DYX1C1 is required for axonemal dynein assembly and ciliary motility. Author Aarti Tarkar, Niki Loges, Christopher Slagle, Richard Francis, Gerard Dougherty, Joel Tamayo V, Brett Shook, Marie Cantino, Daniel Schwartz, Charlotte Jahnke, Heike Olbrich, Claudius Werner, Johanna Raidt, Petra Pennekamp, Marouan Abouhamed, Rim Hjeij, Gabriele Köhler, Matthias Griese, You Li, Kristi Lemke, Nikolas Klena, Xiaoqin Liu, George Gabriel, Kimimasa Tobita, Martine Jaspers, Lucy Morgan, Adam Shapiro, Stef Letteboer, Dorus Mans, Johnny Carson, Margaret Leigh, Whitney Wolf, Serafine Chen, Jane Lucas, Alexandros Onoufriadis, Vincent Plagnol, Miriam Schmidts, Karsten Boldt, Ronald Roepman, Maimoona Zariwala, Cecilia Lo, Hannah Mitchison, Michael Knowles, Rebecca Burdine, Joseph Loturco, Heymut Omran Publication Year 2013 Type Journal Article 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). Journal Nature genetics Volume 45 Issue 9 Pages 995-1003 Date Published 09/2013 ISSN Number 1546-1718 DOI 10.1038/ng.2707 Alternate Journal Nat Genet PMCID PMC4000444 PMID 23872636 PubMedPubMed CentralGoogle ScholarBibTeXEndNote X3 XML