Cited by 540RNA Interference and Gene DeliveryCRISPR and Genetic EngineeringNanoparticle-Based Drug Delivery

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Non‐Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co‐Delivery of Cas9 mRNA and sgRNA

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Miller, Jason B., Zhang, Shuyuan, Kos, Petra, Xiong, Hu, Zhou, Kejin, Perelman, Sofya S., Zhu, Hao, Siegwart, Daniel J.·2017·Angewandte Chemie International Edition

DOI 10.1002/anie.201610209PMC PMC5521011

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Abstract

CRISPR/Cas is a revolutionary gene editing technology with wide‐ranging utility. The safe, non‐viral delivery of CRISPR/Cas components would greatly improve future therapeutic utility. We report the synthesis and development of zwitterionic amino lipids (ZALs) that are uniquely able to (co)deliver long RNAs including Cas9 mRNA and sgRNAs. ZAL nanoparticle (ZNP) delivery of low sgRNA doses (15 n m ) reduces protein expression by >90 % in cells. In contrast to transient therapies (such as RNAi), we show that ZNP delivery of sgRNA enables permanent DNA editing with an indefinitely sustained 95 % decrease in protein expression. ZNP delivery of mRNA results in high protein expression at low doses in vitro (<600 pM) and in vivo (1 mg kg −1 ). Intravenous co‐delivery of Cas9 mRNA and sgLoxP induced expression of floxed tdTomato in the liver, kidneys, and lungs of engineered mice. ZNPs provide a chemical guide for rational design of long RNA carriers, and represent a promising step towards improving the safety and utility of gene editing.

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Authors: Miller, J. B., Zhang, S., Kos, P., Xiong, H., Zhou, K., Perelman, S. S., Zhu, H., & Siegwart, D. J.
Affiliations: Harold C. Simmons Comprehensive Cancer Center (US)
The University of Texas Southwestern Medical Center (US)
University of Texas Southwestern Medical Center Simmons Comprehensive Cancer Center Department of Biochemistry Dallas TX 75390 USA
Corresponding author: Siegwart, Daniel J.
ORCID: Miller, Jason B.
Zhang, Shuyuan
Xiong, Hu
Perelman, Sofya S.
Zhu, Hao
Siegwart, Daniel J.
Venue: Angewandte Chemie International Edition
Publisher: Wiley
Published: 2016-12-16
Volume: 56
Issue: 4
Pages: 1059-1063
ISSN: 1433-7851 (ISSN-L)
1521-3773
DOI: 10.1002/anie.201610209
PMC: PMC5521011
OpenAlex: W2560987571
Cited by: 540
References: 45
Citations by year: 2026: 35 · 2025: 39 · 2024: 47 · 2023: 48 · 2022: 66 · 2021: 86 · 2020: 79 · 2019: 59 · 2018: 55 · 2017: 24 · 2016: 1
FWCI: 24.09
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: onlinelibrary.wiley.com/termsAndConditions#vor (vor)
Locations: Angewandte Chemie International Edition (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Cancer Institute (R01CA190525)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
Keywords: CRISPR gene editing, lipid nanoparticles, Cas9 mRNA, single guide RNA, co-delivery, in vivo gene editing, loxP
MeSH terms: Gene Editing, RNA, Guide, CRISPR-Cas Systems, Lipids, RNA, Messenger, Molecular Structure, RNA, Guide, Kinetoplastida, Gene Transfer Techniques, Nanoparticles, CRISPR-Cas Systems

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Non‐Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co‐Delivery of Cas9 mRNA and sgRNA

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