Lipid-coated mesoporous silica nanoparticles for anti-viral applications via delivery of CRISPR-Cas9 ribonucleoproteins

Annette E. LaBauve, Edwin A. Saada, Iris K.A. Jones, Richard Mosesso, Achraf Noureddine, Jessica Techel, Andrew Gomez, Nicole Collette, Michael B. Sherman, Rita E. Serda, Kimberly S. Butler, C. Jeffery Brinker, Joseph S. Schoeniger, Darryl Sasaki, Oscar A. Negrete

Research output: Contribution to journalArticlepeer-review

6 Scopus citations

Abstract

Emerging and re-emerging viral pathogens present a unique challenge for anti-viral therapeutic development. Anti-viral approaches with high flexibility and rapid production times are essential for combating these high-pandemic risk viruses. CRISPR-Cas technologies have been extensively repurposed to treat a variety of diseases, with recent work expanding into potential applications against viral infections. However, delivery still presents a major challenge for these technologies. Lipid-coated mesoporous silica nanoparticles (LCMSNs) offer an attractive delivery vehicle for a variety of cargos due to their high biocompatibility, tractable synthesis, and amenability to chemical functionalization. Here, we report the use of LCMSNs to deliver CRISPR-Cas9 ribonucleoproteins (RNPs) that target the Niemann–Pick disease type C1 gene, an essential host factor required for entry of the high-pandemic risk pathogen Ebola virus, demonstrating an efficient reduction in viral infection. We further highlight successful in vivo delivery of the RNP-LCMSN platform to the mouse liver via systemic administration.

Original languageEnglish (US)
Article number6873
JournalScientific reports
Volume13
Issue number1
DOIs
StatePublished - Dec 2023
Externally publishedYes

ASJC Scopus subject areas

  • General

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