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Review

The prospects of hepatic drug delivery and gene therapy

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Pages 1795-1817 | Published online: 23 Feb 2005

Bibliography

  • MEIJER DKF, MOLEMA GJE: Targeting of drugs to the liver. Sem. Liver Dis. (1995) 15:202–256.
  • ••A comprehensive review of possible liver targeting ap-proaches, which focuses on various vehicles used for deliv-ery of drugs to the liver.
  • HOOFNAGLE JH, LAU D: New therapies for chronic hepatitis B. J. Viral Hepatitis (1997) 4:S41–S50.
  • •Three promising therapies for hepatitis B infection, antiviral chemotherapy, immunomodulation with cell-based thera-pies, vaccines or cytokines, and gene therapy such as with antisense oligonucleotides, ribozymes or viral mutants, are discussed. The most promising immediate approach to ther-apy is with the new nucleoside antivirals- lamivudine and famciclovir.
  • RENSEN PCN, SCHIFFELERS RM, VERSLUIS AJ et al.: Hu-man recombinant apolipoprotein E-enriched lipo-somes can mimic low-density lipoproteins as carriers for the site-specific delivery of antitumor agents. Mol Pharmacol. (1997) 52:445–455.
  • ALBRECHT C, MEIJER DF, LEBBE C et al.: Targeting ne-prozen coupled serum albumin to non-parenchymal cells reduces endotoxin-induced mortality in rats with biliary cirrhosis. Hepatology (1997) 26:1553–1559.
  • VANBERKEL TJC: Selective hepatic drug delivery - frombasic concept to clinical practice. Hepatology (1997) 26:1680–1681.
  • •A brief highlight of receptor-mediated targeting to the liver, especially, sinusoidal endothelial cells and Kupffer cells.
  • HIRABAYASHI H, NISHIKAWA M, TAKAKURA Y,HASHIDA M: Development and pharmacokinetics of galactosylated poly-t-glutamic acid as a biodegradable carrier for liver-specific drug delivery. Pharmaceut. Res. (1996) 13:880–884.
  • WU J, LIU P, ZHU JL, MADDUKURI S, ZERN MA: Increased liver up-take of liposomes and improved targeting effi-cacy by labeling with asialofetuin in rodents. Hepatol-ogy (1998) 27:772–778.
  • ••A report on efficiency of asialofetuin-labelled, vitamin E-containing liposomes in selectively targeting to hepatocyes.
  • YOSHIOKA S, IMAEDA N, OKANO Y, MIZUKAMI Y, KATA- GIRT Y: Preferential uptake of lactosylceramide-bearing dipalmitoylphosphatidylcholine-liposomes into liver: role of membrane fluidity. Biol. Pharm. (1994) 17:640–644.
  • ZERN MA, KRESINA TF: Hepatic drug delivery and gene therapy. Hepatology (1997) 25:484–491.
  • ••A summary of an NIH-sponsored conference: Targeting ofNovel Therapeutics, to the Liver and GI Tract, (Sept. 21–22, 1995).
  • WU J, ZERN MA: Modification of liposomes for liver tar- geting. J. Hepatol. (1996) 24:757–763.
  • ••A review of approaches to increase liver accumulation ofliposomes, and strategies to selectively target hepatocytes.
  • TORCHILIN VP, TRUBETSKOY VS, WHITEMAN KR et al.:New synthetic amphophilic polymers for steric pro-tection of liposomes in vivo. J. Pharmaceut. ScL (1995) 84:1049–1053.
  • LITZINGER DC, BUITING AMJ, ROOIJEN AV, HUANG L: Ef-fect of liposome size on the circulation time and in-traorgan distribution of amphipathic poly(ethylene glycol)-containing liposomes. Biochim. Biophys. Acta. (1994) 1190:99–107.
  • YU HY, UN CY: Uptake of charged liposomes by the ratliver. J. Formosan Med. Assoc. (1997) 96:409–413.
  • LIM HJ, MASIN D, MADDEN TD, BALLY MB: Influence ofdrug release characteristics on the therapeutic activity of liposomal mitoxantrone. J. Pharmacol. Exp. Ther. (1996) 281:566–573.
  • HUWYLER J, YAN J, PARDRIDGE WM: Receptor mediated delivery of daunomycin using immunoliposomes: pharmacokinetics and tissue distribution in the liver. J. Pharmacol Exp. Ther. (1997) 282:1541–1546.
  • WU J, KUNCIO GS, ZERN MA: Human liver growth in fi-brosis and cirrhosis. In: Liver Growth and Repair: From Basic Science to Clinical Practice. Strain AJ, Diehl AM (Eds.), Chapman & Hall, London (1998):558–576.
  • KRIEGER M, HERZ J: Structures and functions of mul-tiligand lipoprotein receptors: macrophage scavenger receptors and LDL receptor-related protein (LRP). Ann. Rev. Biochem. (1994) 63:601–637.
  • SAMBRANO GR, STEINBER D: Recognition of oxida-tively damaged and apoptotic cells by an oxidized low density lipoprotein receptor on mouse peritoneal macrophages: role of membrane phosphatidyserine. Proc. Natl. Acad. ScL USA (1995) 92:1396–1400.
  • MAENO Y, FUJIOKA H, HOLLINGDALE MR et al.: Ultra-structural localization of CD36 in human hepatic sinu-soidal lining cells, hepatocytes, human hepatoma (HepG2-A16) cells, and C32 amelanotic melanoma cells. Exp. Parasitol. (1994) 79:383–390.
  • DAEMEN T, VELINOVA M, REGTS J et al: Different intra-hepatic distribution of phosphatidylglycerol and phosphatidylserine liposomes in the rat. Hepatology (1997) 26:416–423.
  • TSCHAIKOWSKY K, BRAIN JD: Effects of liposome-encapsulated dichloromethylene diphosphonate on macrophage function and endotoxin-induced mortal-ity. Biochim. Biophys. Acta. (1994) 1222:323–330.
  • FREANSSEN EJF, JANSEN RW, VAALBURG M, MEIJER DKF:Hepatic and intrahepatic targeting of an anti-inflammatory agent with human serum albumin and neoglycoproteins as carrier molecules. Biochem. Phar-macol. (1993) 45:1215–1226.
  • KAMPS JAAM, MORSELT HWM, SWART PJ, MEIJER DKF,SCHERPHOF GL: Massive targeting of liposomes, surface-modified with anionized albumins, to hepatic endothelial cells. Proc. Natl. Acad. ScL USA (1997) 94:11681–11685.
  • WU J, DANIELSSON A: Inhibition of hepatic fibrogene-sis, a review of pharmacological candidates. Scand. J. Gastroenterol. (1994) 29:385–391.
  • EISENSMITH RC, WOO SL: Somatic gene therapy for phenylketonuria and other hepatic deficiencies. J. In-her. Met. Dis. (1996) 19:412–423.
  • BARU M, AXELROD JH, NUR I: Liposome-encapsulated DNA-mediated gene transfer and synthesis of human factor IX in mice. Gene (1995) 161:143–150.
  • MADEN J, BLUM HE: Receptor-mediated delivery of hepatitis B virus DNA and antisense oligodeoxynu-cleotides to avian liver cells. Hepatology (1996) 24:474–481.
  • •A highly efficient receptor-mediated delivery system for DNA and oligodeoxynucleotides (ODNs) to avian liver cells has been established, using complexes of nonmodified hu-man adenovirus particles and a protein conjugate consisting of N-acetyl-glucosamine-modified bovine serum albumin, streptavidin and poly-L-lysine.
  • OZAKI I, DUAN LX, LIU SL, POMERANZ RJ, ZERN MA: Ap-plication of ribozymes for gene therapy of liver dis-ease in alphal-antitrypsin deficiency. In: Molecular Diagnosis and Gene Therapy Blum HE, Wu CH, Wu GY (Eds.), Kluwar Academic Publishers, Dordrecht, (1996) :106–115.
  • SHIMIZU K, MAITANI Y, TAKAYAMA K, NAGAI T: Formu-lation of liposomes with a soybean-derived sterygluco-side mixture and cholesterol for liver targeting. Biol. Pharm. Bull. (1997) 20: 881–886.
  • KAMIMOTO Y, TANABE D, TASHIRO S, HIRAOKA T, MI-YAUCHI Y: Changes in receptor-mediated endocytosis in liver sinusoidal cells after partial hepatectomy in the rat. Liver (1994) 14:141–147.
  • ROSENG L, TOLLESHAUG H, BERG T: Uptake, intracellu-lar transport, and degradation of polyethylene glycol-modified asialofetuin in hepatocytes. J. Biol. Chem. (1992) 267:22987–22993.
  • BECKER S, SPIESS, KLENK HD: The asialoglycoprotein receptor is a potential liver-specific receptor for Mar-burg virus. J. Gen. Vim]. (1995) 76:393–399.
  • CHANG AGY, WU GY: Gene therapy: applications to the treatment of gastrointestinal and liver diseases. Gastro-enterology (1994) 106:1076–1084.
  • ••A leading review article delineating the strategies for genecorrection of GI and liver disorders.
  • YAO T, ESPOSTI SD, HUANG L et al.: Inhibition of carbontetrachloride-induced liver injury by liposomes con-taining vitamin E. Am. J. Physiol. (1994) 267: G476–G484.
  • LIU SL, DEGLI ESPOSTI S, YAO T, DIEHL AM, ZERN MA: Vi-tamin E therapy of acute CC14-induced hepatic injury in mice is associated with inhibition of nuclear factor Kappa B binding. Hepatology (1995) 22:1474–1481.
  • RENSEN PCN, VAN DIJK MCM, HAVENAAR EC et al.: Selec-tive liver targeting of antivirals by recombinant chylo-microns - a new therapeutic approach to hepatitis B. Nature Med. (1995) 1:221–210.
  • ••A new development of targeting approach to the liver, usingrecombinant chylomicrons as carriers for antiviral agents to HBV infection.
  • RENSEN PC, DE VRUEH RL, VAN BERKEL TJ: Targetinghepatitis B therapy to the liver. Clinical pharmacoki-netic considerations. Clin. Pharmacokinet. (1996) 31:131–155.
  • CUMMINGS J: Microspheres as a drug delivery system in cancer therapy. Exp. Opin. Ther. Patents (1998) 8:153–171.
  • ••The present review proposes a hypothesis that the matrixmaterial and method of preparation are critical determinants in defining pharmaceutical characteristics of microspheres, which in turn dictate biologic activity.
  • MAKIN AJ, HUGHES RD, WILLIAMS R: Systemic and he-patic hemodynamic changes in acute liver injury. Am. Physiol. (1997) 35:G617–G625.
  • CODDE JP, LUMSDEN AJ, NAPOLI S, BURTON MA, GRAYBN: A comparative study of the anticancer efficacy of doxorubicin carrying microspheres and liposomes using a rat liver tumour model. Anticancer Res. (1993) 13:539–543.
  • DESOUZA P, D'SOUZA MJ: Formulation and in vitro up-take studies of polylactic co-glycolic microspheres and amino modified latex particles into rat peritoneal macrophages. Pharm. Res. (1990) 7:S162–S169.
  • D'SOUZA MJ, DESOUZA P: Site specific microencapsu-lated drug targeting strategies-liver and gastrointesti-nal tract targeting. Adv. Drug Del. Rev. (1995) 17:247–254.
  • ENGELHARDT JF, YE X, DORANZ B, WILSON JM: Ablationof E2a in recombinant adenoviruses improves trans-gene persistence and decreases inflammatory re-sponse in mouse liver. Proc. Natl. Acad. Sci. USA (1994) 91:6196–6200.
  • WILSON JM: Adenovirus-mediated gene transfer toliver. Adv. Drug. Del. Rev. (1995) 17:303–307.
  • GAO GP, YANG Y, WILSON JM: Biology of adenovirusvectors with El and E4 deletions for liver-directed gene therapy. J. Virol. (1996) 70:8934–8943.
  • •The study indicates that adenovirus vectors with both El and E4 deletions may have advantages in terms of safety and efficacy over first-generation constructs for liver-directed gene therapy.
  • SULLIVAN DE, DASH S, DU H, HIRAMATSU N et al: Liver- directed gene transfer in non-human primates. Hu-man Gene Ther. (1997) 8:1195–1206.
  • •This in vivo study concludes that adenovirus-mediated gene transfer to non-human primate livers via the portal vein or saphenous vein is efficient, but it results in transient expres-sion and is accompanied by an immune response to both vector and transgene products and acute hepatitis. Manipu-lation of the host immune response may expand potential applications of adenovirus vectors for liver-directed gene transfer.
  • ASKARI FK, HITOMI Y, MAO M, WILSON JM: Completecorrection of hyperbilirubinemia in the Gunn rat model of Crigler-Najjar syndrome type I following transient in vivo adenovirus-mediated expression of human bilirubin UDP-glucuronosyltransferase. Gene Ther. (1996) 3:381–388.
  • KIM BH, TAKAHASHI M, TADA K et al.: Cell and genetherapy for inherited deficiency of bilirubin glucu-ronidation. j Perinatol. (1996) 16:S67–S72.
  • ILAN Y, JONA VK, SENGUPTA K et al.: Transient immu-nosuppression with FK506 permits long-term expres-sion of therapeutic genes introduced into the liver using recombinant adenovirus in the rat. Hepatology (1997) 26:949–956.
  • ••This study demonstrated that short-term FK506 treatmentaround the time of virus administration prevents the host im-mune response, permitting long-term gene therapy by re-peated administration of the recombinant virus.
  • YANG Y, GREENOUGH K, WILSON JM: Transient im-mune blockade prevents formation of neutralizing an-tibody to recombinant adenovirus and allows repeated gene transfer to mouse liver. Gene Ther. (1996) 3:412–420.
  • JOOSS K, TURKA LA, WILSON JM: Blunting of immune responses to adenoviral vectors in mouse liver and lung with CTLA41g. Gene Ther. (1998) 5:309–319.
  • ••This study indicates that inhibition of CD4+ T cell activationvia the CD28/B7 pathway at the time of virus instillation can partially interfere with both cellular and humoral immune response to adenovirus-mediated gene transfer circumvent-ing the need for chronic immune suppression.
  • SNYDER RO, MIAO CH, PATIJN GA et al.: Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vec-tors. Nature Genet. (1997) 16:270–276.
  • ••A detailed description of preparation and purification ofAAV vectors and their therapeutical potential in gene ther-apy of hemophilia.
  • FISHER KJ, GAO GP, WEITZMAN MD et al.: Transductionwith recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Vim]. (1996) 70:520–532.
  • LE M, OKUYAMA T, CAI SR et al.: Therapeutic levels offunctional human factor X in rats after retroviral-mediated hepatic gene therapy. Blood (1997) 89:1254–1259.
  • BOWLING WM, KENNEDY SC, CAI SR et al: Portal branchocclusion safely facilitates in vivo retroviral vector transduction of rat liver. Human Gene Ther. (1996) 7:2113–2121.
  • GUNZBURG WH, FLEUCHAUS A, SALLER R, SALMONS B: Retroviral vector targeting for gene therapy. Cytok. Mol. Ther. (1996) 2:177–184.
  • ••In this paper, strategies for redirecting the infection spec-trum of retroviral vectors in order to obtain cell-targeted gene delivery are discussed.
  • KASAHARA N, DOZY AM, KAN YW: Tissue-specific tar-geting of retroviral vectors through ligand-receptor interactions. Science (1994) 266:1373–1375.
  • STRAYER DS: SV40 as an effective gene transfer vector in vivo. J. Biol. Chem. (1996) 271:24741–24746.
  • ••An initial report on the use of replication-deficient recombi-nant 5V40 as a vector for gene transfer in vitro and in vivo.
  • STRAYER DS, MILANO J: SV40 mediates stable genetransfer in vivo. Gene Ther. (1996) 3:581–587.
  • STRAYER DS, KONDO R, MILANO J, DUAN LX: Use ofSV40-based vectors to transduce foreign genes to nor-mal human peripheral blood mononuclear cells. Gene Ther. (1997) 4:219–225.
  • ZERN MA, ZHU J, BAGASRA O, STRAYER DS: SV40-transduced genes are expressed in a high percentage of hepatocytes in vivo. Hepatology (1997) 26:196A.
  • MAHATO RI, ROLLAND A, TOMLINSON E: Cationic lipid-based gene delivery systems: pharmaceutical perspec-tives. Pharmaceut. Res. (1997) 14:853–859
  • ••This review provides a critical discussion on cationic lipid-based gene delivery systems and their possible uses as pharmaceuticals.
