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Title: Efficient spatially targeted gene editing using a near-infrared activatable protein-conjugated nanoparticle for brain applications
Authors: Rebelo, Catarina 
Reis, Tiago 
Guedes, Joana 
Saraiva, Cláudia
Rodrigues, Artur Filipe 
Simões, Susana
Bernardino, Liliana 
Peça, João 
Pinho, Sónia L. C.
Ferreira, Lino 
Issue Date: 2022
Publisher: Nature
Project: POCI-01-0145-FEDER-016390:CANCEL STEM 
info:eu-repo/grantAgreement/FCT/9471 - RIDTI/PTDC/NAN-MAT/28060/2017/PT/BrainEdition: remotely controlling gene edition in neural stem cells 
POCI-01-0145- FEDER-016682 
Interreg 2IQBioNeuro 
info:eu-repo/grantAgreement/EC/H2020/669088/EU/Enhancing Research in Ageing at the University of Coimbra 
EAPA_791/2018 - NEUROATLANTIC entitled: ‘An Atlantic innovation platform on diagnosis and treatment of neurological diseases and aging’ 
info:eu-repo/grantAgreement/EC/H2020/952266/EU/RESEarch for healThy AGEING 
info:eu-repo/grantAgreement/FCT/OE/PD/BD/52337/2013/PT/Nanomedicine to enhance heart regenerative potential 
info:eu-repo/grantAgreement/FCT/FARH/SFRH/BPD/96048/2013/PT/Cellular reprogramming by near infrared NIR-activatable nanoparticles containing transcription factors - NanoTarget: remote modulation for cardiac progenitor cells 
info:eu-repo/grantAgreement/EC/H2020/101003413/EU/LIght-responsive GrapHene-based inTerfaces for Electrical STimulation 
Serial title, monograph or event: Nature Communications
Volume: 13
Issue: 1
Abstract: Spatial control of gene expression is critical to modulate cellular functions and deconstruct the function of individual genes in biological processes. Light-responsive gene-editing formulations have been recently developed; however, they have shown limited applicability in vivo due to poor tissue penetration, limited cellular transfection and the difficulty in evaluating the activity of the edited cells. Here, we report a formulation composed of upconversion nanoparticles conjugated with Cre recombinase enzyme through a photocleavable linker, and a lysosomotropic agent that facilitates endolysosomal escape. This formulation allows in vitro spatial control in gene editing after activation with near-infrared light. We further demonstrate the potential of this formulation in vivo through three different paradigms: (i) gene editing in neurogenic niches, (ii) gene editing in the ventral tegmental area to facilitate monitoring of edited cells by precise optogenetic control of reward and reinforcement, and (iii) gene editing in a localized brain region via a noninvasive administration route (i.e., intranasal).
ISSN: 2041-1723
DOI: 10.1038/s41467-022-31791-6
Rights: openAccess
Appears in Collections:I&D CNC - Artigos em Revistas Internacionais

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