Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
Autor(a) principal: | |
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Data de Publicação: | 2019 |
Outros Autores: | , , , |
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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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 info:eu-repo/semantics/other |
dc.type.status.fl_str_mv |
info:eu-repo/semantics/publishedVersion |
format |
article |
status_str |
publishedVersion |
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 |
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info:eu-repo/semantics/openAccess |
eu_rights_str_mv |
openAccess |
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application/pdf |
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reponame:Repositório Institucional da UFRGS instname:Universidade Federal do Rio Grande do Sul (UFRGS) instacron:UFRGS |
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UFRGS |
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Repositório Institucional da UFRGS |
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Repositório Institucional da UFRGS |
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