The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases

Rare disease patients are more likely to receive a rapid molecular diagnosis nowadays thanks to the wide adoption of next-generation sequencing. However, many cases remain undiagnosed even after exome or genome analysis, because the methods used missed the molecular cause in a known gene, or a novel...

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Autores: Laurie, Steven, Piscia, Davide, Matalonga, Leslie, Corvó, Alberto, Fernández-Callejo, Marcos, Garcia-Linares, Carles, Hernández-Ferrer, Carles, Luengo, Cristina, Martínez, Inés, Papakonstantinou, Anastasios, Picó-Amador, Daniel, Protasio, Joan, Thompson, Rachel, Tonda, Raul, Bayés, Mònica, Bullich, Gemma, Camps-Puchadas, Jordi, Paramonov, Ida, Trotta, Jean-Rémi, Alonso, Angel, Attimonelli, Marcella, Béroud, Christophe, Bros-Facer, Virginie, Buske, Orion J, Cañada-Pallarés, Andrés, Fernández, José M, Hansson, Mats G, Horvath, Rita, Jacobsen, Julius O B, Kaliyaperumal, Rajaram, Lair-Préterre, Séverine, Licata, Luana, Lopes, Pedro, Lopez-Martin, Estrella, Mascalzoni, Deborah, Monaco, Lucia, Pérez-Jurado, Luis Alberto, Posada De la Paz, Manuel, Rambla, Jordi, Rath, Ana, Riess, Olaf, Robinson, Peter N, Salgado, David, Smedley, Damian, Spalding, Dylan, 't Hoen, Peter A C, Töpf, Ana, Zaharieva, Irina, Graessner, Holm, Gut, Ivo G, Lochmüller, Hanns, Beltran, Sergi
Tipo de recurso: artículo
Fecha de publicación:2022
País:España
Institución:Instituto de Salud Carlos III (ISCIII)
Repositorio:Repisalud
Idioma:inglés
OAI Identifier:oai:repisalud.isciii.es:20.500.12105/15877
Acceso en línea:http://hdl.handle.net/20.500.12105/15877
Access Level:acceso abierto
Palabra clave:Genomics
Rare Diseases
Exome
Genetic Association Studies
Humans
Phenotype
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dc.title.none.fl_str_mv The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
title The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
spellingShingle The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
Laurie, Steven
Genomics
Rare Diseases
Exome
Genetic Association Studies
Humans
Phenotype
title_short The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
title_full The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
title_fullStr The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
title_full_unstemmed The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
title_sort The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseases
dc.creator.none.fl_str_mv Laurie, Steven
Piscia, Davide
Matalonga, Leslie
Corvó, Alberto
Fernández-Callejo, Marcos
Garcia-Linares, Carles
Hernández-Ferrer, Carles
Luengo, Cristina
Martínez, Inés
Papakonstantinou, Anastasios
Picó-Amador, Daniel
Protasio, Joan
Thompson, Rachel
Tonda, Raul
Bayés, Mònica
Bullich, Gemma
Camps-Puchadas, Jordi
Paramonov, Ida
Trotta, Jean-Rémi
Alonso, Angel
Attimonelli, Marcella
Béroud, Christophe
Bros-Facer, Virginie
Buske, Orion J
Cañada-Pallarés, Andrés
Fernández, José M
Hansson, Mats G
Horvath, Rita
Jacobsen, Julius O B
Kaliyaperumal, Rajaram
Lair-Préterre, Séverine
Licata, Luana
Lopes, Pedro
Lopez-Martin, Estrella
Mascalzoni, Deborah
Monaco, Lucia
Pérez-Jurado, Luis Alberto
Posada De la Paz, Manuel
Rambla, Jordi
Rath, Ana
Riess, Olaf
Robinson, Peter N
Salgado, David
Smedley, Damian
Spalding, Dylan
't Hoen, Peter A C
Töpf, Ana
Zaharieva, Irina
Graessner, Holm
Gut, Ivo G
Lochmüller, Hanns
Beltran, Sergi
author Laurie, Steven
author_facet Laurie, Steven
Piscia, Davide
Matalonga, Leslie
Corvó, Alberto
Fernández-Callejo, Marcos
Garcia-Linares, Carles
Hernández-Ferrer, Carles
Luengo, Cristina
Martínez, Inés
Papakonstantinou, Anastasios
Picó-Amador, Daniel
Protasio, Joan
Thompson, Rachel
Tonda, Raul
Bayés, Mònica
Bullich, Gemma
Camps-Puchadas, Jordi
Paramonov, Ida
Trotta, Jean-Rémi
Alonso, Angel
Attimonelli, Marcella
Béroud, Christophe
Bros-Facer, Virginie
