Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing
Abstract
:1. Introduction
2. Nonviral Delivery Systems for Genome Editing
2.1. Lipid-Based Vesicles for Genome Editing
2.1.1. Cationic Lipid Nanoparticles
2.1.2. Lipid Nanoshells
2.2. Polymer-Based Delivery Systems
2.2.1. Polyethylenimine
2.2.2. Polyamidoamine
2.2.3. Other Synthetic Polymers
2.2.4. Chitosan
2.3. Extracellular Vesicles
2.3.1. Exosomes
2.3.2. Microvesicles
2.4. Peptide/Protein-Based Systems
2.4.1. Peptides
2.4.2. Proteins
2.4.3. Virus-Like Particles
3. Targeting Ligands for Genome Editing
3.1. Chemical Ligands for Targeted Delivery
3.2. Peptide Ligands for Targeted Delivery
3.3. Antibodies and Aptamers for Targeted Delivery
4. Delivery Strategies for Genome Editing
4.1. Chemical/Molecular Delivery Strategies
4.1.1. Chemical/Peptide-Enhanced Delivery
4.1.2. Light-Enhanced Delivery
4.1.3. Glutathione-Triggered Delivery
4.1.4. pH-Responsive Delivery
4.2. Physical Delivery Strategies
5. Challenges and Future Perspectives
6. Conclusions
Author Contributions
Funding
Conflicts of Interest
Abbreviations
Abbreviation | Full Name |
ANGPTL3 | Angiopoietin like 3 |
APOC3 | Apolipoprotein C |
ASGP | Asialoglycoprotein |
Cas | Clustered regularly interspaced short palindromic repeats-associated protein |
CDC6 | Cell division control protein 6 |
C/EBPα | CCAAT enhancer binding protein alpha |
Chol-PEG | Polyethylene glycol-linked cholesterol |
CPP | Cell-penetrating peptide |
CRISPR | Clustered regularly interspaced short palindromic repeats |
crRNAs | CRISPR RNAs |
CTNNB1 | β-catenin |
DDX3 | DEAD-box RNA helicase 3 |
DNMT1 | DNA-methyltransferase 1 |
DOPE | 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine |
DOTAP | 1,2-dioleoyl-3-trimethylammoniumpropane |
DSPE-PEG-2000 | 1,2-distearoyl-sn-glycero-3-phosphoethanolamine-N-[amino(polyethyleneglycol)-2000] |
EGFP | Enhanced green fluorescent protein |
EMX1 | Empty spiracles homeobox 1 |
FKBP | FK506-binding protein |
FRB | FKBP-rapamycin binding domain |
GFP | Green fluorescent protein |
GPX4 | Glutathione peroxidase-4 |
GSH | Glutathione |
HPD | Hydroxyphenylpyruvate dioxygenase |
HPV16 E6/E7 | Human papillomavirus 16 E6/E7 |
ICAM1 | Intercellular adhesion molecule 1 |
IDUA | α-L-iduronidase |
IQGAP1 | IQ motif-containing GTPase-activating protein 1 |
miRNA | MicroRNA |
MTH1 | MutT homolog 1 |
NIR | Near-infrared |
Nrf2 | Nuclear factor erythroid-2-related factor 2 |
PAMAM | Polyamidoamine |
PARP1 | Poly (ADP-ribose) polymerase-1 |
pCas9 | Plasmid DNA encoding clustered regularly interspaced short palindromic Repeats-associated protein 9 |
PCSK9 | Protease proprotein convertase subtilisin/kexin type 9 |
PD-L1 | Programmed death-ligand 1 |
PEG | Polyethylene glycol |
PEI | Polyethyleneimine |
PLK1 | Polo-like kinase 1 |
PPABLG | Poly(γ-4-((2-(piperidin-1-yl)ethyl)aminomethyl)benzyl-L-glutamate) |
psgRNA | Plasmid DNA encoding single-guide RNA |
RHBDF1 | Rhomboid 5 homolog 1 |
ROS | Reactive oxygen species |
RUNX1 | Runt-related transcription factor 1 |
sgRNA | Single-guide RNA |
siRNA | Small inhibitory RNA |
STAT3 | Signal transducer and activator of transcription 3 |
STING | Stimulator of IFN genes |
TALENs | Transcription activator-like effector nucleases |
tracrRNAs | Trans-activating CRISPR RNAs |
TYR | Tyrosinase |
VEGF-A | Vascular endothelial growth factor A |
VEGFR2 | Vascular endothelial growth factor receptor 2 |
VLP | Virus-like particle |
VSV | Vesicular stomatitis virus |
ZFN | Zinc-finger nuclease |
β-CD | β-cyclodextrin |
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Material | Composition | Cas9 | sgRNA | Target Gene | Editing Type | Target Disease | Ref |
---|---|---|---|---|---|---|---|
