Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity

Autozygosity is associated with an increased risk of genetic rare disease, thus being a relevant factor for clinical genetic studies. More than 2400 exome sequencing data sets were analyzed and screened for autozygosity on the basis of detection of >1 Mbp runs of homozygosity (ROHs). A model was...

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Autores: Matalonga Borrel, Lesley, Laurie, Steven, Papakonstantinou, Anastasios, Piscia, Davide, Mereu, Elisabetta, Bullich, Gemma, Thompson, Rachel, Horvath, Rita, Pérez Jurado, Luis A., Riess, Olaf, Gut, Ivo, van Ommen, Gert Jan, Lochmüller, Hanns, Beltrán, Sergi, Rare Disease-Connect Genome-Phenome Analysis Platform data contributors, Undiagnosed Rare Disease Programme of Catalonia data contibutors, Cormand Rifà, Bru, Balcells Comas, Susana, Grinberg Vaisman, Daniel Raúl, Urreizti, Roser, Garrabou Tornos, Glòria
Tipo de recurso: artículo
Estado:Versión aceptada para publicación
Fecha de publicación:2020
País:España
Institución:Universidad de Barcelona
Repositorio:Dipòsit Digital de la UB
OAI Identifier:oai:diposit.ub.edu:2445/174442
Acceso en línea:https://hdl.handle.net/2445/174442
Access Level:acceso abierto
Palabra clave:Malalties hereditàries
Endogàmia i exogàmia
Consanguinitat
Genetic diseases
Endogamy and exogamy
Consanguinity
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spelling Improved diagnosis of rare disease patients through systematic detection of runs of homozygosityMatalonga Borrel, LesleyLaurie, StevenPapakonstantinou, AnastasiosPiscia, DavideMereu, ElisabettaBullich, GemmaThompson, RachelHorvath, RitaPérez Jurado, Luis A.Riess, OlafGut, Ivovan Ommen, Gert JanLochmüller, HannsBeltrán, SergiRare Disease-Connect Genome-Phenome Analysis Platform data contributorsUndiagnosed Rare Disease Programme of Catalonia data contibutorsCormand Rifà, BruBalcells Comas, SusanaGrinberg Vaisman, Daniel RaúlUrreizti, RoserGarrabou Tornos, GlòriaMalalties hereditàriesEndogàmia i exogàmiaConsanguinitatGenetic diseasesEndogamy and exogamyConsanguinityAutozygosity is associated with an increased risk of genetic rare disease, thus being a relevant factor for clinical genetic studies. More than 2400 exome sequencing data sets were analyzed and screened for autozygosity on the basis of detection of >1 Mbp runs of homozygosity (ROHs). A model was built to predict if an individual is likely to be a consanguineous offspring (accuracy, 98%), and probability of consanguinity ranges were established according to the total ROH size. Application of the model resulted in the reclassification of the consanguinity status of 12% of the patients. The analysis of a subset of 79 consanguineous cases with the Rare Disease (RD)-Connect Genome-Phenome Analysis Platform, combining variant filtering and homozygosity mapping, enabled a 50% reduction in the number of candidate variants and the identification of homozygous pathogenic variants in 41 patients, with an overall diagnostic yield of 52%. The newly defined consanguinity ranges provide, for the first time, specific ROH thresholds to estimate inbreeding within a pedigree on disparate exome sequencing data, enabling confirmation or (re)classification of consanguineous status, hence increasing the efficiency of molecular diagnosis and reporting on secondary consanguinity findings, as recommended by American College of Medical Genetics and Genomics guidelinesAmerican Society for Investigative Pathology and the Association for Molecular Pathology2020info:eu-repo/semantics/articleinfo:eu-repo/semantics/acceptedVersioninfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/2445/174442Articles publicats en revistes (Genètica, Microbiologia i Estadística)reponame:Dipòsit Digital de la UBinstname:Universidad de BarcelonaInglésReproducció del document publicat a: https://doi.org/10.1016/j.jmoldx.2020.06.008Journal of Molecular Diagnostics, 2020, vol. 22, num. 9, p. 1205-1215https://doi.org/10.1016/j.jmoldx.2020.06.008info:eu-repo/grantAgreement/EC/H2020/779257info:eu-repo/grantAgreement/EC/H2020/825575info:eu-repo/grantAgreement/EC/FP7/305444cc-by-nc-nd (c), Matalonga Borrel et. al., 2020http://creativecommons.org/licenses/by-nc-nd/3.0/es/info:eu-repo/semantics/openAccessoai:diposit.ub.edu:2445/1744422026-05-27T06:46:51Z
dc.title.none.fl_str_mv Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
title Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
spellingShingle Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
Matalonga Borrel, Lesley
Malalties hereditàries
Endogàmia i exogàmia
Consanguinitat
Genetic diseases
Endogamy and exogamy
Consanguinity
title_short Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
title_full Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
title_fullStr Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
title_full_unstemmed Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
title_sort Improved diagnosis of rare disease patients through systematic detection of runs of homozygosity
dc.creator.none.fl_str_mv Matalonga Borrel, Lesley
Laurie, Steven
Papakonstantinou, Anastasios
Piscia, Davide
Mereu, Elisabetta
Bullich, Gemma
Thompson, Rachel
Horvath, Rita
Pérez Jurado, Luis A.