  • GAO X, HUANG L: Potentiation of cationic liposome- mediated gene delivery by polycations. Biochemistry (1996) 35:1027–1036.
  • ALINO SF, BOBADILLA M, CRESPO J, LEJARRETA M: Hu-man alpha 1-antitrypsin gene transfer to in vivo mouse hepatocytes. Human Gene Ther. (1996) 7:531–536.
  • TAKEHARA T, HAYASHI N, MIYAMOTO Y et al.: Expres-sion of the hepatitis C virus genome in rat liver after cationic liposome-mediated in vivo gene transfer. He-patology (1995) 21:746–751.
  • RUBIN J, GALANIS E, PITOT HC et al.: Phase I study of im-munotherapy of hepatic metastases of colorectal car-cinoma by direct gene transfer of an allogeneic histocompatibility antigen, HLA-B7. Gene Ther. (1997) 4:419–425.
  • LEWIS JG, LIN KY, KOTHAVALE A et al.: A new serum re-sistant cytofectin for cellular delivery of antisense oli-godeoxynucleotides and plasmid DNA. Proc. Natl. Acad. Sci. USA (1996) 93:3176–3181.
  • SANDIG V, STRAUSS M: Liver-directed gene transfer andapplication to therapy. J. Mol. Med. (1996) 74:205–212.
  • LI S, HUANG L: In vivo gene transfer via intravenous ad-ministration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther. (1997) 4:891–900.
  • ••A report of new formulation of cationic polypeptide-DNA-lipid for in vivo gene delivery, which displays better protec-tion of plasmid DNA against enzymatic digestion and gives high gene expression in the liver following intravenous administration.
  • YANG JP, HUANG L: Overcoming the inhibitory effectof serum on lipofection by increasing the charge ratio of cationic liposome to DNA. Gene Ther. (1997) 4:950–960.
  • EASTMAN SJ, SIEGEL C, TOUSIGNANT J et al.: Biophysical characterization of cationic lipid: DNA complexes. Bio-chim. Biophys. Acta (1997) 1325:41–62.
  • ••One empirical study of characteristics of cationic lipid-DNAcomplexes and optimal charge ratio of the complexes for improving gene delivery efficiency.
  • SCHWARTS B, BENOIST C, ABDALLAH B et al.:Lipospermine-based gene transfer into the newborn mouse brain is otimized by a low lipospermine/DNA charge ratio. Human Gene Ther. (1995) 6:1515–1524.
  • TEMPLETON NS, LASIC DD, FREDERIK PM et al.: Im-proved DNA: liposome complexes for increased sys-temic delivery and gene expression. Nature Biotechnol (1997) 15:647–652.
  • HONG KL, ZHENG WW, BAKER A, PAPAHADJOPHOULOS D: Stabilization of cationic liposome-plasmid com-plexes by polyamines and poly(ethylene glycol)-pho-spholipid conjugates for efficient in vivo gene delivery. FEBS Lett. (1997) 400:233–237.
  • POLLARD H, REMY JS, LOUSSOUARN G et al.: Polyeth-ylenimine but not cationic lipids promotes transgene delivery to the nucleus in mammalian cells. J. Biol. Chem. (1998) 273:7507–7511.
  • ••This study concludes that polymers but not cationic lipidspromote gene delivery from the cytoplasm to the nucleus and that transgene expression in the nucleus is prevented by complexation with cationic lipids but not with cationic polymers.
  • GILL DR, SOUTHERN KW, MOFFORD KA et al.: A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. (1997) 4:199–209.
  • MATTEUCCI MD, WAGNER RW: In pursuit of antisense.Nature (1996) 384:20–22.
  • DEAN NM, MCKAY R, CONDON TP, BENNETT CF: Inhibi-tion of protein kinase C-alpha expression in human A549 cells by antisense oligonucleotides inhibits in-duction of intercellular adhesion molecule 1 (ICAM-1) mRNA by phorbol esters. J. Biol. Chem. (1994) 269:16416–16424.
  • MAHATO RI, KAWABATA K, NOMURA T, TAKAKURA Y,HASHIDA M: Physicochemical and pharmacokinetic characteristics of plasmid DNA cationic liposome complexes. J. Pharmaceut. ScL (1995) 84:1267–1271.
