2019
DOI: 10.1186/s40824-019-0156-z
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Recent advances in the development of gene delivery systems

Abstract: BackgroundGene delivery systems are essentially necessary for the gene therapy of human genetic diseases. Gene therapy is the unique way that is able to use the adjustable gene to cure any disease. The gene therapy is one of promising therapies for a number of diseases such as inherited disorders, viral infection and cancers. The useful results of gene delivery systems depend open the adjustable targeting gene delivery systems. Some of successful gene delivery systems have recently reported for the practical a… Show more

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Cited by 355 publications
(227 citation statements)
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“…Despite these unique strengths, they are still plagued by inherent issues such as the limited size of NAs that can be packed and delivered, random recombination (i.e., oncogenic potential), cytotoxicity, and immunogenicity [38,39]. These concerns, together with the high costs related to large-scale production and quality control, have steered research towards non-viral carriers [40,41]. Since the inception of inorganic matter for plasmid delivery in the 1970s [49], the last decades have thus witnessed a surge of interest in non-viral systems [1,[50][51][52].…”
Section: Nucleic Acid Description Site Of Action Applications/pathwaymentioning
confidence: 99%
“…Despite these unique strengths, they are still plagued by inherent issues such as the limited size of NAs that can be packed and delivered, random recombination (i.e., oncogenic potential), cytotoxicity, and immunogenicity [38,39]. These concerns, together with the high costs related to large-scale production and quality control, have steered research towards non-viral carriers [40,41]. Since the inception of inorganic matter for plasmid delivery in the 1970s [49], the last decades have thus witnessed a surge of interest in non-viral systems [1,[50][51][52].…”
Section: Nucleic Acid Description Site Of Action Applications/pathwaymentioning
confidence: 99%
“…The FDA has predicted, based on current clinical pipelines, that by 2025 10-20 new cell and gene therapies will be approved annually (FDA, 2019). Multiple methods to achieve gene transduction during gene therapy have been proposed, including liposomal transfer, polymeric transfer, and use of viral vectors (Sung & Kim, 2019). One common viral vector used, especially for in vivo gene transfer, is adeno-associated virus (AAV).…”
Section: Introductionmentioning
confidence: 99%
“…Basically, physical methods are vector-free systems based on a controlled and reversible deformation of cytoplasmic membrane during short periods of time that allow the entry of genetic material on target cells [22]. Although highly effective, these methods are normally restricted to ex vivo gene therapy, which is mainly due to the challenge that represents the fine control of physical parameters that produce the formation of transient pores into the cellular membrane in vivo conditions.…”
Section: Physical Methodsmentioning
confidence: 99%
“…Such technological limitations can result not only in a loss of action but also in an increase of the cellular toxicity [45]. Transient pores on cellular and even nuclear membranes can be induced by the application of external electrical pulses, whose amplitude and duration are controlled, and depend on the particular characteristics of the target cell [22]. Pores can also be created if a cell´s membrane is mechanically deformed when cells are forced to pass through microfluidic-based channels which the diameter is smaller than that of the cell [46].…”
Section: Physical Methodsmentioning
confidence: 99%
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