A role for adeno-associated viral vectors in gene therapy
Autor(a) principal: | |
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Data de Publicação: | 2008 |
Outros Autores: | |
Tipo de documento: | Artigo |
Idioma: | eng |
Título da fonte: | Repositório Institucional da UFRGS |
Texto Completo: | http://hdl.handle.net/10183/23403 |
Resumo: | Gene therapy constitutes a therapeutic intervention based on modification of the genetic material of living cells, by correcting genetic defects or overexpressing therapeutic proteins. The success of gene therapy protocols depends on the availability of therapeutically suitable genes, appropriate gene delivery systems and proof of safety and efficacy. Recent advances on the development of gene delivery systems, particularly on viral vectors engineering and improved gene regulatory systems, have led to marked progress in this field. Although the available vector systems can successfully transfer genes into cells, the ideal delivery vehicle has not been found. In this context, adeno-associated virus vectors (AAV) are arising as a promising tool for a wide range of applications, due to a combination of characteristics such as lack of pathogenicity and immunogenicity, wide range of cell tropism and long-term gene expression. Since its isolation, the biological properties of the adeno-associated virus have been increasingly understood, improving our ability to manipulate and use it as a safe and efficient gene therapy vector of wide spectrum. In this work, we review the bases of gene therapy, main types of gene transfer systems and basic properties and use of AAV vectors. |
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Coura, Renata dos SantosNardi, Nance Beyer2010-06-05T04:17:28Z20081415-4757http://hdl.handle.net/10183/23403000630743Gene therapy constitutes a therapeutic intervention based on modification of the genetic material of living cells, by correcting genetic defects or overexpressing therapeutic proteins. The success of gene therapy protocols depends on the availability of therapeutically suitable genes, appropriate gene delivery systems and proof of safety and efficacy. Recent advances on the development of gene delivery systems, particularly on viral vectors engineering and improved gene regulatory systems, have led to marked progress in this field. Although the available vector systems can successfully transfer genes into cells, the ideal delivery vehicle has not been found. In this context, adeno-associated virus vectors (AAV) are arising as a promising tool for a wide range of applications, due to a combination of characteristics such as lack of pathogenicity and immunogenicity, wide range of cell tropism and long-term gene expression. Since its isolation, the biological properties of the adeno-associated virus have been increasingly understood, improving our ability to manipulate and use it as a safe and efficient gene therapy vector of wide spectrum. In this work, we review the bases of gene therapy, main types of gene transfer systems and basic properties and use of AAV vectors.application/pdfengGenetics and molecular biology. Ribeirão Preto. Vol. 31, n. 1 (Mar. 2008), p. 1-11GenéticaTerapia gênicaAdenovírusAdeno-associated virusAAV-based recombinant vectorsGene therapyA role for adeno-associated viral vectors in 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:UFRGSORIGINAL000630743.pdf000630743.pdfTexto completo (inglês)application/pdf165767http://www.lume.ufrgs.br/bitstream/10183/23403/1/000630743.pdf946a8167b4e23955600c6e245118509bMD51TEXT000630743.pdf.txt000630743.pdf.txtExtracted Texttext/plain59294http://www.lume.ufrgs.br/bitstream/10183/23403/2/000630743.pdf.txt1d5b4345f25a39e9a3cacfd5371cb161MD52THUMBNAIL000630743.pdf.jpg000630743.pdf.jpgGenerated Thumbnailimage/jpeg1874http://www.lume.ufrgs.br/bitstream/10183/23403/3/000630743.pdf.jpg9332e76c4230d679b65c049a9df53a6dMD5310183/234032018-10-09 08:00:39.2oai:www.lume.ufrgs.br:10183/23403Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2018-10-09T11:00:39Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false |
dc.title.pt_BR.fl_str_mv |
A role for adeno-associated viral vectors in gene therapy |
title |
A role for adeno-associated viral vectors in gene therapy |
spellingShingle |
A role for adeno-associated viral vectors in gene therapy Coura, Renata dos Santos Genética Terapia gênica Adenovírus Adeno-associated virus AAV-based recombinant vectors Gene therapy |
title_short |
A role for adeno-associated viral vectors in gene therapy |
title_full |
A role for adeno-associated viral vectors in gene therapy |
title_fullStr |
A role for adeno-associated viral vectors in gene therapy |
title_full_unstemmed |
A role for adeno-associated viral vectors in gene therapy |
title_sort |
A role for adeno-associated viral vectors in gene therapy |
author |
Coura, Renata dos Santos |
author_facet |
Coura, Renata dos Santos Nardi, Nance Beyer |
author_role |
author |
author2 |
Nardi, Nance Beyer |
author2_role |
author |
dc.contributor.author.fl_str_mv |
Coura, Renata dos Santos Nardi, Nance Beyer |
dc.subject.por.fl_str_mv |
Genética Terapia gênica Adenovírus |
topic |
Genética Terapia gênica Adenovírus Adeno-associated virus AAV-based recombinant vectors Gene therapy |
dc.subject.eng.fl_str_mv |
Adeno-associated virus AAV-based recombinant vectors Gene therapy |
description |
Gene therapy constitutes a therapeutic intervention based on modification of the genetic material of living cells, by correcting genetic defects or overexpressing therapeutic proteins. The success of gene therapy protocols depends on the availability of therapeutically suitable genes, appropriate gene delivery systems and proof of safety and efficacy. Recent advances on the development of gene delivery systems, particularly on viral vectors engineering and improved gene regulatory systems, have led to marked progress in this field. Although the available vector systems can successfully transfer genes into cells, the ideal delivery vehicle has not been found. In this context, adeno-associated virus vectors (AAV) are arising as a promising tool for a wide range of applications, due to a combination of characteristics such as lack of pathogenicity and immunogenicity, wide range of cell tropism and long-term gene expression. Since its isolation, the biological properties of the adeno-associated virus have been increasingly understood, improving our ability to manipulate and use it as a safe and efficient gene therapy vector of wide spectrum. In this work, we review the bases of gene therapy, main types of gene transfer systems and basic properties and use of AAV vectors. |
publishDate |
2008 |
dc.date.issued.fl_str_mv |
2008 |
dc.date.accessioned.fl_str_mv |
2010-06-05T04:17:28Z |
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 |
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http://hdl.handle.net/10183/23403 |
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1415-4757 |
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000630743 |
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http://hdl.handle.net/10183/23403 |
dc.language.iso.fl_str_mv |
eng |
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eng |
dc.relation.ispartof.pt_BR.fl_str_mv |
Genetics and molecular biology. Ribeirão Preto. Vol. 31, n. 1 (Mar. 2008), p. 1-11 |
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info:eu-repo/semantics/openAccess |
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openAccess |
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