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Young-onset rapidly progressive ALS associated with heterozygous FUS mutation

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Resumo:We report a 36-years-old Cape Verdean man who presented with respiratory insufficiency due to rapidly progressive sporadic amyotrophic lateral sclerosis (ALS), in whom FUS mutation c.1551C > G (p.Hist517Gln) in heterozygosity was identified, a finding previously described as non-pathogenic. The only previous report on this mutation was in a family from Cape Verde in which four members developed ALS; all were homozygous for the mutation. This case shows that this FUS mutation presents a highly variable penetrance and expressivity.
Autores principais:Gromicho, Marta
Outros Autores:Oliveira Santos, Miguel; Pinto, Anabela; Pronto Laborinho, Ana Catarina; Carvalho, Mamede
Assunto:Amyotrophic lateral sclerosis Cape Verde FUS mutation Dominant transmission Young-onset
Ano:2017
País:Portugal
Tipo de documento:artigo
Tipo de acesso:acesso restrito
Instituição associada:Universidade de Lisboa
Idioma:inglês
Origem:Repositório da Universidade de Lisboa
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author Gromicho, Marta
author2 Oliveira Santos, Miguel
Pinto, Anabela
Pronto Laborinho, Ana Catarina
Carvalho, Mamede
author2_role author
author
author
author
author_facet Gromicho, Marta
Oliveira Santos, Miguel
Pinto, Anabela
Pronto Laborinho, Ana Catarina
Carvalho, Mamede
author_role author
contributor_name_str_mv Repositório Científico de Acesso Aberto da ULisboa
country_str PT
creators_json_txt [{\"Person.name\":\"Gromicho, Marta\",\"Person.identifier.orcid\":\"0000-0003-2111-4579\"},{\"Person.name\":\"Oliveira Santos, Miguel\",\"Person.identifier.orcid\":\"0000-0002-8290-0410\"},{\"Person.name\":\"Pinto, Anabela\",\"Person.identifier.orcid\":\"0000-0001-8139-6905\"},{\"Person.name\":\"Pronto Laborinho, Ana Catarina\",\"Person.identifier.orcid\":\"0000-0002-2816-9698\"},{\"Person.name\":\"Carvalho, Mamede\",\"Person.identifier.orcid\":\"0000-0001-7556-0158\"}]
datacite.contributors.contributor.contributorName.fl_str_mv Repositório Científico de Acesso Aberto da ULisboa
datacite.creators.creator.creatorName.fl_str_mv Gromicho, Marta
Oliveira Santos, Miguel
Pinto, Anabela
Pronto Laborinho, Ana Catarina
Carvalho, Mamede
datacite.date.Accepted.fl_str_mv 2017-01-01T00:00:00Z
datacite.date.available.fl_str_mv 2022-09-08T15:10:55Z
datacite.date.embargoed.fl_str_mv 2022-09-08T15:10:55Z
datacite.rights.fl_str_mv http://purl.org/coar/access_right/c_16ec
datacite.subjects.subject.fl_str_mv Amyotrophic lateral sclerosis
Cape Verde
FUS mutation
Dominant transmission
Young-onset
datacite.titles.title.fl_str_mv Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
dc.contributor.none.fl_str_mv Repositório Científico de Acesso Aberto da ULisboa
dc.creator.none.fl_str_mv Gromicho, Marta
Oliveira Santos, Miguel
Pinto, Anabela
Pronto Laborinho, Ana Catarina
Carvalho, Mamede
dc.date.Accepted.fl_str_mv 2017-01-01T00:00:00Z
dc.date.available.fl_str_mv 2022-09-08T15:10:55Z
dc.date.embargoed.fl_str_mv 2022-09-08T15:10:55Z
dc.format.none.fl_str_mv application/pdf
dc.identifier.none.fl_str_mv http://hdl.handle.net/10451/54391
dc.language.none.fl_str_mv eng
dc.publisher.none.fl_str_mv Taylor & Francis
dc.rights.none.fl_str_mv http://purl.org/coar/access_right/c_16ec
dc.subject.none.fl_str_mv Amyotrophic lateral sclerosis
Cape Verde
FUS mutation
Dominant transmission
Young-onset
dc.title.fl_str_mv Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
dc.type.none.fl_str_mv http://purl.org/coar/resource_type/c_6501
description We report a 36-years-old Cape Verdean man who presented with respiratory insufficiency due to rapidly progressive sporadic amyotrophic lateral sclerosis (ALS), in whom FUS mutation c.1551C > G (p.Hist517Gln) in heterozygosity was identified, a finding previously described as non-pathogenic. The only previous report on this mutation was in a family from Cape Verde in which four members developed ALS; all were homozygous for the mutation. This case shows that this FUS mutation presents a highly variable penetrance and expressivity.
