How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?
Fecha
2021-07-14Autor
Sainz Ramos, Myriam
Gallego Garrido, Idoia
Villate Beitia, Ane Ilia
Maldonado Pérez, Iván
Puras Ochoa, Gustavo
Metadatos
Mostrar el registro completo del ítem
International Journal of Molecular Sciences 22(14) : (2021) // Article ID 7545
Resumen
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.
Colecciones
Excepto si se señala otra cosa, la licencia del ítem se describe como 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).