In:
Current Gene Therapy, Bentham Science Publishers Ltd., Vol. 21, No. 5 ( 2021-12-23), p. 431-451
Abstract:
Gene delivery is a promising technology for treating diseases linked to abnormal
gene expression. Since nucleic acids are the therapeutic entities in such approach, a transfecting vector is required because the macromolecules are not able to efficiently enter the cells by themselves.
Viral vectors have been evidenced to be highly effective in this context; however, they suffer from fundamental drawbacks, such as the ability to stimulate immune responses. The development of
synthetic vectors has accordingly emerged as an alternative. Objectives: Gene delivery by using non-viral vectors is a multi-step process that poses many challenges,
either regarding the extracellular or intracellular media. We explore the delivery pathway and afterwards, we review the main classes of non-viral gene delivery vectors. We further focus on the progresses
concerning polyethylenimine-based polymer-nucleic acid polyplexes, which have emerged as one of the most efficient systems for delivering genetic material inside the cells. Discussion: The complexity of the whole transfection pathway, along with a lack of fundamental understanding,
particularly regarding the intracellular trafficking of nucleic acids complexed to non-viral vectors, probably justifies the current (beginning of 2021) limited number of formulations that have
progressed to clinical trials. Truly, successful medical developments still require a lot of basic research. Conclusion: Advances in macromolecular chemistry and high-resolution imaging techniques will be
useful to understand fundamental aspects towards further optimizations and future applications. More investigations concerning the dynamics, thermodynamics and structural parameters of polyplexes
would be valuable since they can be connected to the different levels of transfection efficiency hitherto evidenced.
Type of Medium:
Online Resource
ISSN:
1566-5232
DOI:
10.2174/1566523221666210705130238
Language:
English
Publisher:
Bentham Science Publishers Ltd.
Publication Date:
2021
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