Riess, Olaf
Gut, Ivo
van Ommen, Gert Jan
Lochmüller, Hanns
Beltrán, Sergi
Rare Disease-Connect Genome-Phenome Analysis Platform data contributors
Undiagnosed Rare Disease Programme of Catalonia data contibutors
Cormand Rifà, Bru
Balcells Comas, Susana
Grinberg Vaisman, Daniel Raúl
Urreizti, Roser
Garrabou Tornos, Glòria
author Matalonga Borrel, Lesley
author_facet Matalonga Borrel, Lesley
Laurie, Steven
Papakonstantinou, Anastasios
Piscia, Davide
Mereu, Elisabetta
Bullich, Gemma
Thompson, Rachel
Horvath, Rita
Pérez Jurado, Luis A.
Riess, Olaf
Gut, Ivo
van Ommen, Gert Jan
Lochmüller, Hanns
Beltrán, Sergi
Rare Disease-Connect Genome-Phenome Analysis Platform data contributors
Undiagnosed Rare Disease Programme of Catalonia data contibutors
Cormand Rifà, Bru
Balcells Comas, Susana
Grinberg Vaisman, Daniel Raúl
Urreizti, Roser
Garrabou Tornos, Glòria
author_role author
author2 Laurie, Steven
Papakonstantinou, Anastasios
Piscia, Davide
Mereu, Elisabetta
Bullich, Gemma
Thompson, Rachel
Horvath, Rita
Pérez Jurado, Luis A.
Riess, Olaf
Gut, Ivo
van Ommen, Gert Jan
Lochmüller, Hanns
Beltrán, Sergi
Rare Disease-Connect Genome-Phenome Analysis Platform data contributors
Undiagnosed Rare Disease Programme of Catalonia data contibutors
Cormand Rifà, Bru
Balcells Comas, Susana
Grinberg Vaisman, Daniel Raúl
Urreizti, Roser
Garrabou Tornos, Glòria
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Malalties hereditàries
Endogàmia i exogàmia
Consanguinitat
Genetic diseases
Endogamy and exogamy
Consanguinity
topic Malalties hereditàries
Endogàmia i exogàmia
Consanguinitat
Genetic diseases
Endogamy and exogamy
Consanguinity
description Autozygosity is associated with an increased risk of genetic rare disease, thus being a relevant factor for clinical genetic studies. More than 2400 exome sequencing data sets were analyzed and screened for autozygosity on the basis of detection of >1 Mbp runs of homozygosity (ROHs). A model was built to predict if an individual is likely to be a consanguineous offspring (accuracy, 98%), and probability of consanguinity ranges were established according to the total ROH size. Application of the model resulted in the reclassification of the consanguinity status of 12% of the patients. The analysis of a subset of 79 consanguineous cases with the Rare Disease (RD)-Connect Genome-Phenome Analysis Platform, combining variant filtering and homozygosity mapping, enabled a 50% reduction in the number of candidate variants and the identification of homozygous pathogenic variants in 41 patients, with an overall diagnostic yield of 52%. The newly defined consanguinity ranges provide, for the first time, specific ROH thresholds to estimate inbreeding within a pedigree on disparate exome sequencing data, enabling confirmation or (re)classification of consanguineous status, hence increasing the efficiency of molecular diagnosis and reporting on secondary consanguinity findings, as recommended by American College of Medical Genetics and Genomics guidelines
publishDate 2020
dc.date.none.fl_str_mv 2020
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/acceptedVersion
info:eu-repo/semantics/publishedVersion
format article
status_str acceptedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/2445/174442
url https://hdl.handle.net/2445/174442
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Reproducció del document publicat a: https://doi.org/10.1016/j.jmoldx.2020.06.008
Journal of Molecular Diagnostics, 2020, vol. 22, num. 9, p. 1205-1215
https://doi.org/10.1016/j.jmoldx.2020.06.008
info:eu-repo/grantAgreement/EC/H2020/779257
info:eu-repo/grantAgreement/EC/H2020/825575
info:eu-repo/grantAgreement/EC/FP7/305444
dc.rights.none.fl_str_mv cc-by-nc-nd (c), Matalonga Borrel et. al., 2020
http://creativecommons.org/licenses/by-nc-nd/3.0/es/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv cc-by-nc-nd (c), Matalonga Borrel et. al., 2020
http://creativecommons.org/licenses/by-nc-nd/3.0/es/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv American Society for Investigative Pathology and the Association for Molecular Pathology
publisher.none.fl_str_mv American Society for Investigative Pathology and the Association for Molecular Pathology
dc.source.none.fl_str_mv Articles publicats en revistes (Genètica, Microbiologia i Estadística)
reponame:Dipòsit Digital de la UB
instname:Universidad de Barcelona
instname_str Universidad de Barcelona
reponame_str Dipòsit Digital de la UB
collection Dipòsit Digital de la UB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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