Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population

Intermediate CAG expansions in the gene ataxin-2 (ATXN2) are a known risk factor for ALS, but little is known about their role in FTD risk. Moreover, their contribution to the risk and phenotype of patients might vary in populations with different genetic backgrounds. The aim of this study was to as...

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Autores: Borrego Hernández, Daniel, Vázquez Costa, Juan Francisco, Domínguez Rubio, Raúl, Expósito Blázquez, Laura, Aller, Elena, Padró Miquel, Ariadna, García Casanova, Pilar, Colomina, María J., Martín Arriscado, Cristina, Osta, Rosario, Cordero Vázquez, Pilar, Esteban Pérez, Jesús, Povedano Panadés, Mónica, García Redondo, Alberto
Formato: artículo
Estado:Versión publicada
Fecha de publicación:2024
País:España
Recursos:Universidad de Barcelona
Repositorio:Dipòsit Digital de la UB
OAI Identifier:oai:diposit.ub.edu:2445/209702
Acesso em linha:https://hdl.handle.net/2445/209702
Access Level:acceso abierto
Palavra-chave:Esclerosi lateral amiotròfica
Genètica mèdica
Amyotrophic lateral sclerosis
Medical genetics
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spelling Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish PopulationBorrego Hernández, DanielVázquez Costa, Juan FranciscoDomínguez Rubio, RaúlExpósito Blázquez, LauraAller, ElenaPadró Miquel, AriadnaGarcía Casanova, PilarColomina, María J.Martín Arriscado, CristinaOsta, RosarioCordero Vázquez, PilarEsteban Pérez, JesúsPovedano Panadés, MónicaGarcía Redondo, AlbertoEsclerosi lateral amiotròficaGenètica mèdicaAmyotrophic lateral sclerosisMedical geneticsIntermediate CAG expansions in the gene ataxin-2 (ATXN2) are a known risk factor for ALS, but little is known about their role in FTD risk. Moreover, their contribution to the risk and phenotype of patients might vary in populations with different genetic backgrounds. The aim of this study was to assess the relationship of intermediate CAG expansions in ATXN2 with the risk and phenotype of ALS and FTD in the Spanish population. Repeat-primed PCR was performed in 620 ALS and 137 FTD patients in three referral centers in Spain to determine the exact number of CAG repeats. In our cohort, >= 27 CAG repeats in ATXN2 were associated with a higher risk of developing ALS (odds ratio [OR] = 2.666 [1.471-4.882]; p = 0.0013) but not FTD (odds ratio [OR] = 1.446 [0.558-3.574]; p = 0.44). Moreover, ALS patients with >= 27 CAG repeats in ATXN2 showed a shorter survival rate compared to those with <27 repeats (hazard ratio [HR] 1.74 [1.18, 2.56], p = 0.005), more frequent limb onset (odds ratio [OR] = 2.34 [1.093-4.936]; p = 0.028) and a family history of ALS (odds ratio [OR] = 2.538 [1.375-4.634]; p = 0.002). Intermediate CAG expansions of >= 27 repeats in ATXN2 are associated with ALS risk but not with FTD in the Spanish population. ALS patients carrying an intermediate expansion in ATXN2 show more frequent limb onset but a worse prognosis than those without expansions. In patients carrying C9orf72 expansions, the intermediate ATXN2 expansion might increase the penetrance and modify the phenotype.MDPI AG2024info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/2445/209702Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))reponame:Dipòsit Digital de la UBinstname:Universidad de BarcelonaInglésReproducció del document publicat a: https://doi.org/10.3390/biomedicines12020356Biomedicines, 2024, vol. 12, num. 2https://doi.org/10.3390/biomedicines12020356cc by (c) Borrego Hernández, Daniel et al, 2024http://creativecommons.org/licenses/by/3.0/es/info:eu-repo/semantics/openAccessoai:diposit.ub.edu:2445/2097022026-05-27T06:46:51Z
dc.title.none.fl_str_mv Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
title Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
spellingShingle Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
Borrego Hernández, Daniel
Esclerosi lateral amiotròfica
Genètica mèdica
Amyotrophic lateral sclerosis
Medical genetics
title_short Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
title_full Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
title_fullStr Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
title_full_unstemmed Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
title_sort Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population
dc.creator.none.fl_str_mv Borrego Hernández, Daniel
Vázquez Costa, Juan Francisco
Domínguez Rubio, Raúl
Expósito Blázquez, Laura
Aller, Elena
Padró Miquel, Ariadna
García Casanova, Pilar
Colomina, María J.
