Boy with syndactylies, macrocephaly, and severe skeletal dysplasia: not a new syndrome, but two dominant mutations (GLI3 E543X and COL2A1 G973R) in the same individual.

Détails

ID Serval
serval:BIB_9F197C8F5F8D
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Etude de cas (case report): rapporte une observation et la commente brièvement.
Collection
Publications
Titre
Boy with syndactylies, macrocephaly, and severe skeletal dysplasia: not a new syndrome, but two dominant mutations (GLI3 E543X and COL2A1 G973R) in the same individual.
Périodique
American Journal of Medical Genetics
Auteur⸱e⸱s
Sobetzko D., Eich G., Kalff-Suske M., Grzeschik K.H., Superti-Furga A.
ISSN
0148-7299 (Print)
ISSN-L
0148-7299
Statut éditorial
Publié
Date de publication
2000
Volume
90
Numéro
3
Pages
239-242
Langue
anglais
Notes
Publication types: Case Reports ; Journal Article ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Résumé
An unusual combination of syndactylies, macrocephaly, and severe skeletal dysplasia was observed in a newborn infant. A history of digital anomalies in the father and grandfather lead to the diagnosis of dominantly inherited Greig cephalopolysyndactyly syndrome (GCPS, MIM #175700). Having explained the digital findings and macrocephaly, the skeletal changes were thought to fit best congenital spondyloepiphyseal dysplasia (SEDC MIM #183900), a type II collagen disorder. Molecular analysis confirmed the presence of two dominant mutations in the propositus: a GLI3 mutation (E543X), which was present also in the father and grandfather, and a de novo COL2A1 mutation leading to a G973R substitution. Thus, this boy combined the syndactyly-macrocephaly phenotype of Greig cephalosyndactyly syndrome with a severe form of spondyloepiphyseal dysplasia caused by the structural defect in type II collagen. The diagnostic difficulties posed by the combination of two genetic disorders and the contribution of molecular diagnostics are well illustrated by this case.
Mots-clé
Abnormalities, Multiple/genetics, Collagen/genetics, DNA-Binding Proteins/genetics, Female, Genes, Dominant, Humans, Infant, Kruppel-Like Transcription Factors, Lod Score, Male, Nerve Tissue Proteins, Osteochondrodysplasias/genetics, Pedigree, Point Mutation, Repressor Proteins, Skull/abnormalities, Transcription Factors/genetics, Xenopus Proteins
Pubmed
Web of science
Création de la notice
14/03/2011 17:08
Dernière modification de la notice
20/08/2019 16:05
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