Aberrant Splicing Events Associated to


Journal

Genes
ISSN: 2073-4425
Titre abrégé: Genes (Basel)
Pays: Switzerland
ID NLM: 101551097

Informations de publication

Date de publication:
21 09 2019
Historique:
received: 04 09 2019
revised: 17 09 2019
accepted: 19 09 2019
entrez: 25 9 2019
pubmed: 25 9 2019
medline: 17 3 2020
Statut: epublish

Résumé

The aim of this study was the genetic diagnosis by next generation sequencing (NGS) of a patient diagnosed with Usher syndrome type 2 and the functional evaluation of the identified genetic variants to establish a phenotype-genotype correlation. Whole exome sequencing (WES) analysis identified two heterozygous intronic variants in Two intronic variants were identified in intron 45 of A combination of in vivo and in vitro assays provided a comprehensive view of the physiological effects of NCSS variants, which in this case led to a clinical reassignment of the proband as affected with atypical USH1 syndrome.

Identifiants

pubmed: 31546658
pii: genes10100732
doi: 10.3390/genes10100732
pmc: PMC6826400
pii:
doi:

Substances chimiques

CDH23 protein, human 0
Cadherin Related Proteins 0
Cadherins 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Références

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Auteurs

Rebeca Valero (R)

DBGen Ocular Genomics, 08028 Barcelona, Spain. rvalero@ub.edu.

Marta de Castro-Miró (M)

DBGen Ocular Genomics, 08028 Barcelona, Spain. martadecastromiro@ub.edu.

Sofía Jiménez-Ochoa (S)

DBGen Ocular Genomics, 08028 Barcelona, Spain. sofiacorella96@gmail.com.
Departament de Genètica, Microbiologia i Estadística, Facultat de Biologia, Universitat de Barcelona, Avda.Diagonal 643, 08028 Barcelona, Spain. sofiacorella96@gmail.com.

Juan José Rodríguez-Ezcurra (JJ)

Barraquer-Centro de Oftalmología Barcelona, 08021 Barcelona, Spain. jrezcurra@barraquer.com.

Gemma Marfany (G)

DBGen Ocular Genomics, 08028 Barcelona, Spain. gmarfany@ub.edu.
Departament de Genètica, Microbiologia i Estadística, Facultat de Biologia, Universitat de Barcelona, Avda.Diagonal 643, 08028 Barcelona, Spain. gmarfany@ub.edu.
Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), 08025 Barcelona, Spain. gmarfany@ub.edu.
Institut de Biomedicina de la Universitat de Barcelona-Institut de Recerca Sant Joan de Deu (IBUB-IRSJD), 08028 Barcelona, Spain. gmarfany@ub.edu.

Roser Gonzàlez-Duarte (R)

DBGen Ocular Genomics, 08028 Barcelona, Spain. rgonzalez@dbgen.com.

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Classifications MeSH