Regulation of Gene Expression Using a Conditional RNA Antisense Strategy

Trollet, Capucine; Ibañez-Ruiz, Magdalena; Bloquel, Carole; Valin, Gabrielle; Scherman, Daniel; Bigey, Pascal

doi:10.1166/gl.2004.036

Cited by 4 publications

(6 citation statements)

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“…Finally, it has been reported that coinjection and electrotransfer of several plasmids into skeletal muscle are feasible for the expressions of various transgenes (28,49). We thus evaluated the possibility of obtaining a multivalent neutralizing antiserum against all three toxin serotypes via the electrotransfer procedure.…”

Section: Discussionmentioning

confidence: 99%

Generation of High-Titer Neutralizing Antibodies against Botulinum Toxins A, B, and E by DNA Electrotransfer

et al. 2009

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Botulinum neurotoxins are known to be among the most toxic known substances. They produce severe paralysis by preventing the release of acetylcholine at the neuromuscular junction. Thus, new strategies for efficient production of safe and effective anti-botulinum neurotoxin antisera have been a high priority. Here we describe the use of DNA electrotransfer into the skeletal muscle to enhance antiserum titers against botulinum toxin serotypes A, B, and E in mice. We treated animals with codon-optimized plasmid DNA encoding the nontoxic but highly immunogenic C-terminal heavy chain fragment of the toxin. By employing both codon optimization and the electrotransfer procedure, the immune response and corresponding neutralizing antiserum titers were markedly increased. The cellular localization of the antigen and the immunization regimens were also shown to increase neutralizing titers to >100 IU/ml. This study demonstrates that DNA electrotransfer is an effective procedure for raising neutralizing antiserum titers to remarkably high levels.

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Section: Discussionmentioning

confidence: 99%

Generation of High-Titer Neutralizing Antibodies against Botulinum Toxins A, B, and E by DNA Electrotransfer

et al. 2009

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“…This constitutes an abundant blood vascular system [Lu et al, 2003] and skeletal muscle is therefore able to produce secreted proteins with functional post-translational modifications which can easily reach the blood circulation (for a review see [Goldspink, 2003]). Finally, cotransfection of multiple unlinked genes can be easily performed by electroporation [Trollet et al, 2004]. For all these reasons, muscle is an excellent target for gene therapy.…”

Section: In Vivo Skeletal Muscle Dna Electrotransfermentioning

confidence: 99%

“…Improvement of the transgenic protein structure has been studied by Dalle et al [Dalle et al, 2001] who designed a dimeric EPO protein, which gave greater hematocrit increases and higher erythrocyte production than the monomeric protein, and no immune response against the transgenic dimeric protein was observed. Tetracyclin regulatory systems have also been used to control EPO expression [Dalle et al, 2001;Rizzuto et al, 1999;Trollet et al, 2004]. A slight increase in plasmatic EPO level was obtained by changing the EPO signal peptide by the hTAP (human tissue plasminogen activator) signal peptide, leading to an increased hematocrit level in cynomolgus monkeys [Fattori et al, 2005].…”

Section: Monogenic Diseasesmentioning

confidence: 99%

“…Dilated cardiomyopathy, one of the major causes of severe heart failure, was studied in a hamster inherited model IL-1Ra Epo [Ataka et al, 2003;Dalle et al, 2001;Fattori et al, 2005;Kreiss et al, 1999;Maruyama et al, 2001;Maruyama et al, 2004;Maruyama et al, 2000;Rizzuto et al, 1999;Rizzuto et al, 2000;Trollet et al, 2004] Factor IX [Bettan et al, 2000;Fewell et al, 2001] IL-4 Horiki et al, 2003] HGF [Komamura et al, 2004;Umeda et al, 2004;Xue et al, 2003;Xue et al, 2002] Factor VIII [Long et al, 2005;Mei et al, 2006] IL-5 [Aihara et al, 1998] FGF-1 [Nishikage et al, 2004] Leptin [Xiang et al, 2005;Xiang et al, 2003] IL-10 [Adachi et al, 2002;Mallat et al, 1999;Nakano et al, 2001;SaidenbergKermanac'h et al, 2003;Silvestre et al, 2000;Tavakoli et al, 2006;Watanabe et al, 2001] FGF-2 binding polypeptide [Bossard et al, 2004] Insulin/Proinsulin [Kon et al, 1999;Martinenghi et al, 2002] IL-12 Lucas et al, 2001] VEGFB [Silvestre et al, 2003] Endostatin …”

Section: Organ/tissue-specific Diseasesmentioning

confidence: 99%

“…As this can be achieved by electroporation, Lamartina et al studied the activity of novel doxycycline transactivators in a gene switch system [Lamartina et al, 2002], and we studied a system based on the hypoxiaresponsive element and tetracycline transactivators . We have also studied a system of regulation of gene expression composed of three plasmids using the combination of an antisense strategy and the tetracycline system [Trollet et al, 2004].…”

Section: Electrotransfer As a Toolmentioning

confidence: 99%

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Electrotransfer into Skeletal Muscle for Protein Expression

Trollet

Bloquel²,

Scherman³

et al. 2006

CGT

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An efficient and safe method to deliver DNA in vivo is a requirement for several purposes, such as study of gene function and gene therapy applications. Among the different non-viral delivery methods currently under investigation, in vivo DNA electrotransfer has proven to be one of the most efficient and simple. This technique is a physical method of gene delivery consisting in local application of electric pulses after DNA injection. Although this technique can be applied to almost any tissue of a living animal, including tumors, skin, liver, kidney, artery, retina, cornea or even brain, this review will focus on electrotransfer of plasmid DNA into skeletal muscle and its possible uses in gene therapy, vaccination, or functional studies. Skeletal muscle is a good target for electrotransfer of DNA as it is: a large volume easily accessible, an endocrine organ capable of expressing several local and systemic factors, and muscle fibres as post-mitotic cells have a long lifespan that allows long-term gene expression. In this review, we describe the mechanism of DNA electrotransfer, we assess toxicity and safety considerations related to this technique, and we focus on important therapeutic applications of electrotransfer demonstrated in animal models in recent years.

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Delivery of DNA into Muscle for Treating Systemic Diseases: Advantages and Challenges

Trollet

Scherman

Bigey

2008

Methods in Molecular Biology

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An efficient and safe method to deliver DNA in vivo is a requirement for several purposes, such as the study of gene function and gene therapy applications. Among the different nonviral delivery methods currently under investigation, in vivo DNA electrotransfer has proven to be one of the most efficient and simple methods. This technique is a physical method of gene delivery consisting of a local application of electric pulses after injection of DNA. This technique can be applied to almost any tissue of a living animal, including tumors, skin, liver, kidney, artery, retina, cornea, or even brain, but the focus of this review will be on electrotransfer of plasmid DNA into skeletal muscle and its possible therapeutic uses for systemic diseases. Skeletal muscle is a good target for electrotransfer of DNA because of the following features: a large volume of easily accessible tissue, an endocrine organ capable of expressing several local and systemic factors, and muscle fibers as postmitotic cells have a long lifespan, which allows long-term gene expression. In this review, we will describe the main characteristics of DNA electrotransfer, including toxicity and safety issues related to this technique. We will focus on the important possible therapeutic applications of electrotransfer for systemic diseases demonstrated in animal models in the recent years, in the fields of monogenic diseases, tissue-specific diseases, metabolic disorders, immune-system-related diseases, and cancer. Finally, we will discuss the advantages and challenges of this technique.

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Regulation of Gene Expression Using a Conditional RNA Antisense Strategy

Cited by 4 publications

References 0 publications

Generation of High-Titer Neutralizing Antibodies against Botulinum Toxins A, B, and E by DNA Electrotransfer

Generation of High-Titer Neutralizing Antibodies against Botulinum Toxins A, B, and E by DNA Electrotransfer

Electrotransfer into Skeletal Muscle for Protein Expression

Delivery of DNA into Muscle for Treating Systemic Diseases: Advantages and Challenges

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