Delivery Approaches for Therapeutic Genome Editing and Challenges
Abstract
:1. Introduction
2. Fundamentals of Gene Editing
3. Gene-Editing Tools
4. Delivery Strategies for Therapeutic Applications
4.1. Viral Systems
4.1.1. Adenoviral Vectors
4.1.2. Adeno-Associated Viral Vectors
- i.
- Packaging CRISPR-Cas Components into AAV Vectors
- ii.
- AAV-Mediated Delivery for Therapeutic Applications
4.1.3. Lentiviral Vectors
- i.
- LV-Mediated Delivery for Therapeutic Applications
- ii.
- LV-Mediated Delivery for Genomic Screenings
4.2. Electroporation
4.3. Lipid Nanoparticles
4.4. Hydrodynamic Delivery
- i.
- HD for Therapeutic Applications
- ii.
- HD for Generation of Animal Models of Human Disease
4.5. Cell-Penetrating Peptides
5. Conclusions
Author Contributions
Funding
Acknowledgments
Conflicts of Interest
References
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Method | Delivery Material | Approach | Carrying Capacity | Advantages | Disadvantages |
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Adenoviruses | Double-stranded DNA | in vivo | 7.5–30 kb |
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Adeno-associated viruses | single-stranded DNA | in vivo | 4.8 kb |
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Lentiviruses | single-stranded RNA | in vivo | 8 kb |
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Hydrodynamic delivery | DNA plasmid, ribonucleoprotein (RNP) | in vivo |
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Electroporation | DNA plasmid, mRNA, RNP | ex vivo |
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Lipid nanoparticles | DNA plasmid, mRNA, RNP | in vivo |
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Cell-penetrating peptides | Protein, RNP | in vivo |
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Ates, I.; Rathbone, T.; Stuart, C.; Bridges, P.H.; Cottle, R.N. Delivery Approaches for Therapeutic Genome Editing and Challenges. Genes 2020, 11, 1113. https://doi.org/10.3390/genes11101113
Ates I, Rathbone T, Stuart C, Bridges PH, Cottle RN. Delivery Approaches for Therapeutic Genome Editing and Challenges. Genes. 2020; 11(10):1113. https://doi.org/10.3390/genes11101113
Chicago/Turabian StyleAtes, Ilayda, Tanner Rathbone, Callie Stuart, P. Hudson Bridges, and Renee N. Cottle. 2020. "Delivery Approaches for Therapeutic Genome Editing and Challenges" Genes 11, no. 10: 1113. https://doi.org/10.3390/genes11101113