A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle

Lemieux, Pierre; Guérin, Nadia; Paradis, Goulven G.; Proulx, R; Chistyakova, L.G.; Kabanov, Alexander V.; Alakhov, Valery

doi:10.1038/sj.gt.3301189

Cited by 203 publications

(157 citation statements)

References 31 publications

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“…20 More recently, Lemieux et al reported that an amphiphilic carrier, SP1017, composed of a mixture of the block co-polymers (poloxymers), pluronics L61 and F127, significantly augmented intramuscular expression of several reporter genes. 68 SP1017 enhanced peak gene expression by about 10-fold compared with injected naked plasmid alone and also led to sustained higher levels of expression. We too have observed an improvement in transgene expression in muscle with naked plasmid delivered in conjunction with such a polymer formulation.…”

Section: Lipids Liposomes and Polymersmentioning

confidence: 93%

“…63 Cationic polymers have also been disappointing for muscle transfection. 68,69 The commonly used cationic polymers, poly-(L)-ornithine (PLO), polyamidoamine and branched polyethylemine (PEI) and its analogues mediate highly efficient plasmid delivery in vitro into many types of cells, including myoblasts. [70][71][72] In vivo, however, these polymers did not improve transfection efficiency in skeletal muscle.…”

Section: Lipids Liposomes and Polymersmentioning

confidence: 99%

“…However, SP1017 has been shown Non-viral gene delivery in skeletal muscle QL Lu et al to considerably increase plasmid DNA diffusion within muscle tissue. 68 …”

Section: Lipids Liposomes and Polymersmentioning

confidence: 99%

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Non-viral gene delivery in skeletal muscle: a protein factory

Lu¹,

Bou‐Gharios²,

Partridge³

2003

Gene Ther

169

109

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Ever since the publication of the first reports in 1990 using skeletal muscle as a direct target for expressing foreign transgenes, an avalanche of papers has identified a variety of proteins that can be synthesized and correctly processed by skeletal muscle. The impetus to the development of such applications is not only amelioration of muscle diseases, but also a range of therapeutic applications, from immunization to delivery of therapeutic proteins, such as clotting factors and hormones. Although the most efficient way of introducing transgenes into muscle fibres has been by a variety of recombinant viral vectors, there are potential benefits in the use of non-viral vectors. In this review we assess the recent advances in construction and delivery of naked plasmid DNA to skeletal muscle and highlight the options available for further improvements to raise efficiency to therapeutic levels.

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Section: Lipids Liposomes and Polymersmentioning

confidence: 93%

Section: Lipids Liposomes and Polymersmentioning

confidence: 99%

See 1 more Smart Citation

Non-viral gene delivery in skeletal muscle: a protein factory

Lu¹,

Bou‐Gharios²,

Partridge³

2003

Gene Ther

169

109

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“…Its advantages, compared with other nonviral gene delivery systems, are that gene expression is drastically increased (2-to 4-log fold), longlasting (months), and very specific and localized . This technique has also been successful in various tissues such as skeletal muscle (Muramatsu et al, 1998;Aihara and Miyazaki, 1999;Gehl and Mir, 1999;Imai and Isaka, 1999;Mathiesen, 1999;Rizzuto et al, 1999;Bettan et al, 2000;Lemieux et al, 2000;Maruyama et al, 2000;Vicat et al, 2000;Lucas and Heller, 2001;Muramatsu et al, 2001), liver (Suzuki et al, 1998;Heller et al, 2000), testis (Muramatsu et al, 1997;Yamazaki et al, 1998;Yamazaki et al, 2000), skin (Johnson et al, 1998;Vanbever and Preat, 1999;Glasspool-Malone et al, 2000), cornea (Oshima et al, 1998;Sakamoto et al, 1999), cardiaovascular (Harrison et al, 1998;Martin et al, 2000) and mammary tumor tissue Wells et al, 2000;Lohr et al, 2001). In particular, gene delivery to skeletal muscle is a promising strategy for the systemic secretion of therapeutic proteins.…”

Section: Introductionmentioning

confidence: 99%

Optimal salt concentration of vehicle for plasmid DNA enhances gene transfer mediated by electroporation

Lee

Cho²,

Jang³

et al. 2002

Exp Mol Med

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In vivo electroporation has emerged as a leading technology for developing nonviral gene therapies, and the various technical parameters governing electroporation efficiency have been optimized by both theoretical and experimental analysis. However, most electroporation parameters focused on the electric conditions and the preferred vehicle for plasmid DNA injections has been normal saline. We hypothesized that salts in vehicle for plasmid DNA must affect the efficiency of DNA transfer because cations would alter ionic atmosphere, ionic strength, and conductivity of their medium. Here, we show that half saline (71 mM) is an optimal vehicle for in vivo electroporation of naked DNA in skeletal muscle. With various salt concentrations, two reporter genes, luciferase and β β β β-galactosidase were injected intramuscularly under our optimal electric condition (125 V/cm, 4 pulses x 2 times, 50 ms, 1 Hz). Exact salt concentrations of DNA vehicle were measured by the inductively coupled plasma-atomic emission spectrometer (ICP-AES) and the conductivity change in the tissue induced by the salt in the medium was measured by Low-Frequency (LF) Impedance Analyzer. Luciferase expression increased as cation concentration of vehicle decreased and this result can be visualized by X-Gal staining. However, at lower salt concentration, transfection efficiency was diminished because the hypoosmotic stress and electrical injury by low conductivity induced myofiber damage. At optimal salt concentration (71 mM), we observed a 3-fold average increase in luciferase expression in comparison with the normal saline condition (p < 0.01). These results provide a valuable experimental parameter for in vivo gene therapy mediated by electroporation.

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“…The coinjection of 'naked' pDNA and some Pluronics was shown to allow for an enhancement of intramuscular vector expression [11][12][13]. Similar to Pluronics, another non-ionogenic block copolymer of poly(lactic-co-glycolic acid), PLGA, and polyethylene glycol, PEG (PLGA-PEG-PLGA) improved the transfection process in vivo [14].…”

Section: Introductionmentioning

confidence: 99%

Intracellular delivery of VEGF165 encoding gene therapeutic using trifunctional copolymers of ethylene oxide and propylene oxide

Bondar

Shevchenko

Мартынова

et al. 2015

European Polymer Journal

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New type of copolymers of propylene oxide and ethylene oxide was assessed for promoting delivery of plasmid DNA based gene therapeutics. Lipid-like trifunctional copolymers (TFCs), with both random or diblock structures and relatively low hydrophilic-lipophilic balance, were studied and compared with linear Pluronic™ L61. Structure-dependent relationships for micelleforming, cytotoxic and hemolytic properties of these copolymers were revealed. The TFC with the mean number of propylene oxide and ethylene oxide units of 83.5 and 24.2, respectively, exhibited relatively low adverse effects in vitro. The latter TFC interacted with plasmid DNApolyethyleneimine complexes and improved their intracellular delivery. Furthermore, this TFC efficiently promoted the transfection of dermal fibroblasts with VEGF165-encoding Neovasculgen® plasmid DNA, which has been clinically used for the therapeutic angiogenesis.Our findings demonstrated for the first time that TFCs are promising for the polymer-mediated delivery of gene therapeutics.

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A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle

Cited by 203 publications

References 31 publications

Non-viral gene delivery in skeletal muscle: a protein factory

Non-viral gene delivery in skeletal muscle: a protein factory

Optimal salt concentration of vehicle for plasmid DNA enhances gene transfer mediated by electroporation

Intracellular delivery of VEGF165 encoding gene therapeutic using trifunctional copolymers of ethylene oxide and propylene oxide

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