dirty 0
eu_rights_str_mv restrictedAccess
format article
fulltext.url.fl_str_mv https://repositorio.ulisboa.pt/bitstreams/16d57b65-1f16-44cb-9633-43edc1ed7596/download
funding.funder.alternateName_str_mv FCT
funding.funder.identifier_str_mv http://doi.org/10.13039/501100001871
funding.funder.name_str_mv Fundação para a Ciência e a Tecnologia
funding.name_str_mv 3599-PPCDT
id ul_61d76049542ea2fe58cd68c35cb04061
identifier.url.fl_str_mv http://hdl.handle.net/10451/54391
instacron_str ul
institution Universidade de Lisboa
instname_str Universidade de Lisboa
language eng
network_acronym_str ul
network_name_str Repositório da Universidade de Lisboa
oai_identifier_str oai:repositorio.ulisboa.pt:10451/54391
organization_str_mv urn:organizationAcronym:ul
person_str_mv Gromicho, Marta
Gromicho, Marta
https://www.ciencia-id.pt/EA1D-1979-5C3A
EA1D-1979-5C3A
http://orcid.org/0000-0003-2111-4579
0000-0003-2111-4579
Oliveira Santos, Miguel
Oliveira Santos, Miguel
https://www.ciencia-id.pt/B511-DD32-12D7
B511-DD32-12D7
http://orcid.org/0000-0002-8290-0410
0000-0002-8290-0410
Pinto, Anabela
Pinto, Anabela
http://orcid.org/0000-0001-8139-6905
0000-0001-8139-6905
Pronto Laborinho, Ana Catarina
Pronto Laborinho, Ana Catarina
https://www.ciencia-id.pt/891E-679B-B2DE
891E-679B-B2DE
http://orcid.org/0000-0002-2816-9698
0000-0002-2816-9698
Carvalho, Mamede
Carvalho, Mamede
http://orcid.org/0000-0001-7556-0158
0000-0001-7556-0158
publishDate 2017
publisher.none.fl_str_mv Taylor & Francis
reponame_str Repositório da Universidade de Lisboa
repository_id_str urn:repositoryAcronym:ul
service_str_mv urn:repositoryAcronym:ul
spelling engTaylor & Francispt_PTWe report a 36-years-old Cape Verdean man who presented with respiratory insufficiency due to rapidly progressive sporadic amyotrophic lateral sclerosis (ALS), in whom FUS mutation c.1551C > G (p.Hist517Gln) in heterozygosity was identified, a finding previously described as non-pathogenic. The only previous report on this mutation was in a family from Cape Verde in which four members developed ALS; all were homozygous for the mutation. This case shows that this FUS mutation presents a highly variable penetrance and expressivity.application/pdfpt_PTYoung-onset rapidly progressive ALS associated with heterozygous FUS mutationPersonalGromicho, MartaDSpacehttp://dspace.org/items/c6f0ac72-fdd9-499d-87e2-f13aa1545565DSpacehttp://dspace.org/items/c6f0ac72-fdd9-499d-87e2-f13aa1545565SilvaMarta Luísa Gromicho MorgadoCiência IDhttps://www.ciencia-id.ptEA1D-1979-5C3AORCIDhttp://orcid.org0000-0003-2111-4579Scopus Author IDhttps://www.scopus.com6506583274PersonalOliveira Santos, MiguelDSpacehttp://dspace.org/items/eab02009-54a9-4518-8b22-9426433cea69DSpacehttp://dspace.org/items/eab02009-54a9-4518-8b22-9426433cea69Oliveira SantosMiguelCiência IDhttps://www.ciencia-id.ptB511-DD32-12D7ORCIDhttp://orcid.org0000-0002-8290-0410Scopus