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Radboud Repository
Article . 2007
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Nature Genetics
Article . 2007 . Peer-reviewed
License: Springer TDM
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Nature Genetics
Article . 2007
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Mutations in the gene encoding the basal body protein RPGRIP1L, a nephrocystin-4 interactor, cause Joubert syndrome

Authors: Arts, H.H.; Doherty, D.; Beersum, S.E.C. van; Parisi, M.A.; Letteboer, S.J.F.; Gorden, N.T.; Peters, T.A.; +12 Authors

Mutations in the gene encoding the basal body protein RPGRIP1L, a nephrocystin-4 interactor, cause Joubert syndrome

Abstract

Protein-protein interaction analyses have uncovered a ciliary and basal body protein network that, when disrupted, can result in nephronophthisis (NPHP), Leber congenital amaurosis, Senior-Løken syndrome (SLSN) or Joubert syndrome (JBTS). However, details of the molecular mechanisms underlying these disorders remain poorly understood. RPGRIP1-like protein (RPGRIP1L) is a homolog of RPGRIP1 (RPGR-interacting protein 1), a ciliary protein defective in Leber congenital amaurosis. We show that RPGRIP1L interacts with nephrocystin-4 and that mutations in the gene encoding nephrocystin-4 (NPHP4) that are known to cause SLSN disrupt this interaction. RPGRIP1L is ubiquitously expressed, and its protein product localizes to basal bodies. Therefore, we analyzed RPGRIP1L as a candidate gene for JBTS and identified loss-of-function mutations in three families with typical JBTS, including the characteristic mid-hindbrain malformation. This work identifies RPGRIP1L as a gene responsible for JBTS and establishes a central role for cilia and basal bodies in the pathophysiology of this disorder.

Keywords

Adult, Male, DCN 1: Perception and Action, Eye Diseases, DCN 2: Functional Neurogenomics, Molecular Sequence Data, NCMLS 6: Genetics and epigenetic pathways of disease, NCMLS 5: Membrane transport and intracellular motility, UMCN 5.1: Genetic defects of metabolism, Cell Line, IGMD 3: Genomic disorders and inherited multi-system disorders, Cerebellar Diseases, UMCN 3.3: Neurosensory disorders, Animals, Humans, Cilia, Adaptor Proteins, Signal Transducing, IGMD 9: Renal disorder, Proteins, Syndrome, Pedigree, Rats, Cytoskeletal Proteins, Female, Kidney Diseases, IGMD 5: Health aging / healthy living, Ciliary Motility Disorders

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popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
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influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
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