Mutant prominin 1 found in patients with macular degeneration disrupts photoreceptor disk morphogenesis in mice

Research output: Contribution to journalResearch articleContributedpeer-review

Contributors

  • Z. Yang - (Author)
  • Y. Chen - (Author)
  • C. Lillo - (Author)
  • J. Chien - (Author)
  • Z. Yu - (Author)
  • M. Michaelides - (Author)
  • M. Klein - (Author)
  • K.A. Howes - (Author)
  • Y. Li - (Author)
  • Y. Kaminoh - (Author)
  • H. Chen - (Author)
  • C. Zhao - (Author)
  • Y. Chen - (Author)
  • Y.T. Al-Sheikh - (Author)
  • G. Karan - (Author)
  • D. Corbeil - , Research Group Tissue Engineering, Tissue Engineering (Research Group), Biotechnology Center (Author)
  • P. Escher - (Author)
  • S. Kamaya - (Author)
  • C. Li - (Author)
  • S. Johnson - (Author)
  • J.M. Frederick - (Author)
  • Y. Zhao - (Author)
  • C. Wang - (Author)
  • D.J. Cameron - (Author)
  • W.B. Huttner - (Author)
  • D.F. Schorderet - (Author)
  • F.L. Munier - (Author)
  • A.T. Moore - (Author)
  • D.G. Birch - (Author)
  • W. Baehr - (Author)
  • D.M. Hunt - (Author)
  • D.S. Williams - (Author)
  • K. Zhang - (Author)

Abstract

Familial macular degeneration is a clinically and genetically heterogeneous group of disorders characterized by progressive central vision loss. Here we show that an R373C missense mutation in the prominin 1 gene (PROM1) causes 3 forms of autosomal-dominant macular degeneration. In transgenic mice expressing R373C mutant human PROM1, both mutant and endogenous PROM1 were found throughout the layers of the photoreceptors, rather than at the base of the photoreceptor outer segments, where PROM1 is normally localized. Moreover, the outer segment disk membranes were greatly overgrown and misoriented, indicating defective disk morphogenesis. Immunoprecipitation studies showed that PROM1 interacted with protocadherin 21 (PCDH21), a photoreceptor-specific cadherin, and with actin filaments, both of which play critical roles in disk membrane morphogenesis. Collectively, our results identify what we believe to be a novel complex involved in photoreceptor disk morphogenesis and indicate a possible role for PROM1 and PCDH21 in macular degeneration.

Details

Original languageEnglish
Pages (from-to)2908-2916
Number of pages9
JournalThe Journal of clinical investigation
Volume118
Issue number8
Publication statusPublished - Aug 2008
Peer-reviewedYes

External IDs

PubMed 18654668
PubMed PMC2483685
Scopus 48749110402
ORCID /0000-0003-1181-3659/work/142252250

Keywords