Mutations in CEP78 Cause Cone-Rod Dystrophy and Hearing Loss Associated with Primary-Cilia Defects.

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State: Public
Version: Author's accepted manuscript
Serval ID
serval:BIB_4A7C86DC11B4
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Mutations in CEP78 Cause Cone-Rod Dystrophy and Hearing Loss Associated with Primary-Cilia Defects.
Journal
American journal of human genetics
Author(s)
Nikopoulos K., Farinelli P., Giangreco B., Tsika C., Royer-Bertrand B., Mbefo M.K., Bedoni N., Kjellström U., El Zaoui I., Di Gioia S.A., Balzano S., Cisarova K., Messina A., Decembrini S., Plainis S., Blazaki S.V., Khan M.I., Micheal S., Boldt K., Ueffing M., Moulin A.P., Cremers F.P., Roepman R., Arsenijevic Y., Tsilimbaris M.K., Andréasson S., Rivolta C.
ISSN
1537-6605 (Electronic)
ISSN-L
0002-9297
Publication state
Published
Issued date
01/09/2016
Peer-reviewed
Oui
Volume
99
Number
3
Pages
770-776
Language
english
Notes
Publication types: Case Reports ; Journal Article
Publication Status: ppublish
Abstract
Cone-rod degeneration (CRD) belongs to the disease spectrum of retinal degenerations, a group of hereditary disorders characterized by an extreme clinical and genetic heterogeneity. It mainly differentiates from other retinal dystrophies, and in particular from the more frequent disease retinitis pigmentosa, because cone photoreceptors degenerate at a higher rate than rod photoreceptors, causing severe deficiency of central vision. After exome analysis of a cohort of individuals with CRD, we identified biallelic mutations in the orphan gene CEP78 in three subjects from two families: one from Greece and another from Sweden. The Greek subject, from the island of Crete, was homozygous for the c.499+1G>T (IVS3+1G>T) mutation in intron 3. The Swedish subjects, two siblings, were compound heterozygotes for the nearby mutation c.499+5G>A (IVS3+5G>A) and for the frameshift-causing variant c.633delC (p.Trp212Glyfs(∗)18). In addition to CRD, these three individuals had hearing loss or hearing deficit. Immunostaining highlighted the presence of CEP78 in the inner segments of retinal photoreceptors, predominantly of cones, and at the base of the primary cilium of fibroblasts. Interaction studies also showed that CEP78 binds to FAM161A, another ciliary protein associated with retinal degeneration. Finally, analysis of skin fibroblasts derived from affected individuals revealed abnormal ciliary morphology, as compared to that of control cells. Altogether, our data strongly suggest that mutations in CEP78 cause a previously undescribed clinical entity of a ciliary nature characterized by blindness and deafness but clearly distinct from Usher syndrome, a condition for which visual impairment is due to retinitis pigmentosa.

Keywords
Aged, Alleles, Animals, Cadaver, Cell Cycle Proteins/genetics, Cell Cycle Proteins/metabolism, Cilia/pathology, Cohort Studies, Cone-Rod Dystrophies/complications, Cone-Rod Dystrophies/genetics, Cone-Rod Dystrophies/pathology, Cone-Rod Dystrophies/physiopathology, Exome/genetics, Eye/embryology, Eye/metabolism, Eye Proteins/metabolism, Female, Fibroblasts/pathology, Greece, Hearing Loss, Sensorineural/complications, Hearing Loss, Sensorineural/genetics, Hearing Loss, Sensorineural/pathology, Hearing Loss, Sensorineural/physiopathology, Heterozygote, Homozygote, Humans, Introns/genetics, Male, Mice, Middle Aged, Mutation/genetics, Pedigree, Protein Binding, RNA, Messenger/analysis, Sweden, Transcriptome, Usher Syndromes/pathology
Pubmed
Open Access
Yes
Create date
06/09/2016 8:41
Last modification date
23/10/2020 10:52
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