Protection is not always a good thing: the immune system’s impact on gene therapy
Autor(a) principal: | |
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Data de Publicação: | 2022 |
Outros Autores: | , , |
Tipo de documento: | Artigo |
Idioma: | eng |
Título da fonte: | Repositório Institucional da UFRGS |
Texto Completo: | http://hdl.handle.net/10183/274348 |
Resumo: | There are many clinical trials underway for the development of gene therapies, and some have resulted in gene therapy products being commercially approved already. Significant progress was made to develop safer and more effective strategies to deliver and regulate genetic products. An unsolved aspect is the immune system, which can affect the efficiency of gene therapy in different ways. Here we present an overview of approved gene therapy products and the immune response elicited by gene delivery systems. These include responses against the vector or its content after delivery and against the product of the corrected gene. Strategies to overcome the hurdles include hiding the vector or/and the transgene product from the immune system and hiding the immune system from the vector/transgene product. Combining different strategies, such as patient screening and intelligent vector design, gene therapy is set to make a difference in the life of patients with severe genetic diseases. |
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Freitas, Martiela Vaz deFrâncio, LarianeHaleva, LauraMatte, Ursula da Silveira2024-03-28T06:25:17Z20221415-4757http://hdl.handle.net/10183/274348001161965There are many clinical trials underway for the development of gene therapies, and some have resulted in gene therapy products being commercially approved already. Significant progress was made to develop safer and more effective strategies to deliver and regulate genetic products. An unsolved aspect is the immune system, which can affect the efficiency of gene therapy in different ways. Here we present an overview of approved gene therapy products and the immune response elicited by gene delivery systems. These include responses against the vector or its content after delivery and against the product of the corrected gene. Strategies to overcome the hurdles include hiding the vector or/and the transgene product from the immune system and hiding the immune system from the vector/transgene product. Combining different strategies, such as patient screening and intelligent vector design, gene therapy is set to make a difference in the life of patients with severe genetic diseases.application/pdfengGenetics and molecular biology. Ribeirão Preto. Vol. 45, no. 3, supl. 1 (2022), e20220046 , 18 p.Terapia gênicaResposta imuneGene deliveryProtection is not always a good thing: the immune system’s impact on gene therapyinfo: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:UFRGSTEXT001161965.pdf.txt001161965.pdf.txtExtracted Texttext/plain89227http://www.lume.ufrgs.br/bitstream/10183/274348/2/001161965.pdf.txtfb167c60305030c0e1c6f94f3bd99f03MD52ORIGINAL001161965.pdfTexto completo (inglês)application/pdf4579456http://www.lume.ufrgs.br/bitstream/10183/274348/1/001161965.pdf513f2f9b6a5092c892b4941b56c610c4MD5110183/2743482024-03-29 06:19:38.381334oai:www.lume.ufrgs.br:10183/274348Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2024-03-29T09:19:38Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false |
dc.title.pt_BR.fl_str_mv |
Protection is not always a good thing: the immune system’s impact on gene therapy |
title |
Protection is not always a good thing: the immune system’s impact on gene therapy |
spellingShingle |
Protection is not always a good thing: the immune system’s impact on gene therapy Freitas, Martiela Vaz de Terapia gênica Resposta imune Gene delivery |
title_short |
Protection is not always a good thing: the immune system’s impact on gene therapy |
title_full |
Protection is not always a good thing: the immune system’s impact on gene therapy |
title_fullStr |
Protection is not always a good thing: the immune system’s impact on gene therapy |
title_full_unstemmed |
Protection is not always a good thing: the immune system’s impact on gene therapy |
title_sort |
Protection is not always a good thing: the immune system’s impact on gene therapy |
author |
Freitas, Martiela Vaz de |
author_facet |
Freitas, Martiela Vaz de Frâncio, Lariane Haleva, Laura Matte, Ursula da Silveira |
author_role |
author |
author2 |
Frâncio, Lariane Haleva, Laura Matte, Ursula da Silveira |
author2_role |
author author author |
dc.contributor.author.fl_str_mv |
Freitas, Martiela Vaz de Frâncio, Lariane Haleva, Laura Matte, Ursula da Silveira |
dc.subject.por.fl_str_mv |
Terapia gênica Resposta imune |
topic |
Terapia gênica Resposta imune Gene delivery |
dc.subject.eng.fl_str_mv |
Gene delivery |
description |
There are many clinical trials underway for the development of gene therapies, and some have resulted in gene therapy products being commercially approved already. Significant progress was made to develop safer and more effective strategies to deliver and regulate genetic products. An unsolved aspect is the immune system, which can affect the efficiency of gene therapy in different ways. Here we present an overview of approved gene therapy products and the immune response elicited by gene delivery systems. These include responses against the vector or its content after delivery and against the product of the corrected gene. Strategies to overcome the hurdles include hiding the vector or/and the transgene product from the immune system and hiding the immune system from the vector/transgene product. Combining different strategies, such as patient screening and intelligent vector design, gene therapy is set to make a difference in the life of patients with severe genetic diseases. |
publishDate |
2022 |
dc.date.issued.fl_str_mv |
2022 |
dc.date.accessioned.fl_str_mv |
2024-03-28T06:25:17Z |
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/10183/274348 |
dc.identifier.issn.pt_BR.fl_str_mv |
1415-4757 |
dc.identifier.nrb.pt_BR.fl_str_mv |
001161965 |
identifier_str_mv |
1415-4757 001161965 |
url |
http://hdl.handle.net/10183/274348 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.ispartof.pt_BR.fl_str_mv |
Genetics and molecular biology. Ribeirão Preto. Vol. 45, no. 3, supl. 1 (2022), e20220046 , 18 p. |
dc.rights.driver.fl_str_mv |
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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Universidade Federal do Rio Grande do Sul (UFRGS) |
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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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http://www.lume.ufrgs.br/bitstream/10183/274348/2/001161965.pdf.txt http://www.lume.ufrgs.br/bitstream/10183/274348/1/001161965.pdf |
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