GTMB 7 - Gene Therapy & Molecular Biology

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Maruyama et al: Kidney-targeted plasmid DNA transferproblems of post-transplant ischemic injury and rejection.Some of the above-described techniques (Tsujie et al,2001b; Maruyama et al, 2002a; Lan et al, 2003; Kita et al,2003) could be useful for ex vivo plasmid DNA deliveryduring kidney transplantation. The kidney to be used forgrafting is readily accessible for gene transfer. In addition,the transgene expression in the graft that is induced by exvivo nonviral techniques is, because of the nature of exvivo transfection, confined to the injected kidney.AcknowledgmentsThis work was supported by a grant from theMinistry of Education, Science, Sports and Culture.ReferencesAkami T, Arakawa K, Okamoto M, Akioka K, Fujiwara I, NakaiI, Mitsuo M, Tomita N, Kaneda Y, Tanaka K, and Oka T(1994) Introduction and expression of human CD59 gene inthe canine kidney. Transplant Proc 26, 1315-1317.Arai M, Wada A, Isaka Y, Akagi Y, Sugiura T, Miyazaki M,Moriyama T, Kaneda Y, Naruse K, Naruse M, OritaY, AndoA, Kamada T, Ueda N, and Imai E (1995) In vivotransfection of genes for renin and angiotensinogen into theglomerular cells induced phenotypic change of the mesangialcells and glomerular sclerosis. Biochem Biophys ResCommun 206, 525-532.Boletta A, Benigni A, Lutz J, Remuzzi G, Soria MR, andMonaco L (1997) Nonviral gene delivery to the rat kidneywith polyethylenimine. Hum Gene Ther 8, 1243-1251.Dworkin LD, Sun AM, and Brenner BM (2000) The renalcirculations. In Brenner & Rector’s The kidney.B.M.Brenner, ed. (W.B.Saunders, Philadelphia, USA) pp.277-318.Foglieni C, Bragonzi A, Cortese M, Cantu L, Boletta A,Chiossone I, Soria MR, and Monaco L (2000) Glomerularfiltration is required for transfection of proximal tubular cellsin the rat kidney following injection of DNA complexes intothe renal artery. Gene Ther 7, 279-285.Higuchi N, Maruyama H, Kuroda T, Kameda S, Iino N, KawachiH, Nishikawa Y, Hanawa H, Tahara H, Miyazaki J, andGejyo F (2003) Hydrodynamics-based delivery of the viralinterleukin-10 gene suppresses experimental crescenticglomerulonephritis in Wistar-Kyoto rats. Gene Ther 10,1297-1310.Isaka Y, Fujiwara Y, Ueda N, Kaneda Y, Kamada T, and Imai E(1993) Glomerulosclerosis induced by in vivo transfection oftransforming growth factor-beta or platelet-derived growthfactor gene into the rat kidney. J Clin Invest 92, 2597-2601.Kita J, Kobayashi E, Hishinuma A, and Kaneda Y (2003)Genetic modification of cold-preserved renal grafts usingHSP70 or bcl-2 HVJ-liposome method. TransplantImmunol 11, 7-14.Lai L-W, Moeckel GW, and Lien Y-HH (1997). Kidney-targetedliposome-mediated gene transfer in mice. Gene Ther 4, 426-431.Lai L-W, Chan DM, Erickson RP, HsuSJ, and Lien YH (1998)Correction of renal tubular acidosis in carbonic anhydrase IIdeficientmice with gene therapy. J Clin Invest 101, 1320-1325.Lan HY, Mu W, Tomita N, Huang XR, Li JH, Zhu HJ, MorishitaR, and Johnson RJ. (2003) Inhibition of renal fibrosis bygene transfer of inducible Smad7 using ultrasoundmicrobubblesystem in rat UUO model. J Am Soc Nephrol14, 1535-1548.Lemley, KV, and Kriz, W (1994) Structure and function of therenal vasculature with clinical and functional correlations. InRenal pathology. C.C. Tisher, and B.M. Brenner, eds.(J.B.Lippincott, Philadelphia, USA) pp. 981-1026.Li S, and Huang L (1997) In vivo gene transfer via intravenousadministration of cationic lipid-protamine-DNA (LPD)complexes. Gene Ther 4, 891-900.Liu F, Song Y, and Liu D (1999) Hydrodynamics-basedtransfection in animals by systemic administration of plasmidDNA. Gene Ther 6, 1258-1266.Madry H, Reszka R, Bohlender J, and Wagner J (2001) Efficacyof cationic liposome-mediated gene transfer to mesangialcells in vitro and in vivo. J Mol Med 79, 184-189.Maruyama H, Higuchi N, Nishikawa Y, Hirahara H, Iino N,Kameda S, Kawachi H, Yaoita E, Gejyo F, and Miyazaki J(2002a) Kidney-targeted naked DNA transfer by retrograderenal vein injection in rats. Hum Gene Ther 13, 455-468.Maruyama H, Higuchi N, Nishikawa Y, Kameda S, Iino N,Kazama JJ, Takahashi N, Sugawa M, Hanawa H, Tada N,Miyazaki J, and Gejyo F (2002b) High-level expression ofnaked DNA delivered to rat liver via tail vein injection. JGene Med 4, 333-341.Niwa H, Yamamura K, and Miyazaki J (1991) Efficient selectionfor high-expression transfectants with a novel eukaryoticvector. Gene 108, 193-199.Price J, Turner D, Cepko C (1987) Lineage analysis in thevertebrate nervous system by retrovirus-mediated genetransfer. Proc Natl Acad Sci USA 84, 156-160.Shimizu H, Maruyama S, Yuzawa Y, Kato T, Miki Y, Morita Y,Maruyama H, Egashira K, and Matsuo S (2003) Antimonocytechemoattractant protein-1 gene therapy attenuatesrenal injury induced by protein-overload proteinuria. J AmSoc Nephrol 14, 1496-1505.Takebe Y, Seiki M, Fujisawa J, Hoy P, Yokota K, Arai K,Yoshida M, and Arai N (1988) SRα promoter: an efficientand versatile mammalian cDNA expression systemcomposed of the simian virus 40 early promoter and the R-U5 segment of human T-cell leukemia virus type 1 longterminal repeat. Mol Cell Biol 8, 466-472.Tomita N, Higaki J, Morishita R, Kato K, Mikami H, Kaneda Y,and Ogihara T (1992) Direct in vivo gene introduction intorat kidney. Biochem Biophys Res Commun 186, 129-134.Tsujie M, Isaka Y, Ando Y, Akagi Y, Kaneda Y, Ueda N, ImaiE, and Hori M (2000) Gene transfer targeting interstitialfibroblasts by the artificial viral envelope-typehemagglutinating virus of Japan liposome method. KidneyInt 57, 1973-1980.Tsujie M., Isaka Y., Nakamura H., Kaneda Y., Imai E., and HoriM. (2001a) Prolonged transgene expression in glomeruliusing an EBV replicon vector system combined with HVJliposomes. Kidney Int 59,1390-1396.Tsujie M., Isaka Y., Nakamura H., Imai E., and Hori M (2001b)Electroporation-mediated gene transfer that targetsglomeruli. J Am Soc Nephrol 12, 949-954.Yamada T, Horiuchi M, Morishita R, Zhang L, Pratt RE, andDzau VJ (1995) In vivo identification of a negativeregulatory element in the mouse renin gene using direct genetransfer. J Clin Invest 96, 1230-1237.Zhang G, Budker V, and Wolff JA (1999) High levels of foreigngene expression in hepatocytes after tail vein injections ofnaked plasmid DNA. Hum Gene Ther 10, 1735-1737.Zhu N, Liggitt D, Liu Y, and Debs R (1993) Systemic geneexpression after intravenous DNA delivery into adult mice.Science 261, 209-211.228
Gene Therapy and Molecular Biology Vol 7, page 229Gene Ther Mol Biol Vol 7, 229-238, 2003Hepatocyte-targeted delivery of Sleeping Beautymediates efficient gene transfer in vivoResearch ArticleBetsy T. Kren, 1 Siddhartha S. Ghosh, 2,3 Cheryle L. Linehan, 1,4 Namita Roy-Chowdhury, 2,3 Perry B. Hackett, 4 Jayanta Roy-Chowdhury, 2,3 and Clifford J.Steer 1,4Departments of 1 Medicine and 4 Genetics, Cell Biology and Development, University of Minnesota Medical School,Minneapolis, MN 554552 Departments of Medicine and Molecular Genetics, and 3 Marion Bessin Liver Research Center, Albert Einstein College ofMedicine, Bronx, NY 10461.