role of USAG-1 in Col4a3-/- mice might involve crosstalk between kidney tubules and the glomerulus and that inhibition of USAG-1 may be a promising therapeutic approach for the treatment of Alport syndrome.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Kidney Injury / genetics
  • Acute Kidney Injury / metabolism
  • Acute Kidney Injury / pathology
  • Adaptor Proteins, Signal Transducing
  • Animals
  • Bone Morphogenetic Protein 7 / antagonists & inhibitors*
  • Bone Morphogenetic Protein 7 / genetics
  • Bone Morphogenetic Protein 7 / metabolism
  • Bone Morphogenetic Proteins / genetics
  • Bone Morphogenetic Proteins / metabolism*
  • Collagen Type IV / genetics
  • Collagen Type IV / metabolism
  • Disease Models, Animal
  • Glomerular Basement Membrane / metabolism*
  • Glomerular Basement Membrane / pathology
  • Glomerular Mesangium / metabolism*
  • Glomerular Mesangium / pathology
  • Humans
  • Kidney Tubules / metabolism*
  • Kidney Tubules / pathology
  • Mice
  • Mice, Knockout
  • Mutation
  • Nephritis, Hereditary / genetics
  • Nephritis, Hereditary / metabolism*
  • Nephritis, Hereditary / pathology

Substances

  • Adaptor Proteins, Signal Transducing
  • Bone Morphogenetic Protein 7
  • Bone Morphogenetic Proteins
  • Collagen Type IV
  • Sostdc1 protein, mouse