Therapeutic in vivo delivery of gene editing agents
Broad Institute · Howard Hughes Medical Institute · +2 more institutions
Indexed incrossrefpubmed
Abstract
In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.
Citation impact
467
total citations
- FWCI
- 76.99
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- 100%
- References
- 238
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Authors
3Topics & keywords
Topics
Keywords
- Genome editing
- Biology
- In vivo
- Gene delivery
- Computational biology
- Genetic enhancement
- Gene
- Bioinformatics
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