__________________________________________________________________________________*Correspondence: Clifford J. Steer, M.D., Department of Medicine, Mayo Mail Code 36, Mayo Building, Room A536, University ofMinnesota Medical School, 420 Delaware Street S.E., Minneapolis, MN 55455. Telephone (612) 624-6648; fax: (612) 625-5620, e-mail:steer001@tc.umn.eduKey words: asialoglycoprotein receptor, gene therapy, genomic integration, polyethyleneimine, Sleeping Beauty transposonAbbreviations: Sleeping Beauty (SB); green fluorescent protein (GFP); partial hepatectomy (PH); asialoglycoprotein receptor (ASGR);inverted repeats/direct repeats (IR/DRs); chicken β-actin/rabbit globin intron (CAGGS); elongation factor (EF)-1α; human embryonickidney (HEK293)Received: 22 September 2003; Revised: 19 November 2003;Accepted: 21 November 2003; electronically published: November 2003SummaryCurrently, most gene therapy studies utilize viral vectors that can potentially produce immunological and toxic sideeffects. To circumvent these limitations, we evaluated the efficiency of nonviral hepatocyte-targeted in vivo deliveryof plasmids that mediate stable genomic integration of transgenes via the Sleeping Beauty (SB) transposon system.We constructed plasmids that express a reporter green fluorescent protein (GFP) transposon and the SBtransposase, required for transgene insertion into genomic DNA, from either a single plasmid (cis) or two differentplasmids (trans). The constructs were compacted to an average diameter of < 50 nm with lactosylatedpolyethyleneimine, a polycation, for targeting to the hepatocyte asialoglycoprotein receptor. Intravenousadministration of the cis plasmid resulted in greater efficiency of transgene integration in mouse liver compared totransposase expression from a separate plasmid. Furthermore, by western blot analysis and fluorescencemicroscopy, delivery of the cis plasmid to rat livers resulted in transgene expression that persisted for months evenafter regeneration from partial hepatectomy. Southern blot analysis of the regenerated livers indicated that SBmediated genomic integration of the GFP transgene at random sites, and this correlated with disappearance of SBtransposase. In conclusion, receptor-mediated targeted delivery of a transposon system capable of transgeneintegration and stable expression provides an attractive alternative to viral vectors for gene therapy to the liver.I. IntroductionRecombinant viral vectors are the current mainstay ofgene therapy for inherited metabolic disorders (Kay et al,2001). However, clinical trials have achieved only modestsuccess, in part, because of the limitations set by viralvectors. For example, adenovirus-based vectors do notintegrate into host chromosomes (Harui et al, 1999) andtheir immunogenicity precludes repeated gene transfer.Furthermore, in contrast to the highly efficient genetransfer to livers of laboratory animals, clinical trials withadenovirus have produced low levels of transgeneexpression in human liver (Raper et al, 2002).Recombinant adeno-associated viral vectors also do notintegrate efficiently in liver (Hillgenberg et al, 2001),resulting in progressive loss of the episomal DNA (Nakaiet al, 2001; Ehrhardt and Kay, 2002). Moreover, the lowlevel integration appears to occur preferentially into activegenes and is associated with chromosomal deletions at thesite (Nakai et al, 2003). Although oncoretroviral vectorsintegrate into the host genome, the process is veryinefficient in non-replicating cells such as hepatocytes invivo (Kalpana, 1999). Lentiviruses, which appear topartially overcome this (Pfeifer et al, 2001; Follenzi et al,2002), are difficult to generate in quantities adequate for229
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