Non-viral gene delivery systems
Abstract
Non-viral gene delivery systems have the potential to create viable pharmaceuticals from nucleic acids. These DNA delivery systems contain lipids and/or cationic polymers. In order for these systems to be developed into commercial products, several barriers must be overcome. These include obstacles in manufacturing, formulation and stability. In vivo, problems of extracellular non-specific interactions and intracellular trafficking to the nucleus are also encountered. Recent progress has been made in overcoming these issues.
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Relatively low gene delivery efficiency and transgene expression have been the major barriers in non-viral vectors-based gene therapy.
“So far, relatively low gene delivery efficiency and transgene expression have been the major barriers in non-viral vectors-based gene therapy [76, 77].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [77]
Non-viral vectors face two main limitations in systemic delivery in vivo: serum stability of polyplexes after systemic administration and relatively low transfection efficiency in targeted tissues after administration.
“There are two main limitations of non-viral vectors-mediated CRISPR/Cas9 delivery in vivo. The first one is the serum stability of polyplexes after systemic administration [73]. The second one is the relatively low transfection efficiency in the targeted tissues after administration in vivo [76, 77].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [77]
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Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities
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van der Veen, I., Heredero Berzal, A., Koster, C., ten Asbroek, A. L. M. A., Bergen, A. A., & Boon, C. J. F. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25021267 · Cited by 14 · see citing works
- Authors
- Davis, M. E.
- Affiliations
- California Institute of Technology (US)
- Corresponding author
- Davis, Mark E
- ORCID
- Davis, Mark E
- Venue
- Current Opinion in Biotechnology
- Publisher
- Elsevier BV
- Published
- 2002-04
- Volume
- 13
- Issue
- 2
- Pages
- 128-131
- ISSN
- 0958-1669 (ISSN-L)
- OpenAlex
- W1969497292
- Cited by
- 419
- References
- 34
- Citations by year
- 2026: 1 · 2025: 4 · 2024: 12 · 2023: 8 · 2022: 9 · 2021: 14 · 2020: 18 · 2019: 10 · 2018: 12 · 2017: 14 · 2016: 21 · 2015: 18 · 2014: 18 · 2013: 22 · 2012: 30
- FWCI
- 1.52
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Current Opinion in Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- CaltechAUTHORS (California Institute of Technology) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Keywords
- non-viral gene delivery, nucleic acid therapeutics, cationic polymers, lipoplexes, intracellular trafficking, nuclear delivery
- MeSH terms
- Biological Transport, Active, Cell Nucleus, DNA, Drug Carriers, Drug Stability, Endocytosis, Genetic Vectors, Humans, Polymers, Genetic Therapy, Drug Delivery Systems, Gene Transfer Techniques, Lipid Metabolism
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