Disease mechanisms in late-onset retinal macular degeneration associated with mutation in C1QTNF5

Hum Mol Genet. 2006 May 15;15(10):1680-9. doi: 10.1093/hmg/ddl091. Epub 2006 Apr 6.

Abstract

Late-onset retinal macular degeneration (L-ORMD) is an autosomal dominant condition resembling age-related macular degeneration (AMD) in which a key pathological feature is a thick extracellular sub-retinal pigment epithelial (RPE) deposit. L-ORMD is caused by mutation in the C1QTNF5 (CTRP5) short-chain collagen gene, but the disease mechanism is unknown. Here, we first show that wild-type C1QTNF5 is secreted, whereas mutant C1QTNF5 is misfolded and retained within the endoplasmic reticulum (ER). Secondly, the ER retained mutant protein has a shorter half-life than wild-type C1QTNF5 and is preferentially degraded by proteasomes. Thirdly, C1QTNF5 is shown to interact with the membrane-type frizzled related protein (MFRP), on the basis of yeast two-hybrid, protein pull-down and co-immunoprecipitation assays and RPE co-localization. These data suggest that L-ORMD is due to insufficient levels of secreted C1QTNF5, compromised RPE cell function resulting from ER retention of the mutant protein or both mechanisms.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Age of Onset
  • Animals
  • Cell Adhesion / physiology
  • Cell Line
  • Collagen / genetics
  • Collagen / physiology*
  • Endoplasmic Reticulum / metabolism
  • Eye Proteins / genetics
  • Eye Proteins / metabolism
  • Humans
  • Macular Degeneration / genetics
  • Macular Degeneration / metabolism*
  • Macular Degeneration / pathology
  • Membrane Proteins / genetics
  • Membrane Proteins / metabolism
  • Mice
  • Mutation
  • Pigment Epithelium of Eye / pathology
  • Proteasome Endopeptidase Complex / physiology
  • Protein Binding
  • Protein Folding

Substances

  • C1QTNF5 protein, human
  • Eye Proteins
  • Membrane Proteins
  • Mfrp protein, mouse
  • Collagen
  • Proteasome Endopeptidase Complex