PUBLICATION

ccdc141 is required for left-right axis development by regulating cilia formation in the Kupffer's vesicle of zebrafish

Authors
Wang, P., Shi, W., Liu, S., Shi, Y., Jiang, X., Li, F., Chen, S., Sun, K., Xu, R.
ID
ZDB-PUB-240727-7
Date
2024
Source
Journal of genetics and genomics = Yi chuan xue bao   51(9): 934-946 (Journal)
Registered Authors
Keywords
WES, birth defects, cilia, left-right, zebrafish
MeSH Terms
  • Embryonic Development/genetics
  • Cilia*/genetics
  • Cilia*/metabolism
  • Cilia*/pathology
  • Animals
  • Humans
  • Signal Transduction/genetics
  • Embryo, Nonmammalian
  • Tubulin/genetics
  • Tubulin/metabolism
  • Body Patterning*/genetics
  • Zebrafish*/genetics
  • Mutation/genetics
  • Gene Expression Regulation, Developmental/genetics
  • Zebrafish Proteins*/genetics
  • Zebrafish Proteins*/metabolism
PubMed
39047937 Full text @ J. Genet. Genomics
Abstract
Laterality is a crucial physiological process intricately linked to the cilium-centrosome complex during embryo development. Defects in the process can result in severe organ mispositioning. Coiled-Coil Domain Containing 141 (CCDC141) has been previously known as a centrosome-related gene, but its role in left-right (LR) asymmetry has not been characterized. In this study, we utilize the zebrafish model and human exome analysis to elucidate the function of CCDC141 in laterality defects. The knockdown of ccdc141 in zebrafish disrupts early LR signaling pathways, cilia function, and Kupffer's vesicle (KV) formation. Unlike ccdc141-knockdown embryos exhibiting aberrant LR patterns, ccdc141-null mutants show no apparent abnormality, suggesting a genetic compensation response effect. In parallel, we observe a marked reduction in α-tubulin acetylation levels in the ccdc141 crispants. The treatment with histone deacetylase (HDAC) inhibitors, particularly the HDAC6 inhibitor, rescues the ccdc141 crispant phenotypes. Furthermore, exome analysis of 70 patients with laterality defects reveals an increased burden of CCDC141 mutations, with in-vivo studies verifying the pathogenicity of the patient mutation CCDC141-R123G. Our findings highlight the critical role of CCDC141 in ciliogenesis and demonstrate that CCDC141 mutations lead to abnormal LR patterns. Thus, we identify CCDC141 as a causative gene for lateral defects.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping