How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?
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-...
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-07-01
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| Series: | International Journal of Molecular Sciences |
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| Online Access: | https://www.mdpi.com/1422-0067/22/14/7545 |
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| author | Myriam Sainz-Ramos Idoia Gallego Ilia Villate-Beitia Jon Zarate Iván Maldonado Gustavo Puras Jose Luis Pedraz |
| author_facet | Myriam Sainz-Ramos Idoia Gallego Ilia Villate-Beitia Jon Zarate Iván Maldonado Gustavo Puras Jose Luis Pedraz |
| author_sort | Myriam Sainz-Ramos |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-10T09:37:23Z |
| format | Article |
| id | doaj.art-7e813f68a4d04db78cc672e797a9504f |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T09:37:23Z |
| publishDate | 2021-07-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-7e813f68a4d04db78cc672e797a9504f2023-11-22T04:00:17ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672021-07-012214754510.3390/ijms22147545How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?Myriam Sainz-Ramos0Idoia Gallego1Ilia Villate-Beitia2Jon Zarate3Iván Maldonado4Gustavo Puras5Jose Luis Pedraz6NanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, SpainNanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, SpainNanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, SpainNanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, SpainNanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, SpainNanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, SpainNanoBioCel Group, Faculty of Pharmacy, University of the Basque Country UPV/EHU, 01006 Vitoria-Gasteiz, SpainEfficient 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.https://www.mdpi.com/1422-0067/22/14/7545non-viral vectorsgene therapynanotechnologygene deliveryclinical translation |
| spellingShingle | Myriam Sainz-Ramos Idoia Gallego Ilia Villate-Beitia Jon Zarate Iván Maldonado Gustavo Puras Jose Luis Pedraz How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? International Journal of Molecular Sciences non-viral vectors gene therapy nanotechnology gene delivery clinical translation |
| title | How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? |
| title_full | How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? |
| title_fullStr | How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? |
| title_full_unstemmed | How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? |
| title_short | How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy? |
| title_sort | how far are non viral vectors to come of age and reach clinical translation in gene therapy |
| topic | non-viral vectors gene therapy nanotechnology gene delivery clinical translation |
| url | https://www.mdpi.com/1422-0067/22/14/7545 |
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