Gene Therapy: a new strategy for the treatment of diseases

Detalhes bibliográficos
Autor(a) principal: D. da Silva, Cláudia
Data de Publicação: 2022
Outros Autores: Silveira Matte, Úrsula, Giugliani, Roberto
Tipo de documento: Artigo
Idioma: por
Título da fonte: Clinical and Biomedical Research
Texto Completo: https://seer.ufrgs.br/index.php/hcpa/article/view/126046
Resumo: Gene therapy is based on the simple concept that a genetic disease can be corrected by replacing the defective gene. Early trials encountered unforeseen complications and much of the initial enthusiasm dismayed. This paper is a review about different kinds of vectors, their advantages and disadvantages. Strategies for different target tissues is also discussed. Special interest is on strategies for the treatment of cancer, since nowadays this is the area of most clinical trials. The reason is that cancer treatment proved less complex than treatment of monogenic life-treating disorders.
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spelling Gene Therapy: a new strategy for the treatment of diseasesTerapia gênica: uma nova estratégia para o tratamento de doençasTerapia gênicavetores viraisvetores não viraiscâncerGene therapyviral vectorsnon-viral vectorscancerGene therapy is based on the simple concept that a genetic disease can be corrected by replacing the defective gene. Early trials encountered unforeseen complications and much of the initial enthusiasm dismayed. This paper is a review about different kinds of vectors, their advantages and disadvantages. Strategies for different target tissues is also discussed. Special interest is on strategies for the treatment of cancer, since nowadays this is the area of most clinical trials. The reason is that cancer treatment proved less complex than treatment of monogenic life-treating disorders.A terapia gênica está baseada no conceito de que uma doença genética pode ser corrigida pela substituição ou adição do gene defeituoso. Muita expectativa foi gerada pelos primeiros protocolos, mas o surgimento de complicações inesperadas levou a uma diminuição do entusiasmo inicial. Neste artigo, é feita uma revisão dos diferentes tipos de vetores utilizados para terapia gênica, com discussão das suas vantagens e desvantagens. Além disso, as estratégias para alguns dos principais tecidos alvo são discutidas. Especial ênfase é dada às estratégias para o tratamento do câncer, área na qual atualmente se concentram a maioria dos protocolos clínicos e que apresenta dificuldades menores do que as envolvidas no tratamento de doenças monogênicas.HCPA/FAMED/UFRGS2022-07-22info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionPeer-reviewed Article"A Convite dos Editoresapplication/pdfhttps://seer.ufrgs.br/index.php/hcpa/article/view/126046Clinical & Biomedical Research; Vol. 21 No. 3 (2001): Revista HCPAClinical and Biomedical Research; v. 21 n. 3 (2001): Revista HCPA2357-9730reponame:Clinical and Biomedical Researchinstname:Universidade Federal do Rio Grande do Sul (UFRGS)instacron:UFRGSporhttps://seer.ufrgs.br/index.php/hcpa/article/view/126046/85605http://creativecommons.org/licenses/by/4.0info:eu-repo/semantics/openAccessD. da Silva, Cláudia Silveira Matte, Úrsula Giugliani, Roberto 2022-07-22T13:58:14Zoai:seer.ufrgs.br:article/126046Revistahttps://www.seer.ufrgs.br/index.php/hcpaPUBhttps://seer.ufrgs.br/index.php/hcpa/oai||cbr@hcpa.edu.br2357-97302357-9730opendoar:2022-07-22T13:58:14Clinical and Biomedical Research - Universidade Federal do Rio Grande do Sul (UFRGS)false
dc.title.none.fl_str_mv Gene Therapy: a new strategy for the treatment of diseases
Terapia gênica: uma nova estratégia para o tratamento de doenças
title Gene Therapy: a new strategy for the treatment of diseases
spellingShingle Gene Therapy: a new strategy for the treatment of diseases
D. da Silva, Cláudia
Terapia gênica
vetores virais
vetores não virais
câncer
Gene therapy
viral vectors
non-viral vectors
cancer
title_short Gene Therapy: a new strategy for the treatment of diseases
title_full Gene Therapy: a new strategy for the treatment of diseases
title_fullStr Gene Therapy: a new strategy for the treatment of diseases
title_full_unstemmed Gene Therapy: a new strategy for the treatment of diseases
title_sort Gene Therapy: a new strategy for the treatment of diseases
author D. da Silva, Cláudia
author_facet D. da Silva, Cláudia
Silveira Matte, Úrsula
Giugliani, Roberto
author_role author
author2 Silveira Matte, Úrsula
Giugliani, Roberto
author2_role author
author
dc.contributor.author.fl_str_mv D. da Silva, Cláudia
Silveira Matte, Úrsula
Giugliani, Roberto
dc.subject.por.fl_str_mv Terapia gênica
vetores virais
vetores não virais
câncer
Gene therapy
viral vectors
non-viral vectors
cancer
topic Terapia gênica
vetores virais
vetores não virais
câncer
Gene therapy
viral vectors
non-viral vectors
cancer
description Gene therapy is based on the simple concept that a genetic disease can be corrected by replacing the defective gene. Early trials encountered unforeseen complications and much of the initial enthusiasm dismayed. This paper is a review about different kinds of vectors, their advantages and disadvantages. Strategies for different target tissues is also discussed. Special interest is on strategies for the treatment of cancer, since nowadays this is the area of most clinical trials. The reason is that cancer treatment proved less complex than treatment of monogenic life-treating disorders.
publishDate 2022
dc.date.none.fl_str_mv 2022-07-22
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
Peer-reviewed Article"
A Convite dos Editores
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv https://seer.ufrgs.br/index.php/hcpa/article/view/126046
url https://seer.ufrgs.br/index.php/hcpa/article/view/126046
dc.language.iso.fl_str_mv por
language por
dc.relation.none.fl_str_mv https://seer.ufrgs.br/index.php/hcpa/article/view/126046/85605
dc.rights.driver.fl_str_mv http://creativecommons.org/licenses/by/4.0
info:eu-repo/semantics/openAccess
rights_invalid_str_mv http://creativecommons.org/licenses/by/4.0
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv HCPA/FAMED/UFRGS
publisher.none.fl_str_mv HCPA/FAMED/UFRGS
dc.source.none.fl_str_mv Clinical & Biomedical Research; Vol. 21 No. 3 (2001): Revista HCPA
Clinical and Biomedical Research; v. 21 n. 3 (2001): Revista HCPA
2357-9730
reponame:Clinical and Biomedical Research
instname:Universidade Federal do Rio Grande do Sul (UFRGS)
instacron:UFRGS
instname_str Universidade Federal do Rio Grande do Sul (UFRGS)
instacron_str UFRGS
institution UFRGS
reponame_str Clinical and Biomedical Research
collection Clinical and Biomedical Research
repository.name.fl_str_mv Clinical and Biomedical Research - Universidade Federal do Rio Grande do Sul (UFRGS)
repository.mail.fl_str_mv ||cbr@hcpa.edu.br
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