  • KOIKE K, HARA T, ARAMAK Y, TAKADA S, TSUCHIYA S: Receptor-mediated gene transfer into hepatic cells us-ing asialoglycoprotein-labeled liposomes. Ann. NY Acad. Sci. (1994) 716:331–333.
  • BEHR JP: Gene transfer with a series of lipophilic DNA-binding molecules. Bioconjugate Chem. (1994) 5:647–654.
  • KICHLER A, REMY JS, BOUSSIF O et al: Efficient gene de-livery with neutral complexes of lipospermine and thiol-reactive phospholipids. Biochem. Biophys. Res. Commun. (1995) 209:444–450.
  • HARA T, ARAMAKI Y, TAKADA S, KOIKE K, TSUCHIYA S:Receptor-mediated transfer of pSV2CAT DNA to a hu-man hepatoblastoma cell line Hep G2 using asialofetuin-labeled cationic liposomes. Gene (1995) 159:167–174.
  • LONGINO MA, BAKAN DA, WEICHERT JP, COUNSELL RE: Formulation of polyiodinated triglyceride analogues in a chylomicron remnant-like liver selective delivery vehicle. Pharmacol. Res. (1996) 13:875–879.
  • HARA T, TAN Y, HUANG L: In vivo gene delivery to the liver using reconstituted chylomicron remnants as a novel nonviral vector. Proc. Natl. Acad. Sci. USA (1997) 94:14547–14552.
  • •An initial report of reconstituted chylomicron remnants used as a vector for gene delivery.
  • UN DY, UN SM, LIAW YF: Non-surgical treatment of he- patocellular carcinoma. J. Gastroenterol Hepatol (1997) 12:S319–S328.
  • HALL SJ, CHEN SH, WOO SL: The promise and reality of cancer gene therapy. Am. J. Human Genetics (1997) 61:785–789.
  • ••An overview of adenovirus vector-mediated transfection ofsuicide gene for genetic therapy of HCC and other tumours.
  • HIRSCHOWITZ EA, OHVVADA A, PASCAL WR, RUSSI TJ, CRYSTAL RG: In vivo adenovirus-mediated gene trans-fer of the Escherichia Co/icytosine deaminase gene to human colon carcinoma-derived tumors induces che-mosensitivity to 5-fluorocytosine. Humam Gene Ther. (1995) 6:1055–1063.
  • KANAI F, LAN KH, SHIRATORI Y et al: In vivo gene ther-apy for alpha-fetoprotein-producing hepatocellular carcinoma by adenovirus-mediated transfer of cyto-sine deaminase gene. Cancer Res. (1997) 57:461–465.
  • SU H, CHANG JC, XU SM, KAN YW: Selective killing of AFP-positive hepatocellular carcinoma cells by adeno-associated virus transfer of the herpes simplex virus thymidinekinase gene. Human Gene Ther. (1996) 7:463–470.
  • QIAN C, IDOATE M, BILBAO R et al.: Gene transfer andtherapy with adenoviral vector in rats with diethylnitrosamine-induced hepatocellular carci-noma. Human Gene Ther. (1997) 8:349–358.
  • •In vivo gene transfer efficiency was studied using intraportal injections of recombinant adenovirus carrying the lacZ re-porter gene (AdCMVlacZ), and the therapeutic efficacy of adenovirus-mediated transfer of the thymidine kinase gene of the herpes simplex virus followed by ganciclovir administration.
  • VON WEIZSACKER F, WIELAND S, KOCK J et al.: Gene therapy for chronic viral hepatitis: ribozymes, an-tisense oligonucleotides, and dominant negative mu-tants. Hepatology (1997) 26:251–255.
  • WU GY, WU CH: Specific inhibition of hepatitis B viral gene expression in vitro by targeted antisense oligo-nucleotides. J. Biol. Chem. (1992) 267:12436–12439.
  • KORBA BE, GERIN JL: Antisense oligonucleotides are ef-fective inhibitors of hepatitis B virus replication in vi-tro. Antiviral Res. (1995) 28:225–242.
  • RUIZ J, WU GY: Liver-selective DNA targeting using the asialoglycoprotein receptor. Biogenic Amines (1998). (In Press.)
  • ••This review focuses on general aspects related to targetingthe liver by the asialoglycoprotein receptor as well as strate-gies aimed to increase gene expression.
  • NAKAZONO K, ITO Y, WU CH, WU GY: Inhibition of hepatitis B virus replication by targeted pretreatment of complexed antisense DNA in vitro. Hepatology (1996) 23:1297–1303.
  • JI W, ST CW: Inhibition of hepatitis B virus by retroviral vectors expressing antisense RNA. J. Viral. Hepatitis (1997) 4:167–173.
  • •It was demonstrated that the replication and expression of HBV can be inhibited through antisense gene transfer medi-ated by retroviral vectors and that the antisense-preC/C or antisense-preS/S may be potentially useful for clinical gene therapy against HBV.
  • BRANCH AD: A hitchhiker's guide to antisense and nonsense biochemical pathways. Hepatology (1996) 24:1517–1529.
  • •An overview of advantages and disadvantages of antisense-mediated inhibition of gene expression.
  • BECK J, NASSAL M: Efficient hammerhead ribozyme-mediated cleavage of the structured hepatitis B virus encapsidation signal in vitro and in cell extracts, but not in intact cells. Nucleic Acids Res. (1995) 23:4954–4962.
  • RUIZ J, WU CH, ITO Y, WU GY: Design and preparation of a multimeric self-cleaving hammerhead ribozyme. Biotechniques (1997) 22:338–345.
  • WELCH PJ, TRITZ R, YET S, BARBER J, YU M: Intracellular application of hairpin ribozyme genes against hepati-tis B virus. Gene Ther. (1997) 4:736–743.
  • SAKAMOTO N, WU CH, WU GY: Intracellular cleavage of hepatitis C virus RNA and inhibition of viral protein translation by hammerhead ribozymes. J. Clin. Invest. (1996) 98:2720–2728.
  • LIEBER A, HE CY, POLYAK SJ et al: Elimination of hepati-tis C virus RNA in infected human hepatocytes by adenovirus-mediated expression of ribozymes. J Virol (1996) 70:8782–8791.
  • SCAGLIONI P, MELEGARI M, TAKAHASHI M, CHOWD-HURY JR, WANDS J: Use of dominant negative mutants of the hepadnaviral core protein as antiviral agents. Hepatology (1996) 24:1010–1017.
  • WANDS JR, GEISSLER M, PUTLITZ JZ et al.: Nucleic acid-based antiviral and gene therapy of chronic hepatitis B infection. J. Gastroenterol. Hepatol. (1997) 12:S354–S369.
  • ALINO SF: Long-term expression of the human al-antitrypsin gene in mice: employing anionic and cati-onic liposome vector. Biochem. PharmacoL (1997) 54:9–13.
  • ••A summary of the use of small anionic liposomes and cati-onic liposomes in the delivery of the human al-AT gene in mouse liver.
  • CRESPO J, BLAYA C, CRESPO A, ALINO SF: Long-term ex-pression of humanal-antitrypsin gene in mice em-ploying anionic and cationic liposome vectors. Biochem. Pharmacol. (1996) 51:1309–1314.
  • ALINO SF, CRESPO J, BOBADILLA M et al.: Expression of human al-antitrypsin in mouse after in vivo gene transfer to hepatocytes by small liposomes. Biochem. Biophys. Res. Commun. (1994) 204:1023–1030.
  • KNOELL DL, WEWER MD: Clinical implications of gene therapy for alpha-sub-antitrypsin deficiency. Chest (1995) 107:535–545.
  • ••This comprehensive review delineates pathophysiology ofal-AT deficiency and reviews various approaches for ge-netical delivery of al-AT gene.
  • KAY MA, LI Q, LIU TJ et al.: Hepatic gene therapy: persis-tent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Human Gene Therapy. (1992) 3:641–647
  • KAY MA, BALEY P, ROTHENBERG S et al.: Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc. Natl. Acad. Sci. USA (1992) 89:89–93.
  • HAFENRICHTER DG, PONDER KP, RETTINGER SD et al.: Liver-directed gene therapy: evaluation of liver spe-cific promoter elements. J. Surg. Res. (1994) 56:510–517.
