Cauli: a mouse strain with an Ift140 mutation that results in a skeletal ciliopathy modelling Jeune syndrome

PLoS Genet. 2013 Aug;9(8):e1003746. doi: 10.1371/journal.pgen.1003746. Epub 2013 Aug 29.

Abstract

Cilia are architecturally complex organelles that protrude from the cell membrane and have signalling, sensory and motility functions that are central to normal tissue development and homeostasis. There are two broad categories of cilia; motile and non-motile, or primary, cilia. The central role of primary cilia in health and disease has become prominent in the past decade with the recognition of a number of human syndromes that result from defects in the formation or function of primary cilia. This rapidly growing class of conditions, now known as ciliopathies, impact the development of a diverse range of tissues including the neural axis, craniofacial structures, skeleton, kidneys, eyes and lungs. The broad impact of cilia dysfunction on development reflects the pivotal position of the primary cilia within a signalling nexus involving a growing number of growth factor systems including Hedgehog, Pdgf, Fgf, Hippo, Notch and both canonical Wnt and planar cell polarity. We have identified a novel ENU mutant allele of Ift140, which causes a mid-gestation embryonic lethal phenotype in homozygous mutant mice. Mutant embryos exhibit a range of phenotypes including exencephaly and spina bifida, craniofacial dysmorphism, digit anomalies, cardiac anomalies and somite patterning defects. A number of these phenotypes can be attributed to alterations in Hedgehog signalling, although additional signalling systems are also likely to be involved. We also report the identification of a homozygous recessive mutation in IFT140 in a Jeune syndrome patient. This ENU-induced Jeune syndrome model will be useful in delineating the origins of dysmorphology in human ciliopathies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Carrier Proteins / genetics*
  • Cell Communication / genetics*
  • Cell Polarity
  • Cilia / genetics
  • Cilia / pathology*
  • Disease Models, Animal
  • Ellis-Van Creveld Syndrome / genetics*
  • Ellis-Van Creveld Syndrome / pathology
  • Embryonic Development / genetics*
  • Hedgehog Proteins / genetics
  • Humans
  • Mice
  • Mutation
  • Signal Transduction

Substances

  • Carrier Proteins
  • Hedgehog Proteins
  • IFT140 protein, mouse

Supplementary concepts

  • Jeune syndrome

Grants and funding

This work was supported by the NHMRC grant #1002660 and the Victorian Government's Operational Infrastructure Support (OIS) Program. This work was enabled by the Australian Phenomics Network and part supported by funding from the Australian Government's National Collaborative Research Infrastructure Strategy. TYT is funded by an Early Career Fellowship 607431 and JFB is supported by a Senior Principle Research Fellowship, both from the National Health and Medical Research Council, Australia. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.