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Gene therapy for inherited retinal diseases

2023Recensão Acesso aberto
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dc.contributor.authorCarvalho, Cláudia
dc.contributor.authorLemos, Luísa
dc.contributor.authorAntas, Pedro
dc.contributor.authorSeabra, Miguel C.
dc.contributor.authorC Seabra, Miguel
dc.contributor.institutionNOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)
dc.contributor.institutioniNOVA4Health - pólo NMS
dc.contributor.pblFrontiers Media
dc.date.accessioned2024-02-22T00:11:23Z
dc.date.available2024-02-22T00:11:23Z
dc.date.issued2023
dc.descriptionFunding Information: The authors declare financial support was received for the research, authorship, and/or publication of this article. Research at the Center for the Unknown is supported by Fundação Champalimaud. PA is supported by grant EXPL/MED-OUT/0599/2021, funded by national funds from FCT/MCTES, and individual grant CEECIND/03862/2020. LL is a recipient of a Choroideremia Research Foundation postdoctoral fellowship. Acknowledgments Publisher Copyright: Copyright © 2023 Carvalho, Lemos, Antas and Seabra.
dc.description.abstractInherited retinal diseases (IRDs) encompass a diverse group of genetic disorders that lead to progressive visual impairment and blindness. Over the years, considerable strides have been made in understanding the underlying molecular mechanisms of IRDs, laying the foundation for novel therapeutic interventions. Gene therapy has emerged as a compelling approach for treating IRDs, with notable advancements achieved through targeted gene augmentation. However, several setbacks and limitations persist, hindering the widespread clinical success of gene therapy for IRDs. One promising avenue of research is the development of new genome editing tools. Cutting-edge technologies such as CRISPR-Cas9 nucleases, base editing and prime editing provide unprecedented precision and efficiency in targeted gene manipulation, offering the potential to overcome existing challenges in gene therapy for IRDs. Furthermore, traditional gene therapy encounters a significant challenge due to immune responses to viral vectors, which remain crucial obstacles in achieving long-lasting therapeutic effects. Nanotechnology has emerged as a valuable ally in the quest to optimize gene therapy outcomes for ocular diseases. Nanoparticles engineered with nanoscale precision offer improved gene delivery to specific retinal cells, allowing for enhanced targeting and reduced immunogenicity. In this review, we discuss recent advancements in gene therapy for IRDs and explore the setbacks that have been encountered in clinical trials. We highlight the technological advances in genome editing for the treatment of IRDs and how integrating nanotechnology into gene delivery strategies could enhance the safety and efficacy of gene therapy, ultimately offering hope for patients with IRDs and potentially paving the way for similar advancements in other ocular disorders.en
dc.description.versionpublishersversion
dc.description.versionpublished
dc.format.extent1924928
dc.identifier.doi10.3389/fopht.2023.1270561
dc.identifier.issn2674-0826
dc.identifier.otherPURE: 83868244
dc.identifier.otherPURE UUID: ef5c8663-6ca3-428e-b775-4ea0cffed8f0
dc.identifier.otherScopus: 85183643908
dc.identifier.otherPubMed: 38983081
dc.identifier.urihttp://hdl.handle.net/10362/163912
dc.identifier.urlhttps://www.scopus.com/pages/publications/85183643908
dc.language.isoeng
dc.peerreviewedyes
dc.subjectCRISPR-Cas
dc.subjectgene therapy
dc.subjectgenome editing
dc.subjectinherited retinal diseases
dc.subjectnanoparticles
dc.subjectOphthalmology
dc.titleGene therapy for inherited retinal diseasesen
dc.title.subtitleexploiting new tools in genome editing and nanotechnologyen
dc.typereview
degois.publication.titleFrontiers in Ophthalmology
degois.publication.volume3
dspace.entity.typePublication
person.familyNameSeabra
person.givenNameMiguel
person.identifier.ciencia-id2512-FAEF-E26F
person.identifier.orcid0000-0002-6404-4892
person.identifier.ridM-3280-2013
person.identifier.scopus-author-id7004369652
rcaap.rightsopenAccess
relation.isAuthorOfPublicationd6eeb095-df2d-4fd0-8241-ab826e5ecb1d
relation.isAuthorOfPublication.latestForDiscoveryd6eeb095-df2d-4fd0-8241-ab826e5ecb1d

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