Engineering of monosized lipid-coated mesoporous silica nanoparticles for CRISPR delivery.

Autor: Noureddine A; Department of Chemical and Biological Engineering, University of New Mexico, Albuquerque, NM, United States. Electronic address: anoureddine@unm.edu., Maestas-Olguin A; Department of Chemical and Biological Engineering, University of New Mexico, Albuquerque, NM, United States., Saada EA; Department of Systems Biology, Sandia National Laboratories, Livermore, CA, United States., LaBauve AE; Department of Biotechnology and Bioengineering, Sandia National Laboratories, Livermore, CA, United States., Agola JO; Department of Chemical and Biological Engineering, University of New Mexico, Albuquerque, NM, United States., Baty KE; Department of Chemical and Biological Engineering, University of New Mexico, Albuquerque, NM, United States., Howard T; Cell Biology and Physiology, University of New Mexico Health Science Center, Albuquerque, NM, United States., Sabo JK; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, United States., Espinoza CRS; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, United States., Doudna JA; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, United States., Schoeniger JS; Department of Systems Biology, Sandia National Laboratories, Livermore, CA, United States., Butler KS; Department of Nanobiology, Sandia National Laboratories, Albuquerque, NM, United States., Negrete OA; Department of Biotechnology and Bioengineering, Sandia National Laboratories, Livermore, CA, United States., Brinker CJ; Department of Chemical and Biological Engineering, University of New Mexico, Albuquerque, NM, United States; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, United States. Electronic address: jbrinker@unm.edu., Serda RE; Department of Chemical and Biological Engineering, University of New Mexico, Albuquerque, NM, United States; Advanced Materials Laboratory, Sandia National Laboratories, Albuquerque, NM, United States; Department of Internal Medicine, Molecular Medicine, University of New Mexico Health Science Center, Albuquerque, NM, United States. Electronic address: rserda@salud.unm.edu.
Jazyk: angličtina
Zdroj: Acta biomaterialia [Acta Biomater] 2020 Sep 15; Vol. 114, pp. 358-368. Date of Electronic Publication: 2020 Jul 21.
DOI: 10.1016/j.actbio.2020.07.027
Abstrakt: CRISPR gene editing technology is strategically foreseen to control diseases by correcting underlying aberrant genetic sequences. In order to overcome drawbacks associated with viral vectors, the establishment of an effective non-viral CRISPR delivery vehicle has become an important goal for nanomaterial scientists. Herein, we introduce a monosized lipid-coated mesoporous silica nanoparticle (LC-MSN) delivery vehicle that enables both loading of CRISPR components [145 µg ribonucleoprotein (RNP) or 40 µg plasmid/mg nanoparticles] and efficient release within cancer cells (70%). The RNP-loaded LC-MSN exhibited 10% gene editing in both in vitro reporter cancer cell lines and in an in vivo Ai9-tdTomato reporter mouse model. The structural and chemical versatility of the mesoporous silica core and lipid coating along with framework dissolution-assisted cargo delivery open new prospects towards safe CRISPR component delivery and enhanced gene editing. STATEMENT OF SIGNIFICANCE: After the discovery of CRISPR gene-correcting technology in bacteria. The translation of this technology to mammalian cells may change the face of cancer therapy within the next years. This was first made possible through the use of viral vectors; however, such systems limit the safe translation of CRISPR into clinics because its difficult preparation and immunogenicity. Therefore, biocompatible non-viral nanoparticulate systems are required to successfully deliver CRISPR into cancer cells. The present study presents the use of biomimetic lipid-coated mesoporous silica nanoparticles showing successful delivery of CRISPR ribonucleoprotein and plasmid into HeLa cervical and A549 lung cancer cells as well as successful gene editing in mice brain.
Competing Interests: Declaration of Competing Interest No conflict of interest to declare.
(Copyright © 2020 Elsevier Ltd. All rights reserved.)
Databáze: MEDLINE
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