Please use this identifier to cite or link to this item:
https://hdl.handle.net/10316/116152
DC Field | Value | Language |
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dc.contributor.author | Simões, Susana | - |
dc.contributor.author | Lino, Miguel | - |
dc.contributor.author | Barrera, Angela | - |
dc.contributor.author | Rebelo, Catarina | - |
dc.contributor.author | Tomatis, Francesca | - |
dc.contributor.author | Vilaça, Andreia | - |
dc.contributor.author | Breunig, Christopher | - |
dc.contributor.author | Neuner, Andrea | - |
dc.contributor.author | Peça, João | - |
dc.contributor.author | González, Ricardo | - |
dc.contributor.author | Carvalho, Alexandra | - |
dc.contributor.author | Stricker, Stefan | - |
dc.contributor.author | Ferreira, Lino | - |
dc.date.accessioned | 2024-08-21T14:14:21Z | - |
dc.date.available | 2024-08-21T14:14:21Z | - |
dc.date.issued | 2024-05-21 | - |
dc.identifier.issn | 1521-3773 | pt |
dc.identifier.uri | https://hdl.handle.net/10316/116152 | - |
dc.description.abstract | The CRISPR/Cas9 system has emerged as a promising platform for gene editing; however, the lack of an efficient and safe delivery system to introduce it into cells continues to hinder clinical translation. Here, we report a rationally designed gene-editing nanoparticle (NP) formulation for brain applications: an sgRNA:Cas9 ribonucleoprotein complex is immobilized on the NP surface by oligonucleotides that are complementary to the sgRNA. Irradiation of the formulation with a near-infrared (NIR) laser generates heat in the NP, leading to the release of the ribonucleoprotein complex. The gene-editing potential of the formulation was demonstrated in vitro at the single-cell level. The safety and gene editing of the formulation were also demonstrated in the brains of reporter mice, specifically in the subventricular zone after intracerebral administration and in the olfactory bulb after intranasal administration. The formulation presented here offers a new strategy for the spatially controlled delivery of the CRISPR system to the brain. | pt |
dc.description.sponsorship | L.F. acknowledges the funding of COMPETE2020 and FCT programs [PTDC/NAN-MAT/28060/2017 (acronym: BrainEdition), POCI-01-0145-FEDER-016682], Portugal 2020- COMPETE funding through “Programa Operacional Regional do Centro” CENTRO2020 (CENTRO-01-0145-FEDER- 000014 and CENTRO-01-0145-FEDER-028060), EC project RESETageing (Ref:952266) and EIC project REGENERAR (Ref:101129812). S.S. acknowledge the FCT fellowships (SFRH/BPD/105327/2014) and the funding by COMPETE2020 and FCT program (EXPL/BIA-CEL/0989/ 2021 (acronym: Editing_Vision). A.B. and F.T. to the European Commission for the funding through the Marie Skłodowska-Curie Innovative Training Network “NANOSTEM” (n. 764958) for the fellowship. F.T. acknowledge the FCT fellowships (2021/06297/BD) and the PhD Programme in Experimental Biology from University of Coimbra. A.C. and R.G. acknowledge the funding by FCT and CENTRO2020 under the Portuguese Partnership Agreement 2020 by ERDF (2020.10114.BD, IF/01272/2015, and UIDB/ 04539/2020), and acknowledge computing resources made available by the National Distributed Computing Infrastructure (INCD) funded by FCT and ERDF (01/SAICT/ 2016 n°. 022153), and the Advanced Computing Project (CPCA/A2/4568/2020 and CPCA/A1/447491/2021). | pt |
dc.language.iso | eng | pt |
dc.relation | info:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/UIDB/04539/2020 | pt |
dc.relation | CPCA/A2/4568/2020 | pt |
dc.relation | CPCA/A1/447491/2021 | pt |
dc.relation | info:eu-repo/grantAgreement/FCT/9471 - RIDTI/PTDC/NAN-MAT/28060/2017/PT/BrainEdition: remotely controlling gene edition in neural stem cells | pt |
dc.rights | openAccess | pt |
dc.subject | Brain; CRISPR/Cas9; Gene editing; NIR light activation; Spatial control | pt |
dc.subject.mesh | Animals | pt |
dc.subject.mesh | Mice | pt |
dc.subject.mesh | Ribonucleoproteins | pt |
dc.subject.mesh | Nanoparticles | pt |
dc.subject.mesh | Humans | pt |
dc.subject.mesh | Gene Editing | pt |
dc.subject.mesh | CRISPR-Cas Systems | pt |
dc.subject.mesh | Infrared Rays | pt |
dc.subject.mesh | Brain | pt |
dc.title | Near-Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR-Cas9 Ribonucleoprotein for Brain Gene Editing | pt |
dc.type | article | - |
degois.publication.issue | 21 | pt |
degois.publication.title | Angewandte Chemie International Edition | pt |
dc.relation.publisherversion | https://onlinelibrary.wiley.com/doi/epdf/10.1002/anie.202401004 | pt |
dc.peerreviewed | yes | pt |
dc.identifier.doi | 10.1002/anie.202401004 | pt |
degois.publication.volume | 63 | pt |
dc.date.embargo | 2024-05-21 | * |
uc.date.periodoEmbargo | 0 | pt |
item.languageiso639-1 | en | - |
item.openairecristype | http://purl.org/coar/resource_type/c_18cf | - |
item.grantfulltext | open | - |
item.fulltext | Com Texto completo | - |
item.cerifentitytype | Publications | - |
item.openairetype | article | - |
crisitem.project.grantno | Center for Innovative Biomedicine and Biotechnology - CIBB | - |
crisitem.author.researchunit | CNC - Center for Neuroscience and Cell Biology | - |
crisitem.author.researchunit | CNC - Center for Neuroscience and Cell Biology | - |
crisitem.author.orcid | 0000-0002-7378-0752 | - |
crisitem.author.orcid | 0000-0003-4989-2129 | - |
Appears in Collections: | UC Bibliotecas - Artigos em Revistas Internacionais |
Files in This Item:
File | Description | Size | Format | |
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Angew Chem Int Ed - 2024 - Simões - Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of.pdf | 6.95 MB | Adobe PDF | View/Open |
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