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Review

How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?

1
NanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, Spain
2
Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), 28029 Madrid, Spain
3
Bioaraba, NanoBioCel Resarch Group, 01009 Vitoria-Gasteiz, Spain
*
Authors to whom correspondence should be addressed.
Academic Editor: Anna Maria Piras
Int. J. Mol. Sci. 2021, 22(14), 7545; https://doi.org/10.3390/ijms22147545
Received: 22 June 2021 / Accepted: 10 July 2021 / Published: 14 July 2021
(This article belongs to the Special Issue Non-viral Gene Delivery: Hurdles and Promises)
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. View Full-Text
Keywords: non-viral vectors; gene therapy; nanotechnology; gene delivery; clinical translation non-viral vectors; gene therapy; nanotechnology; gene delivery; clinical translation
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MDPI and ACS Style

Sainz-Ramos, M.; Gallego, I.; Villate-Beitia, I.; Zarate, J.; Maldonado, I.; Puras, G.; Pedraz, J.L. How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? Int. J. Mol. Sci. 2021, 22, 7545. https://doi.org/10.3390/ijms22147545

AMA Style

Sainz-Ramos M, Gallego I, Villate-Beitia I, Zarate J, Maldonado I, Puras G, Pedraz JL. How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? International Journal of Molecular Sciences. 2021; 22(14):7545. https://doi.org/10.3390/ijms22147545

Chicago/Turabian Style

Sainz-Ramos, Myriam, Idoia Gallego, Ilia Villate-Beitia, Jon Zarate, Iván Maldonado, Gustavo Puras, and Jose L. Pedraz 2021. "How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?" International Journal of Molecular Sciences 22, no. 14: 7545. https://doi.org/10.3390/ijms22147545

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