Document Detail


Genotype-phenotype analysis of TCF4 mutations causing Pitt-Hopkins syndrome shows increased seizure activity with missense mutations.
MedLine Citation:
PMID:  19938247     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
PURPOSE: Pitt-Hopkins syndrome is characterized by severe mental retardation, characteristic dysmorphic features, and susceptibility to childhood-onset seizures and intermittent episodes of hyperventilation. This syndrome is caused by haploinsufficiency of TCF4, which encodes a basic helix-loop-helix transcription factor. Missense, nonsense, splice-site mutations, and gene deletions have been found in individuals with Pitt-Hopkins syndrome. Previous reports have suggested that the Pitt-Hopkins syndrome phenotype is independent of mutation or deletion type. METHODS: We screened 13,186 individuals with microarray-based comparative genomic hybridization. We also conducted a review of the literature and statistical analysis of the phenotypic features for all individuals with confirmed mutations or deletions of TCF4. RESULTS: We identified seven individuals with TCF4 deletions. All patients have features consistent with Pitt-Hopkins syndrome, although only three have breathing anomalies, and none has seizures. Our review of previously reported cases with TCF4 mutations and deletions showed that all patients with Pitt-Hopkins syndrome reported to date have severe psychomotor retardation, the onsets of seizures and hyperventilation episodes are limited to the first decade in most reported patients with Pitt-Hopkins syndrome, hyperventilation episodes are more common than seizures and are seen in the oldest patients, and individuals with missense TCF4 mutations are more likely to develop seizures. CONCLUSIONS: On the basis of an analysis of published cases, we propose a genotype-phenotype correlation of increased seizure activity with missense TCF4 mutations.
Authors:
Jill A Rosenfeld; Kathleen Leppig; Blake C Ballif; Heidi Thiese; Christine Erdie-Lalena; Erwati Bawle; Sujatha Sastry; J Edward Spence; Anne Bandholz; Urvashi Surti; Jonathan Zonana; Kory Keller; Wendy Meschino; Bassem A Bejjani; Beth S Torchia; Lisa G Shaffer
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Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Genetics in medicine : official journal of the American College of Medical Genetics     Volume:  11     ISSN:  1530-0366     ISO Abbreviation:  Genet. Med.     Publication Date:  2009 Nov 
Date Detail:
Created Date:  2009-11-17     Completed Date:  2010-01-13     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9815831     Medline TA:  Genet Med     Country:  United States    
Other Details:
Languages:  eng     Pagination:  797-805     Citation Subset:  IM    
Affiliation:
Signature Genomic Laboratories, 2820 N. AstorStreet, Spokane, WA 99207, USA.
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MeSH Terms
Descriptor/Qualifier:
Basic Helix-Loop-Helix Leucine Zipper Transcription Factors / genetics*
Child
Female
Genotype
Humans
Male
Mental Retardation / genetics*,  physiopathology*
Mutation, Missense*
Phenotype
Seizures / genetics*,  physiopathology*
Sequence Deletion
Syndrome
Transcription Factors / genetics*
Chemical
Reg. No./Substance:
0/Basic Helix-Loop-Helix Leucine Zipper Transcription Factors; 0/TCF4 protein, human; 0/Transcription Factors

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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