PUBLICATION

Mutations in TRAF3IP1/IFT54 reveal a new role for IFT proteins in microtubule stabilization

Authors
Bizet, A.A., Becker-Heck, A., Ryan, R., Weber, K., Filhol, E., Krug, P., Halbritter, J., Delous, M., Lasbennes, M.C., Linghu, B., Oakeley, E.J., Zarhrate, M., Nitschké, P., Garfa-Traore, M., Serluca, F., Yang, F., Bouwmeester, T., Pinson, L., Cassuto, E., Dubot, P., Elshakhs, N.A., Sahel, J.A., Salomon, R., Drummond, I.A., Gubler, M.C., Antignac, C., Chibout, S., Szustakowski, J.D., Hildebrandt, F., Lorentzen, E., Sailer, A.W., Benmerah, A., Saint-Mezard, P., Saunier, S.
ID
ZDB-PUB-151022-2
Date
2015
Source
Nature communications   6: 8666 (Journal)
Registered Authors
Delous, Marion, Drummond, Iain, Ryan, Rebecca, Saunier, Sophie, Serluca, Fabrizio, Yang, Fan
Keywords
Cell signalling, Microtubules, Mutation
MeSH Terms
  • Animals
  • Blotting, Western
  • Carrier Proteins/genetics*
  • Carrier Proteins/metabolism
  • Cell Polarity/genetics
  • Circular Dichroism
  • Embryo, Nonmammalian
  • Female
  • Fluorescent Antibody Technique
  • Gene Knockout Techniques
  • HEK293 Cells
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Immunoprecipitation
  • Kidney Diseases, Cystic/genetics*
  • Kidney Diseases, Cystic/metabolism
  • Male
  • Microphthalmos/genetics
  • Microtubule-Associated Proteins/genetics*
  • Microtubule-Associated Proteins/metabolism*
  • Microtubules/metabolism*
  • Mutation*
  • Pedigree
  • Retinal Degeneration/genetics*
  • Retinal Degeneration/metabolism
  • Reverse Transcriptase Polymerase Chain Reaction
  • Zebrafish
  • Zebrafish Proteins/genetics*
  • Zebrafish Proteins/metabolism
PubMed
26487268 Full text @ Nat. Commun.
Abstract
Ciliopathies are a large group of clinically and genetically heterogeneous disorders caused by defects in primary cilia. Here we identified mutations in TRAF3IP1 (TNF Receptor-Associated Factor Interacting Protein 1) in eight patients from five families with nephronophthisis (NPH) and retinal degeneration, two of the most common manifestations of ciliopathies. TRAF3IP1 encodes IFT54, a subunit of the IFT-B complex required for ciliogenesis. The identified mutations result in mild ciliary defects in patients but also reveal an unexpected role of IFT54 as a negative regulator of microtubule stability via MAP4 (microtubule-associated protein 4). Microtubule defects are associated with altered epithelialization/polarity in renal cells and with pronephric cysts and microphthalmia in zebrafish embryos. Our findings highlight the regulation of cytoplasmic microtubule dynamics as a role of the IFT54 protein beyond the cilium, contributing to the development of NPH-related ciliopathies.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping