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-...

Full description

Saved in:
Bibliographic Details
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)
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary: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.
ISSN:1422-0067
1661-6596
1422-0067
DOI:10.3390/ijms22147545