Literature DB >> 25812041

Difficulties in diagnosing Marfan syndrome using current FBN1 databases.

Kristian A Groth1,2, Mette Gaustadnes2, Kasper Thorsen2, John R Østergaard3, Uffe Birk Jensen4, Claus H Gravholt2,5, Niels H Andersen1.   

Abstract

PURPOSE: The diagnostic criteria of Marfan syndrome (MFS) highlight the importance of a FBN1 mutation test in diagnosing MFS. As genetic sequencing becomes better, cheaper, and more accessible, the expected increase in the number of genetic tests will become evident, resulting in numerous genetic variants that need to be evaluated for disease-causing effects based on database information. The aim of this study was to evaluate genetic variants in four databases and review the relevant literature.
METHODS: We assessed background data on 23 common variants registered in ESP6500 and classified as causing MFS in the Human Gene Mutation Database (HGMD). We evaluated data in four variant databases (HGMD, UMD-FBN1, ClinVar, and UniProt) according to the diagnostic criteria for MFS and compared the results with the classification of each variant in the four databases.
RESULTS: None of the 23 variants was clearly associated with MFS, even though all classifications in the databases stated otherwise.
CONCLUSION: A genetic diagnosis of MFS cannot reliably be based on current variant databases because they contain incorrectly interpreted conclusions on variants. Variants must be evaluated by time-consuming review of the background material in the databases and by combining these data with expert knowledge on MFS. This is a major problem because we expect even more genetic test results in the near future as a result of the reduced cost and process time for next-generation sequencing.Genet Med 18 1, 98-102.

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Year:  2015        PMID: 25812041     DOI: 10.1038/gim.2015.32

Source DB:  PubMed          Journal:  Genet Med        ISSN: 1098-3600            Impact factor:   8.822


  17 in total

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2.  Marfan syndrome caused by a recurrent de novo missense mutation in the fibrillin gene.

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Journal:  Nature       Date:  1991-07-25       Impact factor: 49.962

3.  Analysis of phenotype and genotype information for the diagnosis of Marfan syndrome.

Authors:  S Sheikhzadeh; C Kade; B Keyser; M Stuhrmann; M Arslan-Kirchner; M Rybczynski; A M Bernhardt; C R Habermann; M Hillebrand; T Mir; P N Robinson; J Berger; C Detter; S Blankenberg; J Schmidtke; Y von Kodolitsch
Journal:  Clin Genet       Date:  2011-10-05       Impact factor: 4.438

4.  Revised diagnostic criteria for the Marfan syndrome.

Authors:  A De Paepe; R B Devereux; H C Dietz; R C Hennekam; R E Pyeritz
Journal:  Am J Med Genet       Date:  1996-04-24

5.  The revised Ghent nosology for the Marfan syndrome.

Authors:  Bart L Loeys; Harry C Dietz; Alan C Braverman; Bert L Callewaert; Julie De Backer; Richard B Devereux; Yvonne Hilhorst-Hofstee; Guillaume Jondeau; Laurence Faivre; Dianna M Milewicz; Reed E Pyeritz; Paul D Sponseller; Paul Wordsworth; Anne M De Paepe
Journal:  J Med Genet       Date:  2010-07       Impact factor: 6.318

6.  Actionable, pathogenic incidental findings in 1,000 participants' exomes.

Authors:  Michael O Dorschner; Laura M Amendola; Emily H Turner; Peggy D Robertson; Brian H Shirts; Carlos J Gallego; Robin L Bennett; Kelly L Jones; Mari J Tokita; James T Bennett; Jerry H Kim; Elisabeth A Rosenthal; Daniel S Kim; Holly K Tabor; Michael J Bamshad; Arno G Motulsky; C Ronald Scott; Colin C Pritchard; Tom Walsh; Wylie Burke; Wendy H Raskind; Peter Byers; Fuki M Hisama; Deborah A Nickerson; Gail P Jarvik
Journal:  Am J Hum Genet       Date:  2013-09-19       Impact factor: 11.025

7.  Comparison of heteroduplex analysis, direct sequencing, and enzyme mismatch cleavage for detecting mutations in a large gene, FBN1.