Martín Arriscado, Cristina
Osta, Rosario
Cordero Vázquez, Pilar
Esteban Pérez, Jesús
Povedano Panadés, Mónica
García Redondo, Alberto
author Borrego Hernández, Daniel
author_facet Borrego Hernández, Daniel
Vázquez Costa, Juan Francisco
Domínguez Rubio, Raúl
Expósito Blázquez, Laura
Aller, Elena
Padró Miquel, Ariadna
García Casanova, Pilar
Colomina, María J.
Martín Arriscado, Cristina
Osta, Rosario
Cordero Vázquez, Pilar
Esteban Pérez, Jesús
Povedano Panadés, Mónica
García Redondo, Alberto
author_role author
author2 Vázquez Costa, Juan Francisco
Domínguez Rubio, Raúl
Expósito Blázquez, Laura
Aller, Elena
Padró Miquel, Ariadna
García Casanova, Pilar
Colomina, María J.
Martín Arriscado, Cristina
Osta, Rosario
Cordero Vázquez, Pilar
Esteban Pérez, Jesús
Povedano Panadés, Mónica
García Redondo, Alberto
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Esclerosi lateral amiotròfica
Genètica mèdica
Amyotrophic lateral sclerosis
Medical genetics
topic Esclerosi lateral amiotròfica
Genètica mèdica
Amyotrophic lateral sclerosis
Medical genetics
description Intermediate CAG expansions in the gene ataxin-2 (ATXN2) are a known risk factor for ALS, but little is known about their role in FTD risk. Moreover, their contribution to the risk and phenotype of patients might vary in populations with different genetic backgrounds. The aim of this study was to assess the relationship of intermediate CAG expansions in ATXN2 with the risk and phenotype of ALS and FTD in the Spanish population. Repeat-primed PCR was performed in 620 ALS and 137 FTD patients in three referral centers in Spain to determine the exact number of CAG repeats. In our cohort, >= 27 CAG repeats in ATXN2 were associated with a higher risk of developing ALS (odds ratio [OR] = 2.666 [1.471-4.882]; p = 0.0013) but not FTD (odds ratio [OR] = 1.446 [0.558-3.574]; p = 0.44). Moreover, ALS patients with >= 27 CAG repeats in ATXN2 showed a shorter survival rate compared to those with <27 repeats (hazard ratio [HR] 1.74 [1.18, 2.56], p = 0.005), more frequent limb onset (odds ratio [OR] = 2.34 [1.093-4.936]; p = 0.028) and a family history of ALS (odds ratio [OR] = 2.538 [1.375-4.634]; p = 0.002). Intermediate CAG expansions of >= 27 repeats in ATXN2 are associated with ALS risk but not with FTD in the Spanish population. ALS patients carrying an intermediate expansion in ATXN2 show more frequent limb onset but a worse prognosis than those without expansions. In patients carrying C9orf72 expansions, the intermediate ATXN2 expansion might increase the penetrance and modify the phenotype.
publishDate 2024
dc.date.none.fl_str_mv 2024
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/2445/209702
url https://hdl.handle.net/2445/209702
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/10.3390/biomedicines12020356
Biomedicines, 2024, vol. 12, num. 2
https://doi.org/10.3390/biomedicines12020356
dc.rights.none.fl_str_mv cc by (c) Borrego Hernández, Daniel et al, 2024
http://creativecommons.org/licenses/by/3.0/es/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv cc by (c) Borrego Hernández, Daniel et al, 2024
http://creativecommons.org/licenses/by/3.0/es/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv MDPI AG
publisher.none.fl_str_mv MDPI AG
dc.source.none.fl_str_mv Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))
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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