Different Phenotypes in Pseudodominant Inherited Retinal Dystrophies.

pathogenic variants pseudodominant inheritance retinal dystrophies retinitis pigmentosa whole exome sequencing

Journal

Frontiers in cell and developmental biology
ISSN: 2296-634X
Titre abrégé: Front Cell Dev Biol
Pays: Switzerland
ID NLM: 101630250

Informations de publication

Date de publication:
2021
Historique:
received: 03 11 2020
accepted: 11 01 2021
entrez: 26 2 2021
pubmed: 27 2 2021
medline: 27 2 2021
Statut: epublish

Résumé

Retinal dystrophies (RD) are a group of Mendelian disorders caused by rare genetic variations leading to blindness. A pathogenic variant may manifest in both dominant or recessive mode and clinical and genetic heterogeneity makes it difficult to establish a precise diagnosis. In this study, families with autosomal dominant RD in successive generations were identified, and we aimed to determine the disease's molecular origin in these consanguineous families. Whole exome sequencing was performed in the index patient of each family. The aim was to determine whether these cases truly represented examples of dominantly inherited RD, or whether another mode of inheritance might be applicable. Six potentially pathogenic variants in four genes were identified in four families. In index patient with enhanced S-cone syndrome in F1, we identified a new digenetic combination: a heterozygous variant p.[G51A];[=] in

Identifiants

pubmed: 33634125
doi: 10.3389/fcell.2021.625560
pmc: PMC7902019
doi:

Types de publication

Journal Article

Langues

eng

Pagination

625560

Informations de copyright

Copyright © 2021 Habibi, Falfoul, Tran, El Matri, Chebil, El Matri and Schorderet.

Déclaration de conflit d'intérêts

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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Auteurs

Imen Habibi (I)

IRO-Institute for Research in Ophthalmology, Sion, Switzerland.

Yosra Falfoul (Y)

Oculogenetic Laboratory LR14SP01, Faculty of Medicine of Tunis, Hedi Rais Institute of Ophthalmology (Department B), Tunis El Manar University, Tunis, Tunisia.

Hoai Viet Tran (HV)

Hôpital Ophtalmique Jules-Gonin, Unité d'oculogénétique, Lausanne, Switzerland.

Khaled El Matri (K)

Oculogenetic Laboratory LR14SP01, Faculty of Medicine of Tunis, Hedi Rais Institute of Ophthalmology (Department B), Tunis El Manar University, Tunis, Tunisia.

Ahmed Chebil (A)

Oculogenetic Laboratory LR14SP01, Faculty of Medicine of Tunis, Hedi Rais Institute of Ophthalmology (Department B), Tunis El Manar University, Tunis, Tunisia.

Leila El Matri (L)

Oculogenetic Laboratory LR14SP01, Faculty of Medicine of Tunis, Hedi Rais Institute of Ophthalmology (Department B), Tunis El Manar University, Tunis, Tunisia.

Daniel F Schorderet (DF)

IRO-Institute for Research in Ophthalmology, Sion, Switzerland.
Faculty of Biology and Medicine, University of Lausanne, Lausanne, Switzerland.
Faculty of Life Sciences, Ecole Polytechnique Fédérale de Lausanne, Lausanne, Switzerland.

Classifications MeSH