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Journal:  Hum Mutat       Date:  1999       Impact factor: 4.878

8.  Systematic screening of FBN1 gene unclassified missense variants for splice abnormalities.

Authors:  D O Robinson; F Lin; M Lyon; M Raponi; E Cross; H E White; H Cox; J Clayton-Smith; D Baralle
Journal:  Clin Genet       Date:  2011-09-30       Impact factor: 4.438

Review 9.  Update of the UMD-FBN1 mutation database and creation of an FBN1 polymorphism database.

Authors:  Gwenaëlle Collod-Béroud; Saga Le Bourdelles; Lesley Ades; Leena Ala-Kokko; Patrick Booms; Maureen Boxer; Anne Child; Paolo Comeglio; Anne De Paepe; James C Hyland; Katerine Holman; Ilkka Kaitila; Bart Loeys; Gabor Matyas; Lieve Nuytinck; Leena Peltonen; Terhi Rantamaki; Peter Robinson; Beat Steinmann; Claudine Junien; Christophe Béroud; Catherine Boileau
Journal:  Hum Mutat       Date:  2003-09       Impact factor: 4.878

10.  ClinVar: public archive of relationships among sequence variation and human phenotype.

Authors:  Melissa J Landrum; Jennifer M Lee; George R Riley; Wonhee Jang; Wendy S Rubinstein; Deanna M Church; Donna R Maglott
Journal:  Nucleic Acids Res       Date:  2013-11-14       Impact factor: 16.971

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  7 in total

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Authors:  Linnea M Baudhuin; Michelle L Kluge; Katrina E Kotzer; Susan A Lagerstedt
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2.  Aortic events in a nationwide Marfan syndrome cohort.

Authors:  Kristian A Groth; Kirstine Stochholm; Hanne Hove; Kasper Kyhl; Pernille A Gregersen; Niels Vejlstrup; John R Østergaard; Claus H Gravholt; Niels H Andersen
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3.  Evaluating the quality of Marfan genotype-phenotype correlations in existing FBN1 databases.

Authors:  Kristian A Groth; Yskert Von Kodolitsch; Kerstin Kutsche; Mette Gaustadnes; Kasper Thorsen; Niels H Andersen; Claus H Gravholt
Journal:  Genet Med       Date:  2016-12-01       Impact factor: 8.822

4.  Genomic Screening: The Mutation and the Mustard Seed.

Authors:  Thomas M Morgan
Journal:  J Law Med Ethics       Date:  2018-06       Impact factor: 1.604

5.  Prevalence, incidence, and age at diagnosis in Marfan Syndrome.

Authors:  Kristian A Groth; Hanne Hove; Kasper Kyhl; Lars Folkestad; Mette Gaustadnes; Niels Vejlstrup; Kirstine Stochholm; John R Østergaard; Niels H Andersen; Claus H Gravholt
Journal:  Orphanet J Rare Dis       Date:  2015-12-02       Impact factor: 4.123

6.  A novel molecular diagnostics platform for somatic and germline precision oncology.

Authors:  Rubén Cabanillas; Marta Diñeiro; David Castillo; Patricia C Pruneda; Cristina Penas; Guadalupe A Cifuentes; Álvaro de Vicente; Noelia S Durán; Rebeca Álvarez; Gonzalo R Ordóñez; Juan Cadiñanos
Journal:  Mol Genet Genomic Med       Date:  2017-04-23       Impact factor: 2.183

Review 7.  The Molecular Genetics of Marfan Syndrome.

Authors:  Qiu Du; Dingding Zhang; Yue Zhuang; Qiongrong Xia; Taishen Wen; Haiping Jia
Journal:  Int J Med Sci       Date:  2021-05-27       Impact factor: 3.738

  7 in total

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