5
Views
1
CrossRef citations to date
0
Altmetric
Original Article

Section Reviews: Biologicals & Immunologicals: Recent advances in non-viral gene therapy

Pages 1101-1115 | Published online: 03 Mar 2008

References to Primarv Literature

  • WOLFF JA, MALONE RW, WILLIAMS P et al.: Direct gene transfer into mouse muscle in vivo. Science (1990) 247:1465-1468. The first description of direct injection of RNA and DNA expression vectors into skeletal muscle in vivo - reporter gene activity was present in the muscle for at least 2 months post-injection.
  • JIAO S, WILLIAMS P, BERG RN et al.: Direct gene transfer Into nonhuman primate myollