Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases

Detalhes bibliográficos
Autor(a) principal: Poletto, Édina
Data de Publicação: 2019
Outros Autores: Pasqualim, Gabriela, Giugliani, Roberto, Matte, Ursula da Silveira, Baldo, Guilherme
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UFRGS
Texto Completo: http://hdl.handle.net/10183/201049
Resumo: Lysosomal storage diseases (LSDs) are inherited conditions caused by impaired lysosomal function and consequent substrate storage, leading to a range of clinical manifestations, including cardiovascular disease. This may lead to significant symptoms and even cardiac failure, which is an important cause of death among patients. Currently available treatments do not completely correct cardiac involvement in the LSDs. Gene therapy has been tested as a therapeutic alternative with promising results for the heart disease. In this review, we present the results of different approaches of gene therapy for LSDs, mainly in animal models, and its effects in the heart, focusing on protocols with cardiac functional analysis.
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spelling Poletto, ÉdinaPasqualim, GabrielaGiugliani, RobertoMatte, Ursula da SilveiraBaldo, Guilherme2019-10-26T03:50:36Z20191415-4757http://hdl.handle.net/10183/201049001101792Lysosomal storage diseases (LSDs) are inherited conditions caused by impaired lysosomal function and consequent substrate storage, leading to a range of clinical manifestations, including cardiovascular disease. This may lead to significant symptoms and even cardiac failure, which is an important cause of death among patients. Currently available treatments do not completely correct cardiac involvement in the LSDs. Gene therapy has been tested as a therapeutic alternative with promising results for the heart disease. In this review, we present the results of different approaches of gene therapy for LSDs, mainly in animal models, and its effects in the heart, focusing on protocols with cardiac functional analysis.application/pdfengGenetics and molecular biology. Ribeirão Preto. Vol. 42, n. 1, suppl. 1 (2019), p. 261-285Terapia genéticaDoenças cardiovascularesDoença de depósito lisossomalDoença de depósito de glicogênioLysosomal storage diseaseGene therapyCardiovascular diseaseAnimal modelsHeart.Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseasesinfo:eu-repo/semantics/articleinfo:eu-repo/semantics/otherinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/openAccessreponame:Repositório Institucional da UFRGSinstname:Universidade Federal do Rio Grande do Sul (UFRGS)instacron:UFRGSTEXT001101792.pdf.txt001101792.pdf.txtExtracted Texttext/plain112815http://www.lume.ufrgs.br/bitstream/10183/201049/2/001101792.pdf.txtba6be1d8c728af17265f0ea8b1788f37MD52ORIGINAL001101792.pdfTexto completo (inglês)application/pdf982415http://www.lume.ufrgs.br/bitstream/10183/201049/1/001101792.pdf96ab80f4955ec963f3615ef94539ada5MD5110183/2010492023-11-15 04:26:23.841751oai:www.lume.ufrgs.br:10183/201049Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2023-11-15T06:26:23Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false
dc.title.pt_BR.fl_str_mv Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
title Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
spellingShingle Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
Poletto, Édina
Terapia genética
Doenças cardiovasculares
Doença de depósito lisossomal
Doença de depósito de glicogênio
Lysosomal storage disease
Gene therapy
Cardiovascular disease
Animal models
Heart.
title_short Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
title_full Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
title_fullStr Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
title_full_unstemmed Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
title_sort Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
author Poletto, Édina
author_facet Poletto, Édina
Pasqualim, Gabriela
Giugliani, Roberto
Matte, Ursula da Silveira
Baldo, Guilherme
author_role author
author2 Pasqualim, Gabriela
Giugliani, Roberto
Matte, Ursula da Silveira
Baldo, Guilherme
author2_role author
author
author
author
dc.contributor.author.fl_str_mv Poletto, Édina
Pasqualim, Gabriela
Giugliani, Roberto
Matte, Ursula da Silveira
Baldo, Guilherme
dc.subject.por.fl_str_mv Terapia genética
Doenças cardiovasculares
Doença de depósito lisossomal
Doença de depósito de glicogênio
topic Terapia genética
Doenças cardiovasculares
Doença de depósito lisossomal
Doença de depósito de glicogênio
Lysosomal storage disease
Gene therapy
Cardiovascular disease
Animal models
Heart.
dc.subject.eng.fl_str_mv Lysosomal storage disease
Gene therapy
Cardiovascular disease
Animal models
Heart.
description Lysosomal storage diseases (LSDs) are inherited conditions caused by impaired lysosomal function and consequent substrate storage, leading to a range of clinical manifestations, including cardiovascular disease. This may lead to significant symptoms and even cardiac failure, which is an important cause of death among patients. Currently available treatments do not completely correct cardiac involvement in the LSDs. Gene therapy has been tested as a therapeutic alternative with promising results for the heart disease. In this review, we present the results of different approaches of gene therapy for LSDs, mainly in animal models, and its effects in the heart, focusing on protocols with cardiac functional analysis.
publishDate 2019
dc.date.accessioned.fl_str_mv 2019-10-26T03:50:36Z
dc.date.issued.fl_str_mv 2019
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
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dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
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dc.identifier.uri.fl_str_mv http://hdl.handle.net/10183/201049
dc.identifier.issn.pt_BR.fl_str_mv 1415-4757
dc.identifier.nrb.pt_BR.fl_str_mv 001101792
identifier_str_mv 1415-4757
001101792
url http://hdl.handle.net/10183/201049
dc.language.iso.fl_str_mv eng
language eng
dc.relation.ispartof.pt_BR.fl_str_mv Genetics and molecular biology. Ribeirão Preto. Vol. 42, n. 1, suppl. 1 (2019), p. 261-285
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Repositório Institucional da UFRGS
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instacron:UFRGS
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instacron_str UFRGS
institution UFRGS
reponame_str Repositório Institucional da UFRGS
collection Repositório Institucional da UFRGS
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