Author IDhttps://www.scopus.com57217474349PersonalPinto, AnabelaDSpacehttp://dspace.org/items/ac6330ee-bc35-45e5-9d9c-357de0c41f0bDSpacehttp://dspace.org/items/ac6330ee-bc35-45e5-9d9c-357de0c41f0bPintoAnabelaORCIDhttp://orcid.org0000-0001-8139-6905Scopus Author IDhttps://www.scopus.com7402306398PersonalPronto Laborinho, Ana CatarinaDSpacehttp://dspace.org/items/3726b674-773c-4838-b02e-da9bc38ccbe6DSpacehttp://dspace.org/items/3726b674-773c-4838-b02e-da9bc38ccbe6Pronto LaborinhoAna CatarinaCiência IDhttps://www.ciencia-id.pt891E-679B-B2DEORCIDhttp://orcid.org0000-0002-2816-9698Scopus Author IDhttps://www.scopus.com56177475200PersonalCarvalho, MamedeDSpacehttp://dspace.org/items/dd7f55d4-c2b5-4fd2-9bd1-a9542a62f58fDSpacehttp://dspace.org/items/dd7f55d4-c2b5-4fd2-9bd1-a9542a62f58fde CarvalhoMamedeORCIDhttp://orcid.org0000-0001-7556-0158Scopus Author IDhttps://www.scopus.com7101893769HostingInstitutionOrganizationalRepositório Científico de Acesso Aberto da ULisboae-mailmailto:repositorio@reitoria.ulisboa.ptrepositorio@reitoria.ulisboa.ptISSNIsPartOf2167-8421DOIIsPartOf10.1080/21678421.2017.12997622022-09-08T15:10:55Z20172017-01-01T00:00:00ZHandlehttp://hdl.handle.net/10451/54391http://purl.org/coar/access_right/c_16ecrestricted accessAmyotrophic lateral sclerosisCape VerdeFUS mutationDominant transmissionYoung-onset320225 bytesFundação para a Ciência e a TecnologiaONTology-based Web Database for Understanding Amyotrophic Lateral Sclerosis<br>3599-PPCDTCrossref Funder IDhttp://doi.org/10.13039/501100001871literaturehttp://purl.org/coar/resource_type/c_6501journal articlehttp://purl.org/coar/access_right/c_16ecapplication/pdffulltexthttps://repositorio.ulisboa.pt/bitstreams/16d57b65-1f16-44cb-9633-43edc1ed7596/downloadAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration185-6451453
spellingShingle Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
Gromicho, Marta
Amyotrophic lateral sclerosis
Cape Verde
FUS mutation
Dominant transmission
Young-onset
status SINGLETON
subject.fl_str_mv Amyotrophic lateral sclerosis
Cape Verde
FUS mutation
Dominant transmission
Young-onset
title Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
title_full Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
title_fullStr Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
title_full_unstemmed Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
title_short Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
title_sort Young-onset rapidly progressive ALS associated with heterozygous FUS mutation
topic Amyotrophic lateral sclerosis
Cape Verde
FUS mutation
Dominant transmission
Young-onset
topic_facet Amyotrophic lateral sclerosis
Cape Verde
FUS mutation
Dominant transmission
Young-onset
url http://hdl.handle.net/10451/54391
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