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Revisiting gene delivery to the brain

2021-02-21Recensão Acesso aberto
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dc.contributor.authorConniot, João
dc.contributor.authorTalebian, Sepehr
dc.contributor.authorSimões, Susana
dc.contributor.authorFerreira, Lino
dc.contributor.authorConde, João
dc.contributor.authorConde, João
dc.contributor.institutionCentre for Toxicogenomics and Human Health (ToxOmics)
dc.contributor.institutionNOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)
dc.contributor.pblRSC - Royal Society of Chemistry
dc.date.accessioned2023-01-11T22:14:52Z
dc.date.available2023-01-11T22:14:52Z
dc.date.issued2021-02-21
dc.descriptionFunding: J. C. acknowledges the European Research Council Starting Grant (ERC-StG-2019-848325)
dc.description.abstractNeurodegenerative disorders, ischemic brain diseases, and brain tumors are debilitating diseases that severely impact a person's life and could possibly lead to their demise if left untreated. Many of these diseases do not respond to small molecule therapeutics and have no effective long-Term therapy. Gene therapy offers the promise of treatment or even a cure for both genetic and acquired brain diseases, mediated by either silencing or editing disease-specific genes. Indeed, in the last 5 years, significant progress has been made in the delivery of non-coding RNAs as well as gene-editing formulations to the brain. Unfortunately, the delivery is a major limiting factor for the success of gene therapies. Both viral and non-viral vectors have been used to deliver genetic information into a target cell, but they have limitations. Viral vectors provide excellent transduction efficiency but are associated with toxic effects and have limited packaging capacity; however, non-viral vectors are less toxic and show a high packaging capacity at the price of low transfection efficiency. Herein, we review the progress made in the field of brain gene therapy, particularly in the design of non-Toxic and trackable non-viral vectors, capable of controlled release of genes in response to internal/external triggers, and in the delivery of formulations for gene editing. The application of these systems in the context of various brain diseases in pre-clinical and clinical tests will be discussed. Such promising approaches could potentially pave the way for clinical realization of brain gene therapies. This journal isen
dc.description.versionpublishersversion
dc.description.versionpublished
dc.format.extent23
dc.format.extent6036942
dc.identifier.doi10.1039/d0bm01278e
dc.identifier.issn2047-4830
dc.identifier.otherPURE: 28428597
dc.identifier.otherPURE UUID: c4095d15-55d6-4f2f-8020-6c7a98d40e42
dc.identifier.otherScopus: 85101324881
dc.identifier.otherPubMed: 33315025
dc.identifier.otherWOS: 000620899200029
dc.identifier.urihttp://hdl.handle.net/10362/147349
dc.identifier.urlhttps://www.scopus.com/pages/publications/85101324881
dc.language.isoeng
dc.peerreviewedyes
dc.subjectEfficiency
dc.subjectGene therapy
dc.subjectGene transfer
dc.subjectNeurodegenerative diseases
dc.subjectVectors
dc.subjectBiomedical Engineering
dc.subjectGeneral Materials Science
dc.titleRevisiting gene delivery to the brainen
dc.title.subtitleSilencing and editingen
dc.typereview
degois.publication.firstPage1065
degois.publication.issue4
degois.publication.lastPage1087
degois.publication.titleBiomaterials Science
degois.publication.volume9
dspace.entity.typePublication
person.familyNameConde
person.givenNameJoão
person.identifier807432
person.identifier.ciencia-idA71C-B10E-255E
person.identifier.orcid0000-0001-8422-6792
person.identifier.ridF-2231-2011
person.identifier.scopus-author-id56992468300
rcaap.rightsopenAccess
relation.isAuthorOfPublication33ca4178-1b25-4b5e-ae16-b2500abe4c58
relation.isAuthorOfPublication.latestForDiscovery33ca4178-1b25-4b5e-ae16-b2500abe4c58

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