Mutations in JMJD1C are involved in Rett syndrome and intellectual disability

Purpose: autism spectrum disorders are associated with defects in social response and communication that often occur in the context of intellectual disability. Rett syndrome is one example in which epilepsy, motor impairment, and motor disturbance may co-occur. Mutations in histone demethylases are...

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Autores: Sáez, Mauricio A., Fernández Rodríguez, Juana, Moutinho, Cátia, Sanchez-Mut, Jose Vicente, Gómez, Antonio, Vidal, Enrique, Petazzi, Paolo, Szczesna, Karolina, López Serra, Paula, Lucariello, Mario, Lorden, Patricia, Delgado-Morales, Raul, Caridad, Olga J. de la, Huertas, Dori, Gelpí Buchaca, Josep Lluís, Orozco López, Modesto, López Dóriga Guerra, Adriana, Milà i Recasens, Montserrat, Pérez Jurado, Luis A., Pineda, Mercedes, Armstrong i Morón, Judith, Lázaro García, Conxi, Esteller, Manel
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2016
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:2445/113221
Acceso en línea:https://hdl.handle.net/2445/113221
Access Level:acceso abierto
Palabra clave:Síndrome de Rett
Discapacitats mentals
Autisme
Trastorns de l'espectre autista
Mutació (Biologia)
Rett syndrome
People with mental disabilities
Autism
Autism spectrum disorders
Mutation (Biology)
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spelling Mutations in JMJD1C are involved in Rett syndrome and intellectual disabilitySáez, Mauricio A.Fernández Rodríguez, JuanaMoutinho, CátiaSanchez-Mut, Jose VicenteGómez, AntonioVidal, EnriquePetazzi, PaoloSzczesna, KarolinaLópez Serra, PaulaLucariello, MarioLorden, PatriciaDelgado-Morales, RaulCaridad, Olga J. de laHuertas, DoriGelpí Buchaca, Josep LluísOrozco López, ModestoLópez Dóriga Guerra, AdrianaMilà i Recasens, MontserratPérez Jurado, Luis A.Pineda, MercedesArmstrong i Morón, JudithLázaro García, ConxiEsteller, ManelSíndrome de RettDiscapacitats mentalsAutismeTrastorns de l'espectre autistaMutació (Biologia)Rett syndromePeople with mental disabilitiesAutismAutism spectrum disordersMutation (Biology)Purpose: autism spectrum disorders are associated with defects in social response and communication that often occur in the context of intellectual disability. Rett syndrome is one example in which epilepsy, motor impairment, and motor disturbance may co-occur. Mutations in histone demethylases are known to occur in several of these syndromes. Herein, we aimed to identify whether mutations in the candidate histone demethylase JMJD1C (jumonji domain containing 1C) are implicated in these disorders. Methods: we performed the mutational and functional analysis of JMJD1C in 215 cases of autism spectrum disorders, intellectual disability, and Rett syndrome without a known genetic defect. Results: we found seven JMJD1C variants that were not present in any control sample (~ 6,000) and caused an amino acid change involving a different functional group. From these, two de novo JMJD1C germline mutations were identified in a case of Rett syndrome and in a patient with intellectual disability. The functional study of the JMJD1C mutant Rett syndrome patient demonstrated that the altered protein had abnormal subcellular localization, diminished activity to demethylate the DNA damage-response protein MDC1, and reduced binding to MECP2. We confirmed that JMJD1C protein is widely expressed in brain regions and that its depletion compromises dendritic activity. Conclusions: our findings indicate that mutations in JMJD1C contribute to the development of Rett syndrome and intellectual disability.American College of Medical Genetics and Genomics2017201720162017info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersion8 p.application/pdfhttps://hdl.handle.net/2445/113221Articles publicats en revistes (Ciències Fisiològiques)reponame:Recercat. Dipósit de la Recerca de Catalunyainstname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)InglésReproducció del document publicat a: https://doi.org/10.1038/gim.2015.100Genetics in Medicine, 2016, vol. 18, num. 4, p. 378-385https://doi.org/10.1038/gim.2015.100info:eu-repo/grantAgreement/EC/FP7/268626info:eu-repo/grantAgreement/EC/FP7/316758info:eu-repo/grantAgreement/EC/FP7/238242cc-by-nc-nd (c) Sáez, Mauricio A. et al., 2016http://creativecommons.org/licenses/by-nc-nd/3.0/esinfo:eu-repo/semantics/openAccessoai:recercat.cat:2445/1132212026-05-29T05:05:01Z
dc.title.none.fl_str_mv Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
title Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
spellingShingle Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
Sáez, Mauricio A.
Síndrome de Rett
Discapacitats mentals
Autisme
Trastorns de l'espectre autista
Mutació (Biologia)
Rett syndrome
People with mental disabilities
Autism
Autism spectrum disorders
Mutation (Biology)
title_short Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
title_full Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
title_fullStr Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
title_full_unstemmed Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
title_sort Mutations in JMJD1C are involved in Rett syndrome and intellectual disability
dc.creator.none.fl_str_mv Sáez, Mauricio A.
