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Efficiency of RAFT-synthesized PDMAEMA in gene transfer to the retina

2017Journal article Restricted access
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dc.contributor.authorBitoque, Diogo
dc.contributor.authorS, Simão
dc.contributor.authorOliveira, Ana V.
dc.contributor.authorMachado, S.
dc.contributor.authorDuran, Margarita R.
dc.contributor.authorLopes, Eduardo
dc.contributor.authorCosta, Ana M. Rosa da
dc.contributor.authorSilva, Gabriela
dc.date.accessioned2016-01-06T16:20:02Z
dc.date.available2016-01-06T16:20:02Z
dc.date.issued2017
dc.description.abstractGene therapy has long been heralded as the new hope to evolve from symptomatic care of genetic pathologies to a full cure. Recent successes in using gene therapy for treating several ocular and haematopoietic pathologies have shown the great potential of this approach that, in the early days, relied on the use of viral vectors, which were considered by many to be undesirable for human treatment. Therefore, there is considerable interest and effort in developing non-viral vectors, with efficiency close to that of viral vectors. The aim of this study was to develop suitable non-viral carriers for gene therapy to treat pathologies affecting the retina. In this study poly(2-(N,N-dimethylamino)ethyl methacrylate), PDMAEMA was synthesized by reversible addition-fragmentation chain transfer (RAFT) and the in vitro cytocompatibility and transfection efficiency of a range of polymer:DNA ratios evaluated using a retinal cell line; in vivo biocompatibility was evaluated by ocular injection in C57BL/6 mice. The results showed that through RAFT, it is possible to produce a defined-size polymer that is compatible with cell viability in vitro and capable of efficiently directing gene expression in a polymer-DNA ratio-dependent manner. When injected into the eyes of mice, these vectors induced a transient, mild inflammation, characteristic of the implantation of medical devices. These results form the basis of future studies where RAFT-synthesized PDMAEMA will be used to deliver gene expression systems to the retina of mouse models of retinal pathologies. Copyright © 2014 John Wiley & Sons, Ltd.pt_PT
dc.description.sponsorshipPEst-OE_QUI_UI4023_2011; PIRG05-GA-2009–249314
dc.identifier.doihttp://dx.doi.org/10.1002/term.1909pt_PT
dc.identifier.issn1932-6254
dc.identifier.otherAUT: GAS02236; AMC01695;
dc.identifier.urihttp://hdl.handle.net/10400.1/7390
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherJohn Wiley and Sonspt_PT
dc.relationDEVELOPMENT OF NON-VIRAL VECTORS FOR GENE THERAPY FOR PATHOLOGIES OF THE RETINA
dc.relationNEW GENE THERAPY STRATEGIES FOR THE TREATMENT OF RETINAL NEOVASCULARIZATION
dc.relationStrategic Project - LA 23 - 2011-2012
dc.relation.publisherversionhttp://onlinelibrary.wiley.com/doi/10.1002/term.1909/abstractpt_PT
dc.subjectGene therapypt_PT
dc.subjectNon-viral vectorspt_PT
dc.subjectPDMAEMApt_PT
dc.subjectPolymerspt_PT
dc.subjectRAFT synthesispt_PT
dc.subjectRetinapt_PT
dc.titleEfficiency of RAFT-synthesized PDMAEMA in gene transfer to the retinapt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.awardTitleDEVELOPMENT OF NON-VIRAL VECTORS FOR GENE THERAPY FOR PATHOLOGIES OF THE RETINA
oaire.awardTitleNEW GENE THERAPY STRATEGIES FOR THE TREATMENT OF RETINAL NEOVASCULARIZATION
oaire.awardTitleStrategic Project - LA 23 - 2011-2012
oaire.awardURIinfo:eu-repo/grantAgreement/FCT//SFRH%2FBD%2F70318%2F2010/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/OE/SFRH%2FBPD%2F78404%2F2011/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/3599-PPCDT/PTDC%2FSAU-BEB%2F098475%2F2008/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/PEst-OE%2FEQB%2FLA0023%2F2011/PT
oaire.citation.endPage275
oaire.citation.issue1
oaire.citation.startPage265
oaire.citation.titleJournal of Tissue Engineering and Regenerative Medicinept_PT
oaire.citation.volume11
oaire.fundingStreamOE
oaire.fundingStream3599-PPCDT
oaire.fundingStream6817 - DCRRNI ID
person.familyNameBitoque
person.familyNameSimao
person.familyNameMachado
person.familyNameRosa da Costa
person.familyNameSilva
person.givenNameDiogo
person.givenNameSonia
person.givenNameSusana
person.givenNameAna M
person.givenNameGabriela
person.identifier2140108
person.identifier305029
person.identifier.ciencia-idD81C-6457-7F28
person.identifier.ciencia-idA418-A85E-5DB1
person.identifier.ciencia-idDC1E-F164-FDD9
person.identifier.orcid0000-0002-6536-6578
person.identifier.orcid0000-0002-0245-0633
person.identifier.orcid0000-0002-3152-1701
person.identifier.orcid0000-0003-0225-9537
person.identifier.orcid0000-0001-5946-1918
person.identifier.ridK-4033-2012
person.identifier.ridE-2165-2012
person.identifier.ridF-4409-2012
person.identifier.scopus-author-id55755464000
person.identifier.scopus-author-id24067982400
person.identifier.scopus-author-id56201414700
person.identifier.scopus-author-id53986075100
person.identifier.scopus-author-id7101647767
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.nameFundação para a Ciência e a Tecnologia
project.funder.nameFundação para a Ciência e a Tecnologia
project.funder.nameFundação para a Ciência e a Tecnologia
project.funder.nameFundação para a Ciência e a Tecnologia
rcaap.rightsrestrictedAccesspt_PT
rcaap.typearticlept_PT
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