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Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles
Open accessAbstract
A main challenge to broaden the biomedical application of CRISPR/Cas9 (clustered regularly interspaced short palindromic repeat (CRISPR) associated protein 9) genome editing technique is the delivery of Cas9 nuclease and single‐guide RNA (sgRNA) into the specific cell and organ. An effective and very fast CRISPR/Cas9 genome editing in vitro and in vivo enabled by bioreducible lipid/Cas9 messenger RNA (mRNA) nanoparticle is reported. BAMEA‐O16B, a lipid nanoparticle integrated with disulfide bonds, can efficiently deliver Cas9 mRNA and sgRNA into cells while releasing RNA in response to the reductive intracellular environment for genome editing as fast as 24 h post mRNA delivery. It is demonstrated that the simultaneous delivery of Cas9 mRNA and sgRNA using BAMEA‐O16B knocks out green fluorescent protein (GFP) expression of human embryonic kidney cells with efficiency up to 90%. Moreover, the intravenous injection of BAMEA‐O16B/Cas9 mRNA/sgRNA nanoparticle effectively accumulates in hepatocytes, and knocks down proprotein convertase subtilisin/kexin type 9 level in mouse serum down to 20% of nontreatment. The leading lipid nanoparticle, BAMEA‐O16B, represents one of the most efficient CRISPR/Cas9 delivery nanocarriers reported so far, and it can broaden the therapeutic promise of mRNA and CRISPR/Cas9 technique further.
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“Gene-editing components can be delivered by lipid nanoparticle–mRNA formulations to treat genetic diseases, including HTI92,238, hypercholesterolaemia81,96,105,239, lipoprotein metabolism disorders85 and transthyretin amyloidosis239,240, which has been demonstrated in preclinical studies.”
From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [81]
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Bhattacharjee, R., Jana, A., Nandi, A., Sinha, A., Bhattacharjee, A., Mitra, S., Kar, S., Dey, A., Singh, S. K., Varma, R. S., et al. (2022) · Materials & Design · doi:10.1016/j.matdes.2022.111415 · Cited by 29
Lipid-nanoparticle-enabled nucleic acid therapeutics for liver disorders
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Precisely Targeted Nanoparticles for CRISPR-Cas9 Delivery in Clinical Applications
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“So far, a variety of nanocarriers, including synthetic organic nanoparticles based on liposomes [15–18] and polymers [19–21], inorganic nanoparticles such as gold nanoparticles [22–29], nano-silicon dioxide [30–35], nano-iron oxide [36–38], etc., as well as native extracellular vesicles [39–42],…”
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“…(human orthologs) Mature sequence (human orthologs) Heart linked function Cardiac pathology associated function mir-1 hsa-mir-1-1 hsa-miR-1-3p Regulators of cardiac muscle growth and differentiation (Zhao et al., 2005, 2007; Liu et al., 2007, 2008; Callis et al., 2009; Sluijter et al., 2010).”
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New Therapeutics for Extracellular Vesicles: Delivering CRISPR for Cancer Treatment
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“LNP-mediated gene editing via the CRISPR/Cas9 system has been widely reported in the brain [45], lungs [46], liver [47], and tumor tissues [48].”
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Theranostic Lipid Nanoparticles for Renal Cell Carcinoma
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Active Delivery of CRISPR System Using Targetable or Controllable Nanocarriers
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A targeting delivery system for effective genome editing in leukemia cells to reverse malignancy
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CRISPR-Cas9 in Alzheimer’s disease: Therapeutic trends, modalities, and challenges
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Deep Tumor Penetration of CRISPR-Cas System for Photothermal-Sensitized Immunotherapy via Probiotics
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Self-citationExploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing
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Transplacental Gene Delivery (TPGD) as a Noninvasive Tool for Fetal Gene Manipulation in Mice
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Nonviral Delivery of CRISPR/Cas Systems in mRNA Format
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A Lung-Expressing mRNA Delivery Platform with Tunable Activity in Hypoxic Environments
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Current Knowledge on CRISPR Strategies Against Antimicrobial-Resistant Bacteria
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Harnessing mRNA-lipid nanoparticles as innovative therapies for autoimmune diseases
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Metadata
- Authors
- Liu, J., Chang, J., Jiang, Y., Meng, X., Sun, T., Mao, L., Xu, Q., & Wang, M.
- Affiliations
- Chinese Academy of Sciences (CN)
- Jilin University (CN)
- Tufts University (US)
- Corresponding author
- Xu, Qiaobing
- Wang, Ming
- Venue
- Advanced Materials
- Publisher
- Wiley
- Published
- 2019-06-19
- Volume
- 31
- Issue
- 33
- Pages
- e1902575-e1902575
- ISSN
- 0935-9648 (ISSN-L)
- 1521-4095
- PMC
- PMC6732788
- OpenAlex
- W2950978761
- Cited by
- 373
- References
- 33
- Citations by year
- 2026: 33 · 2025: 52 · 2024: 53 · 2023: 55 · 2022: 74 · 2021: 53 · 2020: 45 · 2019: 8
- FWCI
- 14.88
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- Locations
- Advanced Materials (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Basic Research Program of China (2017YFA0208100, 2016YFA0200104)
- National Natural Science Foundation of China (21778056, 21790390, 21790391, 21621062, 21435007)
- National Science Foundation (UG3 TR002636‐01, NIH R21 EB024041)
- 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/Cas9 genome editing, lipid nanoparticles, hepatocyte targeting, in vivo gene editing
- MeSH terms
- Proprotein Convertase 9, Gene Editing, CRISPR-Associated Protein 9, RNA, Guide, CRISPR-Cas Systems, Animals, Biological Transport, Humans, Lipids, Oxidation-Reduction, RNA, Messenger, RNA, Guide, Kinetoplastida, Gene Transfer Techniques, Cell Line, Tumor, Green Fluorescent Proteins, Mice, Nanoparticles, Gene Knockdown Techniques
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