TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis

48 p.-7 fig.-1 graph. abst.

Detalles Bibliográficos
Autores: Palomo, Valle, Tosat-Bitrian, Carlota, Nozal, Vanesa, Nagaraj, Siranjeevi, Martín-Requero, Ángeles, Martínez Gil, Ana
Tipo de recurso: artículo
Estado:Versión aceptada para publicación
Fecha de publicación:2019
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/178631
Acceso en línea:http://hdl.handle.net/10261/178631
Access Level:acceso abierto
Palabra clave:ALS
FTLD
TDP-43
Drug discovery
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spelling TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosisPalomo, ValleTosat-Bitrian, CarlotaNozal, VanesaNagaraj, SiranjeeviMartín-Requero, ÁngelesMartínez Gil, AnaALSFTLDTDP-43Drug discovery48 p.-7 fig.-1 graph. abst.Accumulation of TDP-43 in the cytoplasm of diseased neurons is the pathological hallmark of frontotemporal dementia-TDP (FTLD-TDP) and amyotrophic lateral sclerosis (ALS), two diseases that lack efficacious medicine to prevent or to stop disease progression. The discovery of mutations in the TARDBP gene (encoding the nuclear protein known as TDP-43) in both FTLD and ALS patients provided evidence for a link between TDP-43 alterations and neurodegeneration. Our understanding of TDP-43 function has advanced profoundly in the past several years; however, its complete role and the molecular mechanisms that lead to disease are not fully understood. Here we summarize the recent studies of this protein, its relation to neurodegenerative diseases, and the therapeutic strategies for restoring its homeostasis with small molecules. Finally, we briefly discuss the available cellular and animal models that help to shed light on TDP-43 pathology and could serve as tools for the discovery of pharmacological agents for the treatment of TDP-43-related diseases.Funding from Comunidad de Madrid (B2017/BMD3813 ELA-Madrid), MINECO (SAF2016-76693-R), ISCiii (CIBERNED, CB18/05/00040), and the La Caixa Junior Leader retaining program is acknowledged. V.N. holds a predoctoral FPU grant (FPU16/04466). S.N. was supported by a stipend from the Bio4Med Program (MarieSklodowska-Curie Cofound Grant Agreement 665735).Peer reviewedAmerican Chemical SocietyComunidad de MadridMinisterio de Economía y Competitividad (España)Instituto de Salud Carlos IIILa CaixaEuropean CommissionPalomo, Valle [0000-0002-1473-4086]Nagaraj, Siranjeevi [0000-0001-8132-491X]Martín-Requero, Ángeles [0000-0002-3416-9440]Martínez Gil, Ana [0000-0002-2707-8110]Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]201920192019info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Postprintinfo:eu-repo/semantics/acceptedVersionhttp://hdl.handle.net/10261/178631reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Inglés#PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE#info:eu-repo/grantAgreement/MINECO/Plan Estatal de Investigación Científica y Técnica y de Innovación 2013-2016/SAF2016-76693-Rinfo:eu-repo/grantAgreement/EC/H2020/665735https://doi.org/10.1021/acschemneuro.9b00026Síinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/1786312026-05-22T06:33:51Z
dc.title.none.fl_str_mv TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
title TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
spellingShingle TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
Palomo, Valle
ALS
FTLD
TDP-43
Drug discovery
title_short TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
title_full TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
title_fullStr TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
title_full_unstemmed TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
title_sort TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
dc.creator.none.fl_str_mv Palomo, Valle
Tosat-Bitrian, Carlota
Nozal, Vanesa
Nagaraj, Siranjeevi
Martín-Requero, Ángeles
Martínez Gil, Ana
author Palomo, Valle
author_facet Palomo, Valle
Tosat-Bitrian, Carlota
Nozal, Vanesa
Nagaraj, Siranjeevi
Martín-Requero, Ángeles
Martínez Gil, Ana
author_role author
author2 Tosat-Bitrian, Carlota
Nozal, Vanesa
Nagaraj, Siranjeevi
Martín-Requero, Ángeles
Martínez Gil, Ana
author2_role author
author
author
author
author
dc.contributor.none.fl_str_mv Comunidad de Madrid
Ministerio de Economía y Competitividad (España)
Instituto de Salud Carlos III
La Caixa
European Commission
Palomo, Valle [0000-0002-1473-4086]
Nagaraj, Siranjeevi [0000-0001-8132-491X]
Martín-Requero, Ángeles [0000-0002-3416-9440]
Martínez Gil, Ana [0000-0002-2707-8110]
Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]
dc.subject.none.fl_str_mv ALS
FTLD
TDP-43
Drug discovery
topic ALS
FTLD
TDP-43
Drug discovery
description 48 p.-7 fig.-1 graph. abst.
publishDate 2019
dc.date.none.fl_str_mv 2019
2019
2019
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Postprint
info:eu-repo/semantics/acceptedVersion
format article
status_str acceptedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/178631
url http://hdl.handle.net/10261/178631
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv #PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
info:eu-repo/grantAgreement/MINECO/Plan Estatal de Investigación Científica y Técnica y de Innovación 2013-2016/SAF2016-76693-R
info:eu-repo/grantAgreement/EC/H2020/665735
https://doi.org/10.1021/acschemneuro.9b00026
Sí
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv American Chemical Society
publisher.none.fl_str_mv American Chemical Society
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
collection DIGITAL.CSIC. Repositorio Institucional del CSIC
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15,811543

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