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

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Z. Yang - (Autor:in)
  • Y. Chen - (Autor:in)
  • C. Lillo - (Autor:in)
  • J. Chien - (Autor:in)
  • Z. Yu - (Autor:in)
  • M. Michaelides - (Autor:in)
  • M. Klein - (Autor:in)
  • K.A. Howes - (Autor:in)
  • Y. Li - (Autor:in)
  • Y. Kaminoh - (Autor:in)
  • H. Chen - (Autor:in)
  • C. Zhao - (Autor:in)
  • Y. Chen - (Autor:in)
  • Y.T. Al-Sheikh - (Autor:in)
  • G. Karan - (Autor:in)
  • D. Corbeil - , Forschungsgruppe Tissue Engineering, Gewebe Engineering (FoG), Biotechnologisches Zentrum (BIOTEC) (Autor:in)
  • P. Escher - (Autor:in)
  • S. Kamaya - (Autor:in)
  • C. Li - (Autor:in)
  • S. Johnson - (Autor:in)
  • J.M. Frederick - (Autor:in)
  • Y. Zhao - (Autor:in)
  • C. Wang - (Autor:in)
  • D.J. Cameron - (Autor:in)
  • W.B. Huttner - (Autor:in)
  • D.F. Schorderet - (Autor:in)
  • F.L. Munier - (Autor:in)
  • A.T. Moore - (Autor:in)
  • D.G. Birch - (Autor:in)
  • W. Baehr - (Autor:in)
  • D.M. Hunt - (Autor:in)
  • D.S. Williams - (Autor:in)
  • K. Zhang - (Autor:in)

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

OriginalspracheEnglisch
Seiten (von - bis)2908-2916
Seitenumfang9
FachzeitschriftThe Journal of clinical investigation
Jahrgang118
Ausgabenummer8
PublikationsstatusVeröffentlicht - Aug. 2008
Peer-Review-StatusJa

Externe IDs

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

Schlagworte