Lipid | DOTAP, DOPE, DSPE-PEG, Chol | Plasmid DNA | Plasmid DNA | HPV16E6, E7 | Knockout | Cervical cancer | [30] |
DOTAP, DOPE, DSPE-PEG | Plasmid DNA | Plasmid DNA | Iduronidase | Knock-in | Mucopolysaccharidosis type I | [31] | |
cKK-E12, DOPE, Chol, C14-PEG | mRNA | sgRNA | PCSK9 | Knockout | Hypercholesterolemia | [32] | |
Protamine, chondroitin sulfate, DOTAP, DOPE, DSPE-PEG | Plasmid DNA | Plasmid DNA | PLK1 | Knockout | Melanoma | [33] | |
Gold nanoparticle, TAT, DOTAP, DOPE, DSPE-PEG | Protein | Plasmid DNA | PLK1 | Knockout | Melanoma | [34] | |
MSN, DOTAP, DOPE, DSPE-PEG, Chol | Protein | sgRNA | PCSK9, Apolipo-protein C3, Angiopoietin-like 3 | Knockout | Hypercholesterolemia | [35] | |
Polymer | PEGylated hyaluronan polymer with R8-RGD tandem peptide, fluorinated polyethyleneimine | Plasmid DNA | Plasmid DNA | MTH1 | Knockout | Ovarian cancer | [36] |
Semiconducting polymers composed of eicosane, PEG, diketopyrrolopyrrole and fluorinated PEI | Plasmid DNA | Plasmid DNA | MTH1 | Knockout | - | [37] | |
Branched β-CD-PEI/Ad-PAMAM/Ad-PEG-TAT | Plasmid DNA | Plasmid DNA | Retinoschisin 1, GFP | Knock-in | X-linked juvenile retinoschisis | [38] | |
Quaternary ammonium-terminated poly(propylene oxide)/Pluronic F127 | Plasmid DNA | Plasmid DNA | HPV18-E7 | Knockout | - | [39] | |
Extracellular vesicle | Exosome from SKOV3 | Protein | sgRNA | Poly (ADP-ribose) polymerase 1 | Knockout | Ovarian cancer | [40] |
Exosome modified with TLS11a aptamer-Chol | Protein | sgRNA | WNT10B | Knockout | Hepatocellular cancer | [41] | |
Microvesicle | Protein | sgRNA | IQGAP1 | Knockout | Hepatocellular cancer | [42] | |
Microvesicle with anti-CD19 CAR | Protein | sgRNA | MYC | Knockout | Lymphoma | [43] | |
Peptide/protein | R7L10 peptide-based micelle | Protein | sgRNA | β-secretase 1 | Knockout | Alzheimer’s disease | [44] |
Poly-L-arginine | Protein | sgRNA | GFP, Nuclear Receptor Interacting Protein 1 | Knockout | Obesity | [45] | |
VLP composed of VSV-G | Protein | sgRNA | GFP | Knockout | - | [46] | |
VLP composed of Gag-Cas9 fusion, VSV-G, BaEVRLess, sgRNA | Protein | sgRNA | EMX1, DDX3, Tyrosinase, HPD | Knockout | Hereditary tyrosinemia type I (HT1) | [47] | |
VLP composed of VSV-G, FKBP12-Gag, FRB-Cas9 | Protein | sgRNA | Dystrophin | Knockout | Duchenne muscular dystrophy | [48] |
Targeting | Material | Composition | Cas9 Type | sgRNA Type | Target Gene | Editing Type | Target Disease | Ref |
---|---|---|---|---|---|---|---|---|
Chemical ligand-based targeting | Lipid | Lipid shell composed of Gal-PEG-DSPE, DOTAP, DOPE, Chol, TAT with AuNP | Protein | sgRNA | PCSK9 | Knockout | High LDL cholesterol | [111] |
Polymer | Polymeric nanoparticle composed of cationic, anionic, imidazole, biodegradable crosslinker, acrylate-mPEG-ATRA | Protein | sgRNA | Stop-tdTomato | Knockout | - | [112] | |
Lipid | Liposome composed of DOTAP, cholesterol, folate-PEG-succinyl-Chol | Plasmid DNA | Plasmid DNA | DNMT1 | Knockout | Ovarian cancer | [113] | |
Peptide-based targeting | Lipid | Liposome-templated hydrogel nanoparticle composed of palmitoyl-transportan-iRGD | Protein | sgRNA | GFP | Knockout | - | [114] |
Lipid | Liposome-templated hydrogel nanoparticle composed of PEI-CD, PEI-AD, mHph3, iRGD | Protein | sgRNA | PLK-1 | Knockout | Brain cancer | [115] | |
Polymer | Core-shell polymeric nanoparticle composed of PF33, hyaluronan, RGD-R8-PEG | Plasmid DNA | Plasmid DNA | MTH1 | Knockout | Ovarian cancer | [36] | |
Antibody and aptamer ligand-based targeting | Protein | Protamine-based nanoparticle composed of CaCO3, As1411/TAT functionalized carboxymethyl chitosan | Plasmid DNA | Plasmid DNA | CTNNB1 | Knockout | - | [116] |