Fernández Rodríguez, Juana
Moutinho, Cátia
Sanchez-Mut, Jose Vicente
Gómez, Antonio
Vidal, Enrique
Petazzi, Paolo
Szczesna, Karolina
López Serra, Paula
Lucariello, Mario
Lorden, Patricia
Delgado-Morales, Raul
Caridad, Olga J. de la
Huertas, Dori
Gelpí Buchaca, Josep Lluís
Orozco López, Modesto
López Dóriga Guerra, Adriana
Milà i Recasens, Montserrat
Pérez Jurado, Luis A.
Pineda, Mercedes
Armstrong i Morón, Judith
Lázaro García, Conxi
Esteller, Manel
author Sáez, Mauricio A.
author_facet Sáez, Mauricio A.
Fernández Rodríguez, Juana
Moutinho, Cátia
Sanchez-Mut, Jose Vicente
Gómez, Antonio
Vidal, Enrique
Petazzi, Paolo
Szczesna, Karolina
López Serra, Paula
Lucariello, Mario
Lorden, Patricia
Delgado-Morales, Raul
Caridad, Olga J. de la
Huertas, Dori
Gelpí Buchaca, Josep Lluís
Orozco López, Modesto
López Dóriga Guerra, Adriana
Milà i Recasens, Montserrat
Pérez Jurado, Luis A.
Pineda, Mercedes
Armstrong i Morón, Judith
Lázaro García, Conxi
Esteller, Manel
author_role author
author2 Fernández Rodríguez, Juana
Moutinho, Cátia
Sanchez-Mut, Jose Vicente
Gómez, Antonio
Vidal, Enrique
Petazzi, Paolo
Szczesna, Karolina
López Serra, Paula
Lucariello, Mario
Lorden, Patricia
Delgado-Morales, Raul
Caridad, Olga J. de la
Huertas, Dori
Gelpí Buchaca, Josep Lluís
Orozco López, Modesto
López Dóriga Guerra, Adriana
Milà i Recasens, Montserrat
Pérez Jurado, Luis A.
Pineda, Mercedes
Armstrong i Morón, Judith
Lázaro García, Conxi
Esteller, Manel
author2_role author
author
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 Síndrome de Rett
Discapacitats mentals
Autisme
Trastorns de l'espectre autista
Mutació (Biologia)
Rett syndrome
People with mental disabilities
Autism
Autism spectrum disorders
Mutation (Biology)
topic Síndrome de Rett
Discapacitats mentals
Autisme
Trastorns de l'espectre autista
Mutació (Biologia)
Rett syndrome
People with mental disabilities
Autism
Autism spectrum disorders
Mutation (Biology)
description Purpose: autism spectrum disorders are associated with defects in social response and communication that often occur in the context of intellectual disability. Rett syndrome is one example in which epilepsy, motor impairment, and motor disturbance may co-occur. Mutations in histone demethylases are known to occur in several of these syndromes. Herein, we aimed to identify whether mutations in the candidate histone demethylase JMJD1C (jumonji domain containing 1C) are implicated in these disorders. Methods: we performed the mutational and functional analysis of JMJD1C in 215 cases of autism spectrum disorders, intellectual disability, and Rett syndrome without a known genetic defect. Results: we found seven JMJD1C variants that were not present in any control sample (~ 6,000) and caused an amino acid change involving a different functional group. From these, two de novo JMJD1C germline mutations were identified in a case of Rett syndrome and in a patient with intellectual disability. The functional study of the JMJD1C mutant Rett syndrome patient demonstrated that the altered protein had abnormal subcellular localization, diminished activity to demethylate the DNA damage-response protein MDC1, and reduced binding to MECP2. We confirmed that JMJD1C protein is widely expressed in brain regions and that its depletion compromises dendritic activity. Conclusions: our findings indicate that mutations in JMJD1C contribute to the development of Rett syndrome and intellectual disability.
publishDate 2016
dc.date.none.fl_str_mv 2016
2017
2017
2017
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/2445/113221
url https://hdl.handle.net/2445/113221
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/10.1038/gim.2015.100
Genetics in Medicine, 2016, vol. 18, num. 4, p. 378-385
https://doi.org/10.1038/gim.2015.100
info:eu-repo/grantAgreement/EC/FP7/268626
info:eu-repo/grantAgreement/EC/FP7/316758
info:eu-repo/grantAgreement/EC/FP7/238242
dc.rights.none.fl_str_mv cc-by-nc-nd (c) Sáez, Mauricio A. et al., 2016
http://creativecommons.org/licenses/by-nc-nd/3.0/es
info:eu-repo/semantics/openAccess
rights_invalid_str_mv cc-by-nc-nd (c) Sáez, Mauricio A. et al., 2016
http://creativecommons.org/licenses/by-nc-nd/3.0/es
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv 8 p.
application/pdf
dc.publisher.none.fl_str_mv American College of Medical Genetics and Genomics
publisher.none.fl_str_mv American College of Medical Genetics and Genomics
dc.source.none.fl_str_mv Articles publicats en revistes (Ciències Fisiològiques)
reponame:Recercat. Dipósit de la Recerca de Catalunya
instname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
instname_str Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
reponame_str Recercat. Dipósit de la Recerca de Catalunya
collection Recercat. Dipósit de la Recerca de Catalunya
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repository.mail.fl_str_mv
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