Aarskog-Scott syndrome: clinical update and report of nine novel mutations of the FGD1 gene.

Détails

ID Serval
serval:BIB_D4872E610ACE
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Institution
Titre
Aarskog-Scott syndrome: clinical update and report of nine novel mutations of the FGD1 gene.
Périodique
American Journal of Medical Genetics
Auteur⸱e⸱s
Orrico A., Galli L., Faivre L., Clayton-Smith J., Azzarello-Burri S.M., Hertz J.M., Jacquemont S., Taurisano R., Arroyo Carrera I., Tarantino E., Devriendt K., Melis D., Thelle T., Meinhardt U., Sorrentino V.
ISSN
1552-4833[electronic], 1552-4825[linking]
Statut éditorial
Publié
Date de publication
2010
Volume
152 A
Numéro
2
Pages
313-318
Langue
anglais
Résumé
Mutations in the FGD1 gene have been shown to cause Aarskog-Scott syndrome (AAS), or facio-digito-genital dysplasia (OMIM#305400), an X-linked disorder characterized by distinctive genital and skeletal developmental abnormalities with a broad spectrum of clinical phenotypes. To date, 20 distinct mutations have been reported, but little phenotypic data are available on patients with molecularly confirmed AAS. In the present study, we report on our experience of screening for mutations in the FGD1 gene in a cohort of 60 European patients with a clinically suspected diagnosis of AAS. We identified nine novel mutations in 11 patients (detection rate of 18.33%), including three missense mutations (p.R402Q; p.S558W; p.K748E), four truncating mutations (p.Y530X; p.R656X; c.806delC; c.1620delC), one in-frame deletion (c.2020_2022delGAG) and the first reported splice site mutation (c.1935+3A>C). A recurrent mutation (p.R656X) was detected in three independent families. We did not find any evidence for phenotype-genotype correlations between type and position of mutations and clinical features. In addition to the well-established phenotypic features of AAS, other clinical features are also reported and discussed.
Mots-clé
Aarskog-Scott Syndrome, FGD1 Gene, Clinical Features, Mutations, Digital-Genital Syndrome, Faciogenital-Dysplasia, Missense Mutation, Family, Expression, Domain
Pubmed
Web of science
Création de la notice
02/03/2010 15:21
Dernière modification de la notice
20/08/2019 16:54
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