The DOI matches its Crossref record; 8 of the 8 references it cites were found in a registry or an index.
Biological Gene Delivery Vehicles: Beyond Viral Vectors
Open accessAbstract
Gene therapy covers a broad spectrum of applications, from gene replacement and knockdown for genetic or acquired diseases such as cancer, to vaccination, each with different requirements for gene delivery. Viral vectors and synthetic liposomes have emerged as the vehicles of choice for many applications today, but both have limitations and risks, including complexity of production, limited packaging capacity, and unfavorable immunological features, which restrict gene therapy applications and hold back the potential for preventive gene therapy. While continuing to improve these vectors, it is important to investigate other options, particularly nonviral biological agents which include bacteria, bacteriophage, virus-like particles (VLPs), erythrocyte ghosts, and exosomes. Exploiting the natural properties of these biological entities for specific gene delivery applications will expand the repertoire of gene therapy vectors available for clinical use. Here, we review the prospects for nonviral biological delivery vehicles as gene therapy agents with focus on their unique evolved biological properties and respective limitations and potential applications. The potential of these nonviral biological entities to act as clinical gene therapy delivery vehicles has already been shown in clinical trials using bacteria-mediated gene transfer and with sufficient development, these entities will complement the established delivery techniques for gene therapy applications.
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Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes
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Self-citationsiRNA versus miRNA as therapeutics for gene silencing
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Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering
Billingsley, M. M., Singh, N., Ravikumar, P., Zhang, R., June, C. H., & Mitchell, M. J. (2020) · Nano Letters · doi:10.1021/acs.nanolett.9b04246 · Cited by 636
Exosomes are natural carriers of exogenous siRNA to human cells in vitro
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Metadata
- Authors
- Seow, Y., & Wood, M. J.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2009-05
- Volume
- 17
- Issue
- 5
- Pages
- 767-777
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2153138904
- Cited by
- 327
- References
- 8
- FWCI
- 9.72
- Type
- journal article
- Language
- en
- Open access
- hybrid
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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