LRRC6 mutation causes primary ciliary dyskinesia with dynein arm defects

PLoS One. 2013;8(3):e59436. doi: 10.1371/journal.pone.0059436. Epub 2013 Mar 19.

Abstract

Despite recent progress in defining the ciliome, the genetic basis for many cases of primary ciliary dyskinesia (PCD) remains elusive. We evaluated five children from two unrelated, consanguineous Palestinian families who had PCD with typical clinical features, reduced nasal nitric oxide concentrations, and absent dynein arms. Linkage analyses revealed a single common homozygous region on chromosome 8 and one candidate was conserved in organisms with motile cilia. Sequencing revealed a single novel mutation in LRRC6 (Leucine-rich repeat containing protein 6) that fit the model of autosomal recessive genetic transmission, leading to a change of a highly conserved amino acid from aspartic acid to histidine (Asp146His). LRRC6 was localized to the cytoplasm and was up-regulated during ciliogenesis in human airway epithelial cells in a Foxj1-dependent fashion. Nasal epithelial cells isolated from affected individuals and shRNA-mediated silencing in human airway epithelial cells, showed reduced LRRC6 expression, absent dynein arms, and slowed cilia beat frequency. Dynein arm proteins were either absent or mislocalized to the cytoplasm in airway epithelial cells from a primary ciliary dyskinesia subject. These findings suggest that LRRC6 plays a role in dynein arm assembly or trafficking and when mutated leads to primary ciliary dyskinesia with laterality defects.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Amino Acid Sequence
  • Analysis of Variance
  • Arabs / genetics
  • Base Sequence
  • Chromosomes, Human, Pair 8 / genetics*
  • Cytoskeletal Proteins
  • Dyneins / genetics
  • Dyneins / metabolism*
  • Female
  • Gene Expression Regulation, Developmental / physiology*
  • Gene Silencing
  • Genes, Recessive
  • Genetic Linkage
  • HEK293 Cells
  • Humans
  • Kartagener Syndrome / genetics*
  • Kartagener Syndrome / pathology
  • Male
  • Microscopy, Electron
  • Microscopy, Video
  • Molecular Sequence Data
  • Nasal Mucosa / cytology
  • Nasal Mucosa / metabolism
  • Oligonucleotides / genetics
  • Pedigree
  • Proteins / genetics*
  • RNA, Small Interfering / genetics
  • Reverse Transcriptase Polymerase Chain Reaction
  • Sequence Analysis, DNA

Substances

  • Cytoskeletal Proteins
  • LRRC6 protein, human
  • Oligonucleotides
  • Proteins
  • RNA, Small Interfering
  • Dyneins