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      New Paradigm of Gene Therapy: Skeletal-Muscle-Targeting Gene Therapy for Kidney Disease

      S. Karger AG

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          Most cited references10

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          Efficient selection for high-expression transfectants with a novel eukaryotic vector

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            Gene transfer into muscle by electroporation in vivo.

            Among the nonviral techniques for gene transfer in vivo, the direct injection of plasmid DNA into muscle is simple, inexpensive, and safe. Applications of this method have been limited by the relatively low expression levels of the transferred gene. We investigated the applicability of in vivo electroporation for gene transfer into muscle, using plasmid DNA expressing interleukin-5 (IL-5) as the vector. The tibialis anterior muscles of mice were injected with the plasmid DNA, and then a pair of electrode needles were inserted into the DNA injection site to deliver electric pulses. Five days later, the serum IL-5 levels were assayed. Mice that did not receive electroporation had serum levels of 0.2 ng/ml. Electroporation enhanced the levels to over 20 ng/ml. Histochemical analysis of muscles injected with a lacZ expression plasmid showed that in vivo electroporation increased both the number of muscle fibers taking up plasmid DNA and the copy number of plasmids introduced into the cells. These results demonstrate that gene transfer into muscle by electroporation in vivo is more efficient than simple intramuscular DNA injection.
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              Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain.

              Adeno-associated viral (AAV) vectors are non-pathogenic, integrating DNA vectors in which all viral genes are removed and helper virus is completely eliminated. To evaluate this system in the post-mitotic cells of the brain, we found that an AAV vector containing the lacZ gene (AAVlac) resulted in expression of beta-galactosidase up to three months post-injection in vivo. A second vector expressing human tyrosine hydroxylase (AAVth) was injected into the denervated striatum of unilateral 6-hydroxydopamine-lesioned rats. Tyrosine hydroxylase (TH) immunoreactivity was detectable in striatal neurons and glia for up to four months and we also found significant behavioural recovery in lesioned rats treated with AAVth versus AAVlac controls. Safe and stable TH gene transfer into the denervated striatum may have potential for the genetic therapy of Parkinson's disease.

                Author and article information

                S. Karger AG
                December 1999
                30 November 1999
                : 83
                : 4
                : 296-300
                Division of Nephrology, Department of Internal Medicine and Therapeutics, Osaka University Graduate School of Medicine, Osaka, Japan
                45420 Nephron 1999;83:296–300
                © 1999 S. Karger AG, Basel

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                Page count
                References: 29, Pages: 5
                Self URI (application/pdf): https://www.karger.com/Article/Pdf/45420
                Self URI (text/html): https://www.karger.com/Article/FullText/45420
                Self URI (journal page): https://www.karger.com/SubjectArea/Nephrology
                Molecular Biology in Renal Diseases<br>Section Editor: F.P. Schena, Bari

                Cardiovascular Medicine,Nephrology
                Cardiovascular Medicine, Nephrology


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