GTMB 7 - Gene Therapy & Molecular Biology

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Maruyama et al: Kidney-targeted plasmid DNA transfervia the pelvic route (Lai et al, 1997, 1998) or renal artery(Lai et al, 1997), anionic artificial viral envelope (AVE)-type hemagglutinating virus of Japan (HVJ)-liposomecomplex via the renal artery (Kita et al, 2003), or cationicpolymer/DNA complex via the renal artery (Boletta et al,1997; Foglieni et al, 2000), or by ultrasound-Optisonmediatednaked plasmid DNA transfer via the renal artery(Lan et al, 2003).The glomerulus has been targeted by delivering anHVJ-liposome complex via the renal artery (Tomita et al,1992; Isaka et al, 1993; Akami et al, 1994; Arai et al,1995), an AVE-type HVJ-liposome complex via the renalartery (Tsujie et al, 2001a), by electroporation-mediatednaked plasmid DNA transfer via the renal artery (Tsujie etal, 2001b), and by ultrasound-Optison-mediated nakedplasmid DNA transfer via the renal artery (Lan et al,2003).Interstitial fibroblasts have been targeted by injectingan AVE-type HVJ-liposome complex retrogradely via theureter (Tsujie et al, 2000), by the retrograde injection ofnaked plasmid DNA via the renal vein usinghydrodynamics-based transfection (Maruyama et al,2002a), or by ultrasound-Optison-mediated naked plasmidDNA transfer via the renal artery (Lan et al, 2003).Most nonviral techniques of kidney-targeted genetransfer have resulted in transient gene expression forrelatively short periods of time: the HVJ-liposome (Tomitaet al, 1992; Isaka et al, 1993; Arai et al, 1995),electroporation-mediated naked plasmid DNA transfer(Tsujie et al, 2001b), and ultrasound-Optison-mediatednaked plasmid DNA transfer (Lan et al, 2003) methodshave resulted in expression for 1 week or less. Thecationic polymer/DNA complex (Boletta et al, 1997) andAVE-type HVJ-liposome delivered retrogradely via theureter (Tsujie et al, 2000) have resulted in 2 weeks ofexpression. Genes delivered by cationic lipid/DNAcomplex were expressed for 5 weeks (Lai et al, 1997,1998), and AVE-type HVJ-liposome complex deliveredvia the renal artery for 8 weeks (Tsujie et al, 2001a).Recently, the technique of retrograde injection via therenal vein has achieved long-term gene expression, lastingmore than 24 weeks (Maruyama et al, 2002a).Thus, the recent history of kidney-targeted plasmidDNA transfer shows the rapid development of nonviraltechniques for use in gene therapy. In this article, wereview kidney-targeted plasmid DNA transfer usingnonviral techniques via several routes into different sites,their transfection efficiency, preclinical studies using thesemethods, safety concerns, and future clinical applications.II. Kidney-targeted gene transfer viathe renal arteryTable 1 provides an overview of studies that haveused kidney-targeted plasmid DNA transfer via the renalartery.A. Liposomes1 HVJ-liposomeTomita et al, (1992) first reported that the HVJliposomeis efficient for glomerulus-targeted gene transfer.Its expression is transient. Four days after the injection ofHVJ-liposome, SV40 large T antigen is detectedimmunohistochemically in 15% of the glomerular cells inthe kidney. Isaka et al, (1993) demonstrated a 35%transfection efficiency of the glomerulus with plasmidscarrying the gene for chloramphenicol acetyltransferase(CAT), transforming growth factor (TGF)-β, and plateletderivedgrowth factor (PDGF)-B genes, driven by thechicken β actin promoter. The blood urea nitrogen andplasma creatinine concentrations are not changedsignificantly by this procedure (Tomita et al, 1992; Isakaet al, 1993). Proteinuria is not observed in the rats treatedwith the HVJ-liposome method (Tomita et al, 1992; Isakaet al, 1993). Isaka et al (1993) demonstrated that theintroduction of either the TGF-β1 or PDGF-B gene intothe kidney induces glomerulosclerosis. Akami et al, (1994)reported the HVJ-liposome mediated gene transfer of ahuman CD59 expression plasmid, pSRαCD59, into thecanine kidney. Human CD59 was detectedimmunohistochemically in the canine glomerular cells for7 days with a maximum on day 2. Yamada et al (1995)identified a silencer in the mouse renin gene promoterregion by transferring renin gene promoter-CAT fusionconstructs into the kidney using the HVJ-liposometechnique. Arai et al, (1995) demonstrated human reninexpression in approximately 30% of the glomeruli on day3 after the introduction of the human renin andangiotensinogen genes. In addition, on day 7 after thetransfection of the renin and angiotensinogen genes, theextracellular matrix had expanded in the glomeruli and α-smooth muscle was expressed in the mesangial cells.These findings suggest that the locally activated reninangiotensin system induces glomerular sclerosis and aphenotypic change in mesangial cells.The mechanism of the HVJ-liposome technique thatresults in glomerulus-specific gene expression is unknown.A possible explanation is that the intrarenal hydrodynamicstate facilitates the contact of HVJ-liposomes withglomerular cell membranes, and another is that thephagocytotic activity of glomerular mesangial cellsfacilitates the uptake of HVJ-liposomes (Tomita et al,1992).3. Cationic lipid/DNA complexesCationic lipid/DNA complexes have been used forkidney-targeted gene transfer via the renal artery.3.1 LipofectinLipofectin (Invitrogen, San Diego, CA) contains twolipid species, a cationic lipid, N[1-(2,3-dioleyloxy)propyl]-N,N,N-trimethylammonium chloride (DOTMA), and aneutral phospholipid, dioleoylphosphatidyl ethanolamine(DOPE). After intrarenal-arterial injection of pCMV-βgal-Lipofectin,lacZ was expressed mainly in thecytoplasm of renal tubular cells in the outer medulla withsome staining in the cortex (Lai et al, 1997). It is not clearwhy there was no transgene expression in the innermedulla. The authors speculate that the high salt and ureacontent in the inner medulla might have affected the entryof the plasmid DNA-Lipofectin complex.222
Gene Therapy and Molecular Biology Vol 7, page 223Table 1: Gene transfer via renal arteryHVJ, HVJ lipospme; pAct, gene expression vector driven by the chicken β_actin promoter (Fregien and Davidson, 1986); CAT,chloramphenicol acetyltransferase; TGF-β1, transforming growth factor-β1; PDGF-B, platelet-derived growth factor-B; SD rat, Sprague-Dawley rat; pSRα, cDNA expression vector composed of the simian virus 40 early promoter and the R-U5’ segment of human T-cellleukemia virus type 1 long terminal repeat (Tatebe et al, 1988); pUtkCAT, mammalian expression vector encoding CAT under thecontrol of a truncated herpes simplex virus thymidine kinase (tk) promoter; pCAGGS, gene expression vector driven by the CAG(cytomegalovirus(CMV) immediate-early enhancer/chicken β-actin hybrid) promoter (Niwa et al, 1991); Lipofectin (Invitrogen),contains two lipid species, a cationic lipid, N[1-(2,3-dioleyloxy)propyl]-N,N,N-trimethylammonium chloride (DOTMA), and a neutralphospholipid, dioleoylphosphatidyl ethanolamine (DOPE); pCMV, gene expression vector driven by CMV promoter; β-gal, Escherichiacoli β-galactosidase; DOGS, dioctadecylamidoglycyl spermine; P. pyralis Luc, firefly Photinus pyralis Luc; AVE-type HVJ, artificialviral envelope-type HVJ liposome; pEBAct, the EBV replicon vector, which was constructed by cloning the Epstein-Barr virus nuclearantigen-1 (EBNA-1) and oriP sequences in the same plasmid; Luc, luciferase; Lipofectamine (Invitrogen), contains two lipid species, acationic lipid, 2,3-dioleyloxy-N-[2(sperminecarboxamido)ethyl]-N,N-dimethyl-1-propanaminium trifluoroacetate (DOSPA), and aneutral phospholipid DOTMA; DAC-Chol, conposed of 3β-[N-(N,N-dimethylaminoethrane)carbamoyl] cholesterol and DOPE; DMRIE,3-dimethyl-hydroxy-ethylammonium bromide; pBAG, β-gal-transducing vector driven by Moloney murine leukemia virus promoter(Price et al, 1987); Renin FURIN , the human renin cDNA fragment is modofied by insertion of a furin cleavage site at the end of the reninprosegment, redering renin active following furin cleavage; 25-kD PEI, branched 25-kD cationic polymer polyethylenimine; β-galNuc,lacZ gene equipped with a nuclear localization signal; pcDNA3 (Invitrogen), expression vector driven by thecytomegalovirusimmediate-early enhancer/promoter; pTRE (Clontech), tetracycline-inducible vector driven by tetracyclin responseelement/minCMV promoter (CMV promoter deleted enhancer; pEFpurop-Tet-on (Clontech), improved pTet-on vector driven by theCMV promoter; m2Smad7, Smad7 cDNA with a flag tag (m2) at its NH 2 terminus; UUO model, unilateral ureteral obstruction model;pRL-CMV (Promega), Renilla luciferase expression vector driven by the cytomegalovirusimmediate-early enhancer/promoter; HSP70,heat shock protein 70. Note that in cases where no incubation time is given, this information was not reported in the cited study.223
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