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https://hdl.handle.net/10316/116152
Title: | Near-Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR-Cas9 Ribonucleoprotein for Brain Gene Editing | Authors: | Simões, Susana Lino, Miguel Barrera, Angela Rebelo, Catarina Tomatis, Francesca Vilaça, Andreia Breunig, Christopher Neuner, Andrea Peça, João González, Ricardo Carvalho, Alexandra Stricker, Stefan Ferreira, Lino |
Keywords: | Brain; CRISPR/Cas9; Gene editing; NIR light activation; Spatial control | Issue Date: | 21-May-2024 | Project: | info:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/UIDB/04539/2020 CPCA/A2/4568/2020 CPCA/A1/447491/2021 info:eu-repo/grantAgreement/FCT/9471 - RIDTI/PTDC/NAN-MAT/28060/2017/PT/BrainEdition: remotely controlling gene edition in neural stem cells |
Serial title, monograph or event: | Angewandte Chemie International Edition | Volume: | 63 | Issue: | 21 | 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. | URI: | https://hdl.handle.net/10316/116152 | ISSN: | 1521-3773 | DOI: | 10.1002/anie.202401004 | Rights: | openAccess |
Appears in Collections: | UC Bibliotecas - Artigos em Revistas Internacionais |
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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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