Production and clinical development of nanoparticles for gene delivery
Open accessAbstract
Gene therapy is a promising strategy for specific treatment of numerous gene-associated human diseases by intentionally altering the gene expression in pathological cells. A successful clinical application of gene-based therapy depends on an efficient gene delivery system. Many efforts have been attempted to improve the safety and efficiency of gene-based therapies. Nanoparticles have been proved to be the most promising vehicles for clinical gene therapy due to their tunable size, shape, surface, and biological behaviors. In this review, the clinical development of nanoparticles for gene delivery will be particularly highlighted. Several promising candidates, which are closest to clinical applications, will be briefly reviewed. Then, the recent developments of nanoparticles for clinical gene therapy will be identified and summarized. Finally, the development of nanoparticles for clinical gene delivery in future will be prospected.
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Nanoparticles have low production cost.
“The other advantage associated with NPs is low production cost [22].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [22]
Non-viral gene delivery involves delivering a circular, double-stranded plasmid DNA encoding the gene of interest directly into the target cell type.
“Non-viral gene delivery involves delivering a circular, double-stranded plasmid DNA encoding the gene of interest directly into the target cell type [22].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [22]
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References 83 · Cited by 274
- Authors
- Chen, J., Guo, Z., Tian, H., & Chen, X.
- Affiliations
- Chinese Academy of Sciences (CN)
- Corresponding author
- Tian, Huayu
- Venue
- Molecular Therapy - Methods & Clinical Development
- Publisher
- Elsevier BV
- Published
- 2016
- Volume
- 3
- Pages
- 16023
- ISSN
- 2329-0501 (ISSN-L)
- PMC
- PMC4822651
- OpenAlex
- W2319745661
- Cited by
- 274
- References
- 83
- Citations by year
- 2026: 13 · 2025: 22 · 2024: 27 · 2023: 22 · 2022: 42 · 2021: 38 · 2020: 28 · 2019: 30 · 2018: 22 · 2017: 21 · 2016: 9
- FWCI
- 2.47
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc-sa)
- PubMed Central (publishedVersion, cc-by-nc-sa)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- 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 › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- nanoparticles, gene delivery, clinical gene therapy, gene therapy vectors, nanoparticle design
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