DYX1C1 is required for axonemal dynein assembly and ciliary motility

Nat Genet. 2013 Sep;45(9):995-1003. doi: 10.1038/ng.2707. Epub 2013 Jul 21.

Abstract

DYX1C1 has been associated with dyslexia and neuronal migration in the developing neocortex. Unexpectedly, we found that deleting exons 2-4 of Dyx1c1 in mice caused a phenotype resembling primary ciliary dyskinesia (PCD), a disorder characterized by chronic airway disease, laterality defects and male infertility. This phenotype was confirmed independently in mice with a Dyx1c1 c.T2A start-codon mutation recovered from an N-ethyl-N-nitrosourea (ENU) mutagenesis screen. Morpholinos targeting dyx1c1 in zebrafish also caused laterality and ciliary motility defects. In humans, we identified recessive loss-of-function DYX1C1 mutations in 12 individuals with PCD. Ultrastructural and immunofluorescence analyses of DYX1C1-mutant motile cilia in mice and humans showed disruptions of outer and inner dynein arms (ODAs and IDAs, respectively). DYX1C1 localizes to the cytoplasm of respiratory epithelial cells, its interactome is enriched for molecular chaperones, and it interacts with the cytoplasmic ODA and IDA assembly factor DNAAF2 (KTU). Thus, we propose that DYX1C1 is a newly identified dynein axonemal assembly factor (DNAAF4).

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Axonemal Dyneins / genetics*
  • Axonemal Dyneins / metabolism*
  • Cilia / genetics*
  • Cilia / metabolism*
  • Cilia / ultrastructure
  • Disease Models, Animal
  • Ependyma / metabolism
  • Ependyma / pathology
  • Gene Knockdown Techniques
  • Gene Order
  • Gene Targeting
  • Humans
  • Intracellular Space / metabolism
  • Kartagener Syndrome / genetics
  • Kartagener Syndrome / metabolism
  • Male
  • Mice
  • Mice, Knockout
  • Mutation
  • Nerve Tissue Proteins / genetics*
  • Nerve Tissue Proteins / metabolism
  • Phenotype
  • Protein Binding
  • Protein Transport
  • Respiratory Mucosa / metabolism
  • Respiratory Mucosa / pathology
  • Zebrafish

Substances

  • DYX1C1 protein, mouse
  • Nerve Tissue Proteins
  • Axonemal Dyneins