Buske, Orion J
Cañada-Pallarés, Andrés
Fernández, José M
Hansson, Mats G
Horvath, Rita
Jacobsen, Julius O B
Kaliyaperumal, Rajaram
Lair-Préterre, Séverine
Licata, Luana
Lopes, Pedro
Lopez-Martin, Estrella
Mascalzoni, Deborah
Monaco, Lucia
Pérez-Jurado, Luis Alberto
Posada De la Paz, Manuel
Rambla, Jordi
Rath, Ana
Riess, Olaf
Robinson, Peter N
Salgado, David
Smedley, Damian
Spalding, Dylan
't Hoen, Peter A C
Töpf, Ana
Zaharieva, Irina
Graessner, Holm
Gut, Ivo G
Lochmüller, Hanns
Beltran, Sergi
author_role author
author2 Piscia, Davide
Matalonga, Leslie
Corvó, Alberto
Fernández-Callejo, Marcos
Garcia-Linares, Carles
Hernández-Ferrer, Carles
Luengo, Cristina
Martínez, Inés
Papakonstantinou, Anastasios
Picó-Amador, Daniel
Protasio, Joan
Thompson, Rachel
Tonda, Raul
Bayés, Mònica
Bullich, Gemma
Camps-Puchadas, Jordi
Paramonov, Ida
Trotta, Jean-Rémi
Alonso, Angel
Attimonelli, Marcella
Béroud, Christophe
Bros-Facer, Virginie
Buske, Orion J
Cañada-Pallarés, Andrés
Fernández, José M
Hansson, Mats G
Horvath, Rita
Jacobsen, Julius O B
Kaliyaperumal, Rajaram
Lair-Préterre, Séverine
Licata, Luana
Lopes, Pedro
Lopez-Martin, Estrella
Mascalzoni, Deborah
Monaco, Lucia
Pérez-Jurado, Luis Alberto
Posada De la Paz, Manuel
Rambla, Jordi
Rath, Ana
Riess, Olaf
Robinson, Peter N
Salgado, David
Smedley, Damian
Spalding, Dylan
't Hoen, Peter A C
Töpf, Ana
Zaharieva, Irina
Graessner, Holm
Gut, Ivo G
Lochmüller, Hanns
Beltran, Sergi
author2_role author
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author
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author
author
author
author
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author
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dc.contributor.none.fl_str_mv Unión Europea. Comisión Europea. H2020
Unión Europea. Comisión Europea. 7 Programa Marco
Instituto de Salud Carlos III
Instituto Nacional de Bioinformatica (España)
Unión Europea. Fondo Europeo de Desarrollo Regional (FEDER/ERDF)
Government of Catalonia (España)
Gobierno de Navarra (España)
NIH - National Institute of Child Health and Human Development (NICHD) (Estados Unidos)
Ministerio de Economía, Industria y Competitividad (España)
Ministerio de Ciencia e Innovación. Centro de Excelencia Severo Ochoa (España)
Canadian Institutes of Health Research

dc.subject.none.fl_str_mv Genomics
Rare Diseases
Exome
Genetic Association Studies
Humans
Phenotype
topic Genomics
Rare Diseases
Exome
Genetic Association Studies
Humans
Phenotype
description Rare disease patients are more likely to receive a rapid molecular diagnosis nowadays thanks to the wide adoption of next-generation sequencing. However, many cases remain undiagnosed even after exome or genome analysis, because the methods used missed the molecular cause in a known gene, or a novel causative gene could not be identified and/or confirmed. To address these challenges, the RD-Connect Genome-Phenome Analysis Platform (GPAP) facilitates the collation, discovery, sharing, and analysis of standardized genome-phenome data within a collaborative environment. Authorized clinicians and researchers submit pseudonymised phenotypic profiles encoded using the Human Phenotype Ontology, and raw genomic data which is processed through a standardized pipeline. After an optional embargo period, the data are shared with other platform users, with the objective that similar cases in the system and queries from peers may help diagnose the case. Additionally, the platform enables bidirectional discovery of similar cases in other databases from the Matchmaker Exchange network. To facilitate genome-phenome analysis and interpretation by clinical researchers, the RD-Connect GPAP provides a powerful user-friendly interface and leverages tens of information sources. As a result, the resource has already helped diagnose hundreds of rare disease patients and discover new disease causing genes.
