Minicircle DNA vectors for gene therapy: advances and applications
Autor(a) principal: | |
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Data de Publicação: | 2015 |
Outros Autores: | , , , , , , |
Tipo de documento: | Artigo |
Idioma: | eng |
Título da fonte: | Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
Texto Completo: | http://hdl.handle.net/10400.6/4670 |
Resumo: | Introduction: Nucleic-acid-based biopharmaceuticals enclose a remarkable potential for treating debilitating or life-threatening diseases that currently remain incurable. This promising area of research envisages the creation of state-of-the-art DNA vaccines, pluripotent cells or gene-based therapies, which can be used to overcome current issues. To achieve this goal, DNA minicircles are emerging as ideal nonviral vectors due to their safety and persistent transgene expression in either quiescent or actively dividing cells. Areas covered: This review focuses on the characteristics of minicircle DNA (mcDNA) technology and the current advances in their production. The possible modifications to further improve minicircle efficacy are also emphasized and discussed in light of recent advances. As a final point, the main therapeutic applications of mcDNA are summarized, with a special focus on pluripotent stem cells production and cancer therapy. Expert opinion: Achieving in-target and persistent transgene expression is a challenging issue that is of critical importance for a successful therapeutic outcome. The use of miniaturized mcDNA cassettes with additional modifications that increase and prolong expression may contribute to an improved generation of biopharmaceuticals. The unique features of mcDNA render it an attractive alternative to overcome current technical issues and to bridge the significant gap that exists between basic research and clinical applications. |
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Minicircle DNA vectors for gene therapy: advances and applicationsCell therapyDNA-vaccinesGene therapyMinicircle DNATransgene expressionIntroduction: Nucleic-acid-based biopharmaceuticals enclose a remarkable potential for treating debilitating or life-threatening diseases that currently remain incurable. This promising area of research envisages the creation of state-of-the-art DNA vaccines, pluripotent cells or gene-based therapies, which can be used to overcome current issues. To achieve this goal, DNA minicircles are emerging as ideal nonviral vectors due to their safety and persistent transgene expression in either quiescent or actively dividing cells. Areas covered: This review focuses on the characteristics of minicircle DNA (mcDNA) technology and the current advances in their production. The possible modifications to further improve minicircle efficacy are also emphasized and discussed in light of recent advances. As a final point, the main therapeutic applications of mcDNA are summarized, with a special focus on pluripotent stem cells production and cancer therapy. Expert opinion: Achieving in-target and persistent transgene expression is a challenging issue that is of critical importance for a successful therapeutic outcome. The use of miniaturized mcDNA cassettes with additional modifications that increase and prolong expression may contribute to an improved generation of biopharmaceuticals. The unique features of mcDNA render it an attractive alternative to overcome current technical issues and to bridge the significant gap that exists between basic research and clinical applications.Taylor & FrancisuBibliorumGaspar, Vítor Manuel AbreuDiogo, Duarte Miguel de MeloCosta, Elisabete C.Moreira, AndréQueiroz, JoãoPichon, ChantalCorreia, Ilídio Joaquim SobreiraSousa, Fani2018-03-21T09:03:57Z20152015-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10400.6/4670engGaspar, V.M., De Melo-Diogo, D., Costa, E.C., Moreira, A.F., Queiroz, J.A., Pichon, C., Correia, I.J. e Sousa, F. (2015) “Minicircle DNA Vectors for Gene Therapy: Advances and Applications”, Expert Opinion on Biological Therapy, Vol. 15, pp. 353-37910.1517/14712598.2015.996544metadata only accessinfo:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-12-15T09:41:53Zoai:ubibliorum.ubi.pt:10400.6/4670Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T00:45:43.466212Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse |
dc.title.none.fl_str_mv |
Minicircle DNA vectors for gene therapy: advances and applications |
title |
Minicircle DNA vectors for gene therapy: advances and applications |
spellingShingle |
Minicircle DNA vectors for gene therapy: advances and applications Gaspar, Vítor Manuel Abreu Cell therapy DNA-vaccines Gene therapy Minicircle DNA Transgene expression |
title_short |
Minicircle DNA vectors for gene therapy: advances and applications |
title_full |
Minicircle DNA vectors for gene therapy: advances and applications |
title_fullStr |
Minicircle DNA vectors for gene therapy: advances and applications |
title_full_unstemmed |
Minicircle DNA vectors for gene therapy: advances and applications |
title_sort |
Minicircle DNA vectors for gene therapy: advances and applications |
author |
Gaspar, Vítor Manuel Abreu |
author_facet |
Gaspar, Vítor Manuel Abreu Diogo, Duarte Miguel de Melo Costa, Elisabete C. Moreira, André Queiroz, João Pichon, Chantal Correia, Ilídio Joaquim Sobreira Sousa, Fani |
author_role |
author |
author2 |
Diogo, Duarte Miguel de Melo Costa, Elisabete C. Moreira, André Queiroz, João Pichon, Chantal Correia, Ilídio Joaquim Sobreira Sousa, Fani |
author2_role |
author author author author author author author |
dc.contributor.none.fl_str_mv |
uBibliorum |
dc.contributor.author.fl_str_mv |
Gaspar, Vítor Manuel Abreu Diogo, Duarte Miguel de Melo Costa, Elisabete C. Moreira, André Queiroz, João Pichon, Chantal Correia, Ilídio Joaquim Sobreira Sousa, Fani |
dc.subject.por.fl_str_mv |
Cell therapy DNA-vaccines Gene therapy Minicircle DNA Transgene expression |
topic |
Cell therapy DNA-vaccines Gene therapy Minicircle DNA Transgene expression |
description |
Introduction: Nucleic-acid-based biopharmaceuticals enclose a remarkable potential for treating debilitating or life-threatening diseases that currently remain incurable. This promising area of research envisages the creation of state-of-the-art DNA vaccines, pluripotent cells or gene-based therapies, which can be used to overcome current issues. To achieve this goal, DNA minicircles are emerging as ideal nonviral vectors due to their safety and persistent transgene expression in either quiescent or actively dividing cells. Areas covered: This review focuses on the characteristics of minicircle DNA (mcDNA) technology and the current advances in their production. The possible modifications to further improve minicircle efficacy are also emphasized and discussed in light of recent advances. As a final point, the main therapeutic applications of mcDNA are summarized, with a special focus on pluripotent stem cells production and cancer therapy. Expert opinion: Achieving in-target and persistent transgene expression is a challenging issue that is of critical importance for a successful therapeutic outcome. The use of miniaturized mcDNA cassettes with additional modifications that increase and prolong expression may contribute to an improved generation of biopharmaceuticals. The unique features of mcDNA render it an attractive alternative to overcome current technical issues and to bridge the significant gap that exists between basic research and clinical applications. |
publishDate |
2015 |
dc.date.none.fl_str_mv |
2015 2015-01-01T00:00:00Z 2018-03-21T09:03:57Z |
dc.type.status.fl_str_mv |
info:eu-repo/semantics/publishedVersion |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
http://hdl.handle.net/10400.6/4670 |
url |
http://hdl.handle.net/10400.6/4670 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.none.fl_str_mv |
Gaspar, V.M., De Melo-Diogo, D., Costa, E.C., Moreira, A.F., Queiroz, J.A., Pichon, C., Correia, I.J. e Sousa, F. (2015) “Minicircle DNA Vectors for Gene Therapy: Advances and Applications”, Expert Opinion on Biological Therapy, Vol. 15, pp. 353-379 10.1517/14712598.2015.996544 |
dc.rights.driver.fl_str_mv |
metadata only access info:eu-repo/semantics/openAccess |
rights_invalid_str_mv |
metadata only access |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
application/pdf |
dc.publisher.none.fl_str_mv |
Taylor & Francis |
publisher.none.fl_str_mv |
Taylor & Francis |
dc.source.none.fl_str_mv |
reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação instacron:RCAAP |
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Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação |
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RCAAP |
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RCAAP |
reponame_str |
Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
collection |
Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
repository.name.fl_str_mv |
Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação |
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1799136354485927936 |