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.

Details

Serval ID
serval:BIB_9F197C8F5F8D
Type
Article: article from journal or magazin.
Publication sub-type
Case report (case report): feedback on an observation with a short commentary.
Collection
Publications
Title
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.
Journal
American Journal of Medical Genetics
Author(s)
Sobetzko D., Eich G., Kalff-Suske M., Grzeschik K.H., Superti-Furga A.
ISSN
0148-7299 (Print)
ISSN-L
0148-7299
Publication state
Published
Issued date
2000
Volume
90
Number
3
Pages
239-242
Language
english
Notes
Publication types: Case Reports ; Journal Article ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Abstract
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.
Keywords
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
Create date
14/03/2011 17:08
Last modification date
20/08/2019 16:05
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