Gene therapy and DNA delivery systems

Ibraheem, D.; Elaı̈ssari, Abdelhamid; Fessi, Hatem

doi:10.1016/j.ijpharm.2013.11.041

Cited by 407 publications

(262 citation statements)

References 178 publications

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“…Although a few studies have reported that naked DNA directly injected into target regions can also express, most results have indicated that it is more effective to administer DNA using a well-designed drug delivery system. 17,18 SWNT has been widely studied in biomedical research and plays a significant role in drug delivery systems. 19,20 …”

Section: Construction and Confirmation Of The Pei-swntmentioning

confidence: 99%

Photoactivatable RNAi for cancer gene therapy triggered by near-infrared-irradiated single-walled carbon nanotubes

Ren¹,

Lin²,

Wang³

et al. 2017

IJN

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The efficacy of RNA interference (RNAi)-based cancer gene therapy is limited by its unexpected side effects, thus necessitating a strategy to precisely trigger conditional gene knockdown. In this study, we engineered a novel photoactivatable RNAi system, named as polyetherimide-modified single-wall carbon nanotube (PEI-SWNT)/pHSP-shT, that enables optogenetic control of targeted gene suppression in tumor cells. PEI-SWNT/pHSP-shT comprises a stimulus-responsive nanocarrier (PEI-SWNT), and an Hsp70B′-promoter-driven RNAi vector (pHSP-shT). In response to near-infrared (NIR) light irradiation, heating of PEI-SWNT in breast MCF-7 cells triggered gene knockdown targeting human telomerase reverse transcriptase through RNAi, with the gene-knockdown activity capable of being switched off by extinguishing the NIR. Furthermore, we demonstrated that the photoactivatable RNAi system exhibited higher antitumor activity by combining gene therapy and photothermal therapy, both in vitro and in vivo. Optogenetic control of RNAi based on an NIR-activated nanocarrier will potentially facilitate improved understanding of molecular-targeted gene therapy in human malignant tumors.

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Section: Construction and Confirmation Of The Pei-swntmentioning

confidence: 99%

Photoactivatable RNAi for cancer gene therapy triggered by near-infrared-irradiated single-walled carbon nanotubes

Ren¹,

Lin²,

Wang³

et al. 2017

IJN

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“…[36], siRNA sequences directed against E2F1 transcription factor (siE2F1-1324) were encapsulated inside positively charged vesicles purposely designed and produced to enhance the interaction with both the negatively charged siRNA, improving its encapsulation efficiency, and the cell-plasmatic membrane, also negatively charged, improving siRNA incorporation in the target cells [37,38]. It was demonstrated that E2F1 promotes the aggressiveness of human colorectal cancer by activating the ribonucleotide reductase small subunit M2 whose high expression induces cancer and contributes to tumour growth and invasion [39].…”

Section: Pharmaceutical Application: Nanoliposome Vectors For Sirnas mentioning

confidence: 99%

Phenomenological and Formulation Aspects in Tailored Nanoliposome Production

Bochicchio¹,

Lamberti²,

Barba³

2017

Liposomes

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Liposomes as cell-mimetic system have attracted wide attention of researchers in various branches of the drug delivery topic as they can be highly functionalized and personalized, thus solving the major drawbacks of bioactive molecules linked to their low stability, limited membrane permeability, short half-life and low bioavailability. The development of sustainable processes able to produce ad hoc liposomes in a rapid manner through the use of not-laboured techniques, avoiding drastic conditions, is of great relevance for the industrial sector. In this chapter, two novel liposome production processes, the ultrasound-assisted thin-film hydration and the simil-microfluidic techniques sharing the same size reduction/homogenization preparative step, are presented. The phenomenological aspects involved in vectors constitution through the duty cycle sonication process (bilayer rupture/vesicles formation mechanisms) and through the simil-microfluidic approach (intubated flows interdiffusion mechanisms) are described. Finally, two applications as case histories involving the use of the developed techniques for relevant classes of active molecule delivery are described. In particular, a pharmaceutical application concerns the encapsulation of short-interfering RNA (siRNA) molecule, used for gene therapy, inside cationic nanoliposomes, and a nutraceutical application consists in the production of ferrous sulphate anionic liposomal formulations with improved features compared to those already present on the market.

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“…Gene therapy is one of the most promising strategies for treatment of various diseases such as cancer and inflammatory, neurological, cardiovascular or metabolic disorders (Ibraheem et al, 2014;Ragelle et al, 2014). Simultaneously, the choice of a good transfection agent for delivery of nucleic acids is critical for each gene transfer procedure, however most commercially available transfection agents have still some limitations (PerezMartinez et al, 2011).…”

Section: Introductionmentioning

confidence: 99%