Protein | Protamine-based nanoparticle composed of CaCO3, As1411/mucin 1 aptamer functionalized heparin | Plasmid DNA | Plasmid DNA | Focal adhesion kinase | Knockout | - | [117] | |
Polymer | Peptide-lipid micelle composed of PEG-PEI-Chol, LC09 aptamer | Plasmid DNA | Plasmid DNA | VEGF-A | Knockout | Osteo-sarcoma | [118] |
Strategy | Mechanism | Material | Composition | Cas9 Type | sgRNA Type | Target Gene | Editing Type | Ref |
---|---|---|---|---|---|---|---|---|
Endosomal escape | Cationic charge-induced membrane disruption | Liposome | Pardaxin peptide-modified cationic liposome | Plasmid DNA | Plasmid DNA | CDC6 | Knockout | [131] |
Polymer | Glucuronylglucosyl-β-cyclodextrin conjugated dendrimer | Protein | sgRNA | Rosa26 | Knockout | [132] | ||
Polymer | Cationic diethylenetriamine-conjugated amphiphilic polyaspartamide derivatives | mRNA | sgRNA | Luciferase | Knockout | [133] | ||
Polypeptide/polymer | α-helical polypeptide incorporating PEG-polythymine 40-based nanocomplex | Plasmid DNA | Plasmid DNA | PLK1 | Knockout | [126] | ||
Photo-responsive delivery | Photodynamic effect | Polymer | Chlorin e6-loaded/cationic iRGD-modified copolymer-coated polymeric micelle | Protein | sgRNA | Nrf2 | Knockout | [134] |
Polymer | PEI-thioester-semiconducting polymer | Plasmid DNA | Plasmid DNA | GFP | Knockout | [135] | ||
Photothermal effect | Lipid | Lipid coated-gold NP-TAT peptide | Plasmid DNA | Plasmid DNA | PLK-1 | Knockout | [127] | |
Polymer | Semiconducting polymers composed of eicosane, PEG, diketopyrrolopyrrole, and fluorinated PEI | Plasmid DNA | Plasmid DNA | MTH1 | Knockout | [37] | ||
GSH-responsive delivery | GSH-mediated bond cleavage | Lipid | Cationic lipid nanoparticle composed of 8-O14B, DOPE, Chol, C16-PEG-ceramide | Protein | sgRNA | GFP | Knockout | [128] |
Lipid | Cationic lipid nanoparticle composed of BAMEA-O16B, DOPE, DSPE-PEG, Chol | mRNA | sgRNA | PCSK9 | Knockout | [136] | ||
Polymer | Cationic block copolymer composed of poly(Asp-AED-ICA)-PEG | Protein | sgRNA | mCherry | Knockout | [137] | ||
Polymer | Polymeric nanoparticle composed of cationic, anionic, imidazole, biodegradable crosslinker, acrylate-mPEG-ATRA | Protein | sgRNA | Stop-tdTomato | Knockout | [112] | ||
pH-responsive delivery | pH-dependent degradation | Polymer | Ortho ester polymer/PEI | Protein | sgRNA | Survivin | Knockout | [138] |
Polymer | PLys100-CAmPEG77 | Protein | sgRNA | STAT3, RUNX1 | Knockout | [129] | ||
pH-dependent charge reversion | Lipid | 1-Aminoethyl)iminobis [N-(oleoylcysteinyl-1- aminoethyl)propionamide lipid | Plasmid DNA | Plasmid DNA | GFP | Knockout | [139] | |
Physical delivery | Mechanical membrane deformation | Nanoneedle | Silicon based atomic force microscope cantilever type nanoneedle | Protein | sgRNA | GFP, Nestin | Knockout | [140] |
Nanoblade | Photolithography and reactive ion etching fabricated microfluidic chip with silicon nanoblade | Protein | sgRNA | C/EBPα | Knockout | [130] | ||
Ultrasound mediated membrane penetration | Metal | Cysteine modified asymmetric gold nanowire | Protein | sgRNA | GFP | Knockout | [141] |
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Share and Cite
Kim, D.; Le, Q.-V.; Wu, Y.; Park, J.; Oh, Y.-K. Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing. Pharmaceutics 2020, 12, 1233. https://doi.org/10.3390/pharmaceutics12121233
Kim D, Le Q-V, Wu Y, Park J, Oh Y-K. Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing. Pharmaceutics. 2020; 12(12):1233. https://doi.org/10.3390/pharmaceutics12121233
Chicago/Turabian StyleKim, Dongyoon, Quoc-Viet Le, Yina Wu, Jinwon Park, and Yu-Kyoung Oh. 2020. "Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing" Pharmaceutics 12, no. 12: 1233. https://doi.org/10.3390/pharmaceutics12121233