The Development of Functional Non-Viral Vectors for Gene Delivery

Patil, Suryaji; Gao, Yongguang; Lin, Xiuping; Liu, Yu; Dang, Kai; Tian, Ye; Zhang, Wenjuan; Jiang, Shuai; Qadir, Abdul; Qian, Airong

doi:10.3390/ijms20215491

Cited by 212 publications

(154 citation statements)

References 124 publications

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“…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]. Because they are relatively safe, display easily tunable physico-chemical properties, can be produced in large quantities with high reproducibility and affordable costs, and show unlimited ferrying capacity [53][54][55], non-viral vectors are nowadays at the forefront of gene delivery [1].…”

Section: Nucleic Acid Description Site Of Action Applications/pathwaymentioning

confidence: 99%

Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!

et al. 2020

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Transfection by means of non-viral gene delivery vectors is the cornerstone of modern gene delivery. Despite the resources poured into the development of ever more effective transfectants, improvement is still slow and limited. Of note, the performance of any gene delivery vector in vitro is strictly dependent on several experimental conditions specific to each laboratory. The lack of standard tests has thus largely contributed to the flood of inconsistent data underpinning the reproducibility crisis. A way researchers seek to address this issue is by gauging the effectiveness of newly synthesized gene delivery vectors with respect to benchmarks of seemingly well-known behavior. However, the performance of such reference molecules is also affected by the testing conditions. This survey points to non-standardized transfection settings and limited information on variables deemed relevant in this context as the major cause of such misalignments. This review provides a catalog of conditions optimized for the gold standard and internal reference, 25 kDa polyethyleneimine, that can be profitably replicated across studies for the sake of comparison. Overall, we wish to pave the way for the implementation of standardized protocols in order to make the evaluation of the effectiveness of transfectants as unbiased as possible.

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Section: Nucleic Acid Description Site Of Action Applications/pathwaymentioning

confidence: 99%

Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!

et al. 2020

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“…In recent years, gene therapy has had considerable progress. However, the big challenge still remains: for gene therapy to be successful, the TNAs must be delivered and expressed within the target cells (75). In order to direct gene expression of TNAs on the desired cells or tissues, a specific carrier of TNAs must be developed, that is, a genetic vehicle must be designed in a fashion that it can target specific cells, such as cancer cells, and deliver TNAs in a localized manner (76)(77)(78)(79).…”

Section: Directing Gene Therapy In Cancermentioning

confidence: 99%

Strategies for Targeting Gene Therapy in Cancer Cells With Tumor-Specific Promoters

et al. 2020

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Cancer is the second cause of death worldwide, surpassed only by cardiovascular diseases, due to the lack of early diagnosis, and high relapse rate after conventional therapies. Chemotherapy inhibits the rapid growth of cancer cells, but it also affects normal cells with fast proliferation rate. Therefore, it is imperative to develop other safe and more effective treatment strategies, such as gene therapy, in order to significantly improve the survival rate and life expectancy of patients with cancer. The aim of gene therapy is to transfect a therapeutic gene into the host cells to express itself and cause a beneficial biological effect. However, the efficacy of the proposed strategies has been insufficient for delivering the full potential of gene therapy in the clinic. The type of delivery vehicle (viral or non viral) chosen depends on the desired specificity of the gene therapy. The first gene therapy trials were performed with therapeutic genes driven by viral promoters such as the CMV promoter, which induces non-specific toxicity in normal cells and tissues, in addition to cancer cells. The use of tumor-specific promoters over-expressed in the tumor, induces specific expression of therapeutic genes in a given tumor, increasing their localized activity. Several cancer- and/or tumor-specific promoters systems have been developed to target cancer cells. This review aims to provide up-to-date information concerning targeting gene therapy with cancer- and/or tumor-specific promoters including cancer suppressor genes, suicide genes, anti-tumor angiogenesis, gene silencing, and gene-editing technology, as well as the type of delivery vehicle employed. Gene therapy can be used to complement traditional therapies to provide more effective treatments.

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“…Some reports suggest the use of ASO or siRNAs, miRNAs, and circRNAs in different diseases, such as cardiovascular or cancers (Holdt et al, 2018;Lucas et al, 2018;Gao et al, 2020b). The major problem that hinders the use of ncRNAs, including lncRNAs and circRNAs, as potential therapeutics is their stability and delivery to the target cells, but advances in chemical modifications and strategies have proved that delivery systems can be improved to reduce unwanted side effects (Patil et al, 2019).…”

Section: Conclusion and Perspectivementioning

confidence: 99%