publishDate 2022
dc.date.none.fl_str_mv 2022
2022-06-01
2022
2022-06-01
2023
2023-04-24
dc.type.none.fl_str_mv research article
http://purl.org/coar/resource_type/c_2df8fbb1
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv http://hdl.handle.net/20.500.12105/15877
url http://hdl.handle.net/20.500.12105/15877
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.relation.none.fl_str_mv European Commission http://dx.doi.org/10.13039/501100000780 Horizon 2020 Framework Programme 779257
European Commission http://dx.doi.org/10.13039/501100000780 Seventh Framework Programme 305444
European Commission http://dx.doi.org/10.13039/501100000780 Horizon 2020 Framework Programme 825575
European Commission http://dx.doi.org/10.13039/501100000780 Horizon 2020 Framework Programme 676559
European Commission http://dx.doi.org/10.13039/501100000780 Seventh Framework Programme 313010
European Commission http://dx.doi.org/10.13039/501100000780 Seventh Framework Programme 305121
EC 739510 Not available
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Atribución 4.0 Internacional
http://creativecommons.org/licenses/by/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
Atribución 4.0 Internacional
http://creativecommons.org/licenses/by/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Wiley
publisher.none.fl_str_mv Wiley
dc.source.none.fl_str_mv reponame:Repisalud
instname:Instituto de Salud Carlos III (ISCIII)
instname_str Instituto de Salud Carlos III (ISCIII)
reponame_str Repisalud
collection Repisalud
repository.name.fl_str_mv
repository.mail.fl_str_mv
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spelling The RD-Connect Genome-Phenome Analysis Platform: Accelerating diagnosis, research, and gene discovery for rare diseasesLaurie, StevenPiscia, DavideMatalonga, LeslieCorvó, AlbertoFernández-Callejo, MarcosGarcia-Linares, CarlesHernández-Ferrer, CarlesLuengo, CristinaMartínez, InésPapakonstantinou, AnastasiosPicó-Amador, DanielProtasio, JoanThompson, RachelTonda, RaulBayés, MònicaBullich, GemmaCamps-Puchadas, JordiParamonov, IdaTrotta, Jean-RémiAlonso, AngelAttimonelli, MarcellaBéroud, ChristopheBros-Facer, VirginieBuske, Orion JCañada-Pallarés, AndrésFernández, José MHansson, Mats GHorvath, RitaJacobsen, Julius O BKaliyaperumal, RajaramLair-Préterre, SéverineLicata, LuanaLopes, PedroLopez-Martin, EstrellaMascalzoni, DeborahMonaco, LuciaPérez-Jurado, Luis AlbertoPosada De la Paz, ManuelRambla, JordiRath, AnaRiess, OlafRobinson, Peter NSalgado, DavidSmedley, DamianSpalding, Dylan't Hoen, Peter A CTöpf, AnaZaharieva, IrinaGraessner, HolmGut, Ivo GLochmüller, HannsBeltran, SergiGenomicsRare DiseasesExomeGenetic Association StudiesHumansPhenotypeRare disease patients are more likely to receive a rapid molecular diagnosis nowadays thanks to the wide adoption of next-generation sequencing. However, many cases remain undiagnosed even after exome or genome analysis, because the methods used missed the molecular cause in a known gene, or a novel causative gene could not be identified and/or confirmed. To address these challenges, the RD-Connect Genome-Phenome Analysis Platform (GPAP) facilitates the collation, discovery, sharing, and analysis of standardized genome-phenome data within a collaborative environment. Authorized clinicians and researchers submit pseudonymised phenotypic profiles encoded using the Human Phenotype Ontology, and raw genomic data which is processed through a standardized pipeline. After an optional embargo period, the data are shared with other platform users, with the objective that similar cases in the system and queries from peers may help diagnose the case. Additionally, the platform enables bidirectional discovery of similar cases in other databases from the Matchmaker Exchange network. To facilitate genome-phenome analysis and interpretation by clinical researchers, the RD-Connect GPAP provides a powerful user-friendly interface and leverages tens of information sources. As a result, the resource has already helped diagnose hundreds of rare disease patients and discover new disease causing genes.WileyUnión Europea. Comisión Europea. H2020Unión Europea. Comisión Europea. 7 Programa MarcoInstituto de Salud Carlos IIIInstituto Nacional de Bioinformatica (España)Unión Europea. Fondo Europeo de Desarrollo Regional (FEDER/ERDF)Government of Catalonia (España)Gobierno de Navarra (España)NIH - National Institute of Child Health and Human Development (NICHD) (Estados Unidos)Ministerio de Economía, Industria y Competitividad (España)Ministerio de Ciencia e Innovación. Centro de Excelencia Severo Ochoa (España)Canadian Institutes of Health Research20232023-04-2420222022-06-0120222022-06-01research articlehttp://purl.org/coar/resource_type/c_2df8fbb1VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/20.500.12105/15877reponame:Repisaludinstname:Instituto de Salud Carlos III (ISCIII)InglésengEuropean Commission http://dx.doi.org/10.13039/501100000780 Horizon 2020 Framework Programme 779257European Commission http://dx.doi.org/10.13039/501100000780 Seventh Framework Programme 305444European Commission http://dx.doi.org/10.13039/501100000780 Horizon 2020 Framework Programme 825575European Commission http://dx.doi.org/10.13039/501100000780 Horizon 2020 Framework Programme 676559European Commission http://dx.doi.org/10.13039/501100000780 Seventh Framework Programme 313010European Commission http://dx.doi.org/10.13039/501100000780 Seventh Framework Programme 305121EC 739510 Not availableopen accesshttp://purl.org/coar/access_right/c_abf2Atribución 4.0 Internacionalhttp://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:repisalud.isciii.es:20.500.12105/158772026-06-12T12:43:37Z
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