  • OKUYAMA T, HUBER RM, BOWLING W et al.: Liver-directed gene therapy: a retroviral vector with a com-plete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of hu-man alpha 1-antitrypsin in vivo. Human Gene Ther. (1996) 7:637–645.
  • SCHIEDNER G, MORRAL N, PARKS RJ et al.: Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nature Genet. (1998) 18:180–183.
  • EISENSMITH RC, WOO SL: Viral vector-mediated gene therapy for hemophilia B. Thrombosis Haemostasis (1997) 78:24–30.
  • KAY MA: Hepatic gene therapy for hemophilia B. In: In-hibitors to Coagulation Factors, Aledort LM et al., (Eds.), Ple-num Press, New York (1995):229–234.
  • GROVE RI, WU GY: Preclinical trials using hepatic gene therapy. Adv. Drug Del. Revs. (1998) 30:199–204.
  • ••This review outlines the more commonly used approachesand discusses the progress that has been made toward de-veloping a widely applicable, clinically relevant gene trans-fer procedure for the liver.
  • OVYANG EC, WU GY: Gene therapy: recent advances and application in gastroenterology and hepatology. J. Gastroenterol. Hepatol (1998) (In Press.)
  • ••An extensive overview of progress in the development ofvectors for gene therapy, as well as a description of experi-mental and clinical trials for the correction of the genetic de-ficiencies, viral hepatitis, and hepatocellular carcinoma.
  • KOEBERL DD, ALEXANDER IE, HALBERT CL, RUSSELL DW, MILLER AD: Persistent expression of human clot-ting factor IX from mouse liver after intravenous injec-tion of adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA (1997) 94:1426–1431.
  • ••A report of the application of AAV vectors in correction ofhemophilia in a mouse model. Injection of mice with AAV vectors encoding human clotting factor IX after 7-irradiation resulted in synthesis of low levels of human clotting factor IX for the 5-month period of observation.
  • HERZOG RW, HAGSTROM JN, KUNG SH et al.: Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA (1997) 94: 5804–5809.
  • GROSSMAN M, RADER DJ, MULLER WM et al.: A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia. Nature Med. (1995) 1:1148–1154.
  • GROSSMAN M, RAPE SE, KOZARSKY K et al.: Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia. Nature Genetics (1994) 6:335–341.
  • RAPER SE, GROSSMAN M, RADER DJ et al: Safety and fea-sibility of liver-directed ex vivo gene therapy for ho-mozygous familial hypercholesterolemia. Ann. Surg. (1996) 223:116–126.
  • LI J, FANG B, EISENSMITH RC et al.: In vivo gene therapy for hyperlipidemia: phenotypic correction in Watan-abe rabbits by hepatic delivery of the rabbit LDL recep-tor gene. J. Clin. Invest. (1995) 95:768–773.
  • WILSON JM, GROSSMANJ M, WU CH et al.: Hepatocyte-directed gene transfer in vivo leads to transient im-provement of hypercholesterolemia in LDL receptor-deficient rabbits. J. Biol. Chem. (1992) 267:963–967.
  • WILSON JM, GROSSMAN M, CABRERA JM, WU CH, WU GY: A novel mechanism for achieving transgene persis-tence in vivo following somatic gene transfer into he-patocytes. J. Biol. Chem. (1992) 267:11483–11489.
  • GERARD RD, COLLEN D: Adenovirus gene therapy for hypercholesterolemia, thrombosis and restenosis. Cardiovascular Res. (1997) 35:451–458.
  • ••This review summarises general properties of recombinantadenovirus vectors and their application in metabolic and vascular disorders, including hypercholaesterolemia.
  • ROY CHOWDHURY N, HAYS RM, BOMMINENI VR et al.: Microtubular disruption prolongs the expression of human bilirubin-uridinediphosphoglucuronate glucuronosyltransferase-1 gene transferred into Gunn rat livers. J. Biol. Chem. (1996) 271:2341–2346.
  • TAKAHASHI M, ILAN Y, ROY CHOWDHURY N et al: Long term correction of bilirubin-UDP-glucuronosyl trans-ferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period. J. Biol. Chem. (1996) 271:26536–26542.
  • KORMIS KK, WU GY: Prospects of therapy of liver dis-eases with foreign genes. Sem. Liver Dis. (1995) 15:257–267.

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