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Mitochondrial Gene Therapy: Advances in Mitochondrial Gene Cloning, Plasmid Production, and Nanosystems Targeted to Mitochondria

2017Journal article Restricted access
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dc.contributor.authorCoutinho, Eduarda
dc.contributor.authorBatista, Cátia
dc.contributor.authorSousa, Fani
dc.contributor.authorQueiroz, João
dc.contributor.authorCosta, Diana
dc.date.accessioned2020-01-09T15:59:50Z
dc.date.available2020-01-09T15:59:50Z
dc.date.issued2017
dc.description.abstractMitochondrial gene therapy seems to be a valuable and promising strategy to treat mitochondrial disorders. The use of a therapeutic vector based on mitochondrial DNA, along with its affinity to the site of mitochondria, can be considered a powerful tool in the reestablishment of normal mitochondrial function. In line with this and for the first time, we successfully cloned the mitochondrial gene ND1 that was stably maintained in multicopy pCAG-GFP plasmid, which is used to transform E. coli. This mitochondrial-gene-based plasmid was encapsulated into nanoparticles. Furthermore, the functionalization of nanoparticles with polymers, such as cellulose or gelatin, enhances their overall properties and performance for gene therapy. The fluorescence arising from rhodamine nanoparticles in mitochondria and a fluorescence microscopy study show pCAG-GFP-ND1-based nanoparticles' cell internalization and mitochondria targeting. The quantification of GFP expression strongly supports this finding. This work highlights the viability of gene therapy based on mitochondrial DNA instigating further in vitro research and clinical translation.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.doi10.1021/acs.molpharmaceut.6b00823pt_PT
dc.identifier.urihttp://hdl.handle.net/10400.6/8174
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherACS Publicationspt_PT
dc.relationGene Therapy for Mitochondrial DNA disease: Development of an efficient and mitochondrial-targeted vector
dc.relation.publisherversionhttps://pubs.acs.org/doi/10.1021/acs.molpharmaceut.6b00823pt_PT
dc.subjectAnimalspt_PT
dc.subjectCloning, Molecularpt_PT
dc.subjectDNA Mitochondrialpt_PT
dc.subjectEscherichia colipt_PT
dc.subjectFluorescencept_PT
dc.subjectGelatinpt_PT
dc.subjectGenes, Mitochondrialpt_PT
dc.subjectGenetic Therapypt_PT
dc.subjectGenetic Vectorspt_PT
dc.subjectGreen Fluorescent Proteinspt_PT
dc.subjectHumanspt_PT
dc.subjectMicept_PT
dc.subjectMitochondriapt_PT
dc.subjectNanoparticlespt_PT
dc.subjectPlasmidspt_PT
dc.subjectPolymerspt_PT
dc.subjectRhodaminespt_PT
dc.subjectTransfectionpt_PT
dc.titleMitochondrial Gene Therapy: Advances in Mitochondrial Gene Cloning, Plasmid Production, and Nanosystems Targeted to Mitochondriapt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.awardTitleGene Therapy for Mitochondrial DNA disease: Development of an efficient and mitochondrial-targeted vector
oaire.awardURIinfo:eu-repo/grantAgreement/FCT//SFRH%2FBPD%2F103592%2F2014/PT
oaire.citation.endPage638pt_PT
oaire.citation.issue3pt_PT
oaire.citation.startPage626pt_PT
oaire.citation.titleMolecular Pharmaceuticspt_PT
oaire.citation.volume14pt_PT
person.familyNameCoutinho
person.familyNameSousa
person.familyNameQueiroz
person.familyNameCosta
person.givenNameEduarda
person.givenNameFani
person.givenNameJoão
person.givenNameDiana
person.identifier1137823
person.identifier.ciencia-id991D-2E13-A840
person.identifier.ciencia-id931E-B66D-E341
person.identifier.ciencia-idFA1D-8901-2ED6
person.identifier.orcid0000-0002-9923-2164
person.identifier.orcid0000-0001-9996-2194
person.identifier.orcid0000-0002-3096-8325
person.identifier.orcid0000-0002-5049-4518
person.identifier.ridI-5845-2015
person.identifier.ridA-2014-2017
person.identifier.ridL-3104-2014
person.identifier.scopus-author-id7005110268
person.identifier.scopus-author-id7003705645
person.identifier.scopus-author-id9437315900
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.nameFundação para a Ciência e a Tecnologia
rcaap.embargofctCopyright cedido à editora no momento da publicaçãopt_PT
rcaap.rightsclosedAccesspt_PT
rcaap.typearticlept_PT
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relation.isAuthorOfPublication2935de97-fc90-4d38-9505-4caa364f8a10
relation.isAuthorOfPublicationc798c033-c160-4f99-a8ad-04416923943f
relation.isAuthorOfPublicationa0261bc4-da2c-4630-9361-19ce01cbdc7e
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