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Near-Infrared Light Activated Formulation for the Spatially Controlled Release ...

Simões, Susana; Lino, Miguel; Barrera, Angela; Rebelo, Catarina; Tomatis, Francesca; Vilaça, Andreia; Breunig, Christopher; Neuner, Andrea; Peça, João

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 co...


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