Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR‐Cas9 Ribonucleoprotein for Brain Gene Editing

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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.

Location: Deutsche Nationalbibliothek Frankfurt am Main

Extent: Online-Ressource

Language: Englisch

Bibliographic citation: Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR‐Cas9 Ribonucleoprotein for Brain Gene Editing ; day:18 ; month:03 ; year:2024 ; extent:9
Angewandte Chemie / International edition. International edition ; (18.03.2024) (gesamt 9)

Creator: 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

DOI: 10.1002/anie.202401004

URN: urn:nbn:de:101:1-2024031913113116233935

Rights: Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.

Last update: 14.08.2025, 10:58 AM CEST

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Associated

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

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Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR‐Cas9 Ribonucleoprotein for Brain Gene Editing

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Modulation of CRISPR‐Cas9 Cleavage with an Oligo‐Ribonucleoprotein Design

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Generation of double knockout cattle via CRISPR-Cas9 ribonucleoprotein (RNP) electroporation

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Targeted delivery of CRISPR-Cas9 ribonucleoprotein into arthropod ovaries for heritable germline gene editing

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Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein

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Structure of the IscB–ωRNA ribonucleoprotein complex, the likely ancestor of CRISPR-Cas9

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Expansion of targetable sites for the ribonucleoprotein-based CRISPR/Cas9 system in the silkworm Bombyx mori

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Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo

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Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR‐Cas9 Ribonucleoprotein for Brain Gene Editing

Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR‐Cas9 Ribonucleoprotein for Brain Gene Editing

Modulation of CRISPR‐Cas9 Cleavage with an Oligo‐Ribonucleoprotein Design

Modulation of CRISPR‐Cas9 Cleavage with an Oligo‐Ribonucleoprotein Design

Generation of double knockout cattle via CRISPR-Cas9 ribonucleoprotein (RNP) electroporation

Generation of double knockout cattle via CRISPR-Cas9 ribonucleoprotein (RNP) electroporation

Inhibition of CRISPR-Cas9 ribonucleoprotein complex assembly by anti-CRISPR AcrIIC2

Inhibition of CRISPR-Cas9 ribonucleoprotein complex assembly by anti-CRISPR AcrIIC2

CRISPR-Cas9 ribonucleoprotein-mediated co-editing and counterselection in the rice blast fungus

CRISPR-Cas9 ribonucleoprotein-mediated co-editing and counterselection in the rice blast fungus

Editing of the ethylene biosynthesis gene in carnation using CRISPR-Cas9 ribonucleoprotein complex

Editing of the ethylene biosynthesis gene in carnation using CRISPR-Cas9 ribonucleoprotein complex

Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing

Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing

Targeted delivery of CRISPR-Cas9 ribonucleoprotein into arthropod ovaries for heritable germline gene editing

Targeted delivery of CRISPR-Cas9 ribonucleoprotein into arthropod ovaries for heritable germline gene editing

Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein

Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein

Structure of the IscB–ωRNA ribonucleoprotein complex, the likely ancestor of CRISPR-Cas9

Structure of the IscB–ωRNA ribonucleoprotein complex, the likely ancestor of CRISPR-Cas9

Expansion of targetable sites for the ribonucleoprotein-based CRISPR/Cas9 system in the silkworm Bombyx mori

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Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo

Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo

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