Physical Methods for Gene Transfer

Alsaggar, Mohammad; Liu, Dexi

doi:10.1016/bs.adgen.2014.10.001

Cited by 28 publications

(19 citation statements)

References 146 publications

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“…Since the first attempts to treat genetically based diseases using retroviral vectors to deliver transgenes to host cells, 1 , 2 various non-viral vectors and transgene delivery methods have been developed, including nanoparticle-mediated gene delivery, 3 physical methods, 4 , 5 , 6 plasmids, 7 , 8 , 9 and DNA transposons, which are DNA sequences that can move from one location and become integrated into another locus of the genome. 10 Even though transposons were discovered in the 1950s, it has been shown only recently that the transposon named Sleeping Beauty ( SB ), and especially its hyperactive form ( SB100X ), is able to efficiently and safely integrate transgenes into the host genome, providing sustained transgene expression in quiescent and dividing cells.…”

Section: Introductionmentioning

confidence: 99%

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

Pastor

Johnen

Harmening

et al. 2018

Molecular Therapy - Nucleic Acids

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The anti-angiogenic and neurogenic pigment epithelium-derived factor (PEDF) demonstrated a potency to control choroidal neovascularization in age-related macular degeneration (AMD) patients. The goal of the present study was the development of an efficient and safe technique to integrate, ex vivo, the PEDF gene into retinal pigment epithelial (RPE) cells for later transplantation to the subretinal space of AMD patients to allow continuous PEDF secretion in the vicinity of the affected macula. Because successful gene therapy approaches require efficient gene delivery and stable gene expression, we used the antibiotic-free pFAR4 mini-plasmid vector to deliver the hyperactive Sleeping Beauty transposon system, which mediates transgene integration into the genome of host cells. In an initial study, lipofection-mediated co-transfection of HeLa cells with the SB100X transposase gene and a reporter marker delivered by pFAR4 showed a 2-fold higher level of genetically modified cells than when using the pT2 vectors. Similarly, with the pFAR4 constructs, electroporation-mediated transfection of primary human RPE cells led to 2.4-fold higher secretion of recombinant PEDF protein, which was still maintained 8 months after transfection. Thus, our results show that the pFAR4 plasmid is a superior vector for the delivery and integration of transgenes into eukaryotic cells.

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Section: Introductionmentioning

confidence: 99%

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

Pastor

Johnen

Harmening

et al. 2018

Molecular Therapy - Nucleic Acids

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“…To increase the efficiency of gene transfer in non-viral vectors, physical stimuli such as electroporation can be applied. [85, 86] Although genes can be delivered in vivo to the target tissue, the lack of spatiotemporal control of the gene delivery process is a major limitation of this approach. Another approach is the ex vivo genetic modification of cells that are subsequently implanted.…”

Section: Biomechanical and Biochemical Factors In Skeletal Muscle Tismentioning

confidence: 99%

Engineering Biomimetic Materials for Skeletal Muscle Repair and Regeneration

Nakayama

Shayan

Huang

2019

Adv Healthcare Materials

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Although skeletal muscle is highly regenerative following injury or disease, endogenous self-regeneration is severely impaired in conditions of volume traumatic muscle loss. Consequently, tissue engineering approach is a promising approach to regenerate skeletal muscle. Biological scaffolds serve as not only structural support for the promotion of cellular ingrowth, but they also impart potent modulatory signaling cues that may be beneficial for tissue regeneration. In this work, the progress of tissue engineering approaches for skeletal muscle engineering and regeneration is overviewed, with a focus on the techniques to create biomimetic engineered tissue using extracellular cues. These factors include mechanical and electrical stimulation, geometric patterning, and delivery of growth factors or other bioactive molecules. We further describe the progress of evaluating the therapeutic efficacy of these approaches in preclinical models of muscle injury.

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“…However, two major barriers must be overcome for this technology to be efficient: both DNA and RNA must first be protected from the action of hydrolyzing enzymes present in the extracellular matrix and, second, must be helped to cross the cell membrane since they are not able to enter the cell spontaneously mainly due to electrostatic repulsion with glycoproteins and phospholipids. In order to surmount such barriers, many physical methods have been developed, encompassing, among others, mechanical, electrical, hydrodynamic, and ultrasonic [20]. Another way to achieve these goals is to develop a biological or chemical helper, referred to as a vector, that should be able to pack and protect the gene outside the cell, cross the cell membrane, and finally deliver the gene into the target organelle.…”

Section: Gene Deliverymentioning

confidence: 99%

Aminoglycosides: From Antibiotics to Building Blocks for the Synthesis and Development of Gene Delivery Vehicles

Bellucci

Volonterio

2020

Antibiotics

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Aminoglycosides are a class of naturally occurring and semi synthetic antibiotics that have been used for a long time in fighting bacterial infections. Due to acquired antibiotic resistance and inherent toxicity, aminoglycosides have experienced a decrease in interest over time. However, in the last decade, we are seeing a renaissance of aminoglycosides thanks to a better understanding of their chemistry and mode of action, which had led to new trends of application. The purpose of this comprehensive review is to highlight one of these new fields of application: the use of aminoglycosides as building blocks for the development of liposomal and polymeric vectors for gene delivery. The design, synthetic strategies, ability to condensate the genetic material, the efficiency in transfection, and cytotoxicity as well as when available, the antibacterial activity of aminoglycoside-based cationic lipids and polymers are covered and critically analyzed.

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Physical Methods for Gene Transfer

Cited by 28 publications

References 146 publications

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

Engineering Biomimetic Materials for Skeletal Muscle Repair and Regeneration

Aminoglycosides: From Antibiotics to Building Blocks for the Synthesis and Development of Gene Delivery Vehicles

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