Protection is not always a good thing: The immune system’s impact on gene therapy

Detalhes bibliográficos
Autor(a) principal: Freitas,Martiela Vaz de
Data de Publicação: 2022
Outros Autores: Frâncio,Lariane, Haleva,Laura, Matte,Ursula da Silveira
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Genetics and Molecular Biology
Texto Completo: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572022000400101
Resumo: Abstract 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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spelling Protection is not always a good thing: The immune system’s impact on gene therapyGene deliverygene therapyimmune responseAbstract 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.Sociedade Brasileira de Genética2022-01-01info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersiontext/htmlhttp://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572022000400101Genetics and Molecular Biology v.45 n.3 suppl.1 2022reponame:Genetics and Molecular Biologyinstname:Sociedade Brasileira de Genética (SBG)instacron:SBG10.1590/1678-4685-gmb-2022-0046info:eu-repo/semantics/openAccessFreitas,Martiela Vaz deFrâncio,LarianeHaleva,LauraMatte,Ursula da Silveiraeng2022-07-11T00:00:00Zoai:scielo:S1415-47572022000400101Revistahttp://www.gmb.org.br/ONGhttps://old.scielo.br/oai/scielo-oai.php||editor@gmb.org.br1678-46851415-4757opendoar:2022-07-11T00:00Genetics and Molecular Biology - Sociedade Brasileira de Genética (SBG)false
dc.title.none.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
Gene delivery
gene therapy
immune response
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 Gene delivery
gene therapy
immune response
topic Gene delivery
gene therapy
immune response
description Abstract 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.none.fl_str_mv 2022-01-01
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572022000400101
url http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572022000400101
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 10.1590/1678-4685-gmb-2022-0046
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv text/html
dc.publisher.none.fl_str_mv Sociedade Brasileira de Genética
publisher.none.fl_str_mv Sociedade Brasileira de Genética
dc.source.none.fl_str_mv Genetics and Molecular Biology v.45 n.3 suppl.1 2022
reponame:Genetics and Molecular Biology
instname:Sociedade Brasileira de Genética (SBG)
instacron:SBG
instname_str Sociedade Brasileira de Genética (SBG)
instacron_str SBG
institution SBG
reponame_str Genetics and Molecular Biology
collection Genetics and Molecular Biology
repository.name.fl_str_mv Genetics and Molecular Biology - Sociedade Brasileira de Genética (SBG)
repository.mail.fl_str_mv ||editor@gmb.org.br
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