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Minicircle DNA vectors for gene therapy: advances and applications

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dc.contributor.authorGaspar, Vítor Manuel Abreu
dc.contributor.authorDiogo, Duarte Miguel de Melo
dc.contributor.authorCosta, Elisabete C.
dc.contributor.authorMoreira, André
dc.contributor.authorQueiroz, João
dc.contributor.authorPichon, Chantal
dc.contributor.authorCorreia, Ilídio Joaquim Sobreira
dc.contributor.authorSousa, Fani
dc.date.accessioned2018-03-21T09:03:57Z
dc.date.available2018-03-21T09:03:57Z
dc.date.issued2015
dc.description.abstractIntroduction: 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.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationGaspar, 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. 353-379pt_PT
dc.identifier.doi10.1517/14712598.2015.996544pt_PT
dc.identifier.urihttp://hdl.handle.net/10400.6/4670
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherTaylor & Francispt_PT
dc.relationBolsa de Doutoramento FCT: Biosynthesis and purification of minicircle DNA for application in diabetes cell-specific gene therapy [SFRH/BD/80402/2011]
dc.relationStrategic Project - UI 709 - 2011-2012 [PEst-C/SAU/UI0709/2011]
dc.relation2014 - Strategic Project [PEst-OE/SAU/UI0709/2014]
dc.relation.publisherversionhttps://www.tandfonline.com/doi/full/10.1517/14712598.2015.996544?scroll=top&needAccess=truept_PT
dc.subjectCell therapypt_PT
dc.subjectDNA-vaccinespt_PT
dc.subjectGene therapypt_PT
dc.subjectMinicircle DNApt_PT
dc.subjectTransgene expressionpt_PT
dc.titleMinicircle DNA vectors for gene therapy: advances and applicationspt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.awardNumberSFRH/BD/80402/2011
oaire.awardNumberPEst-C/SAU/UI0709/2011
oaire.awardNumberPEst-OE/SAU/UI0709/2014
oaire.awardTitleBolsa de Doutoramento FCT: Biosynthesis and purification of minicircle DNA for application in diabetes cell-specific gene therapy [SFRH/BD/80402/2011]
oaire.awardTitleStrategic Project - UI 709 - 2011-2012 [PEst-C/SAU/UI0709/2011]
oaire.awardTitle2014 - Strategic Project [PEst-OE/SAU/UI0709/2014]
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/FARH/SFRH%2FBD%2F80402%2F2011/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6820 - DCRRNI ID/PEst-C%2FSAU%2FUI0709%2F2011/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/PEst-OE%2FSAU%2FUI0709%2F2014/PT
oaire.citation.endPage379pt_PT
oaire.citation.startPage353pt_PT
oaire.citation.titleExpert Opinion on Biological Therapypt_PT
oaire.citation.volume15pt_PT
oaire.fundingStreamFARH
oaire.fundingStream6820 - DCRRNI ID
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person.familyNameGaspar
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person.givenNameElisabete Cristina
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project.funder.nameFundação para a Ciência e a Tecnologia
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project.funder.nameFundação para a Ciência e a Tecnologia
rcaap.embargofctCopyright cedido à editora no momento da publicaçãopt_PT
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