Utilize este identificador para referenciar este registo: https://hdl.handle.net/10316/116152
Título: Near-Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR-Cas9 Ribonucleoprotein for Brain Gene Editing
Autor: 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
Palavras-chave: Brain; CRISPR/Cas9; Gene editing; NIR light activation; Spatial control
Data: 21-Mai-2024
Projeto: 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 
Título da revista, periódico, livro ou evento: Angewandte Chemie International Edition
Volume: 63
Número: 21
Resumo: 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
Direitos: openAccess
Aparece nas coleções:UC Bibliotecas - Artigos em Revistas Internacionais

Mostrar registo em formato completo

Visualizações de página

36
Visto em 28/ago/2024

Downloads

6
Visto em 28/ago/2024

Google ScholarTM

Verificar

Altmetric

Altmetric


Todos os registos no repositório estão protegidos por leis de copyright, com todos os direitos reservados.