Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 22, Issue 14, Pages -Publisher
MDPI
DOI: 10.3390/ijms22147545
Keywords
non-viral vectors; gene therapy; nanotechnology; gene delivery; clinical translation
Funding
- Basque Country Government (Department of Education, University and Research) [IT907-16]
- Spanish Ministry of Science and Innovation [PID2019-106199RB-C21]
- University of the Basque Country (UPV/EHU) [ESPDOC19/47, PIF17/79]
- CIBER of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), an initiative of the Carlos III Health Institute (ISCIII)
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This review highlights the critical importance of efficient delivery of genetic material into cells for translating gene therapy into clinical practice, with a focus on non-viral vector systems as a promising alternative to virus-based gene delivery. The challenges and potential strategies for clinical application of non-viral vectors in mainstream medicine, including efficiency, biocompatibility, and long-lasting effect, are discussed in order to promote safe and efficient non-viral gene delivery systems for clinical applications in the gene therapy field.
Efficient delivery of genetic material into cells is a critical process to translate gene therapy into clinical practice. In this sense, the increased knowledge acquired during past years in the molecular biology and nanotechnology fields has contributed to the development of different kinds of non-viral vector systems as a promising alternative to virus-based gene delivery counterparts. Consequently, the development of non-viral vectors has gained attention, and nowadays, gene delivery mediated by these systems is considered as the cornerstone of modern gene therapy due to relevant advantages such as low toxicity, poor immunogenicity and high packing capacity. However, despite these relevant advantages, non-viral vectors have been poorly translated into clinical success. This review addresses some critical issues that need to be considered for clinical practice application of non-viral vectors in mainstream medicine, such as efficiency, biocompatibility, long-lasting effect, route of administration, design of experimental condition or commercialization process. In addition, potential strategies for overcoming main hurdles are also addressed. Overall, this review aims to raise awareness among the scientific community and help researchers gain knowledge in the design of safe and efficient non-viral gene delivery systems for clinical applications to progress in the gene therapy field.
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