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

• Published in: International journal of molecular sciences 2021-07, Vol.22 (14), p.7545
• Main Authors: Sainz-Ramos, Myriam, Gallego, Idoia, Villate-Beitia, Ilia, Zarate, Jon, Maldonado, Iván, Puras, Gustavo, Pedraz, Jose Luis
• Format: Article
• Language: English
• Subjects: Biocompatibility; clinical translation; Clinical trials; Commercialization; Coronaviruses; CRISPR; Cystic fibrosis; Cytoplasm; Disease; Drugs; Efficiency; Expression vectors; gene delivery; Gene expression; Gene therapy; Gene transfer; Genetic disorders; Genetic engineering; Genomes; Immunogenicity; Knowledge acquisition; Laboratories; MicroRNAs; Molecular biology; Mutation; Nanotechnology; non-viral vectors; Proteins; Review; Severe acute respiratory syndrome coronavirus 2; Toxicity; Vectors (Biology)
• ISSN: 1422-0067; 1661-6596; 1422-0067
• DOI: 10.3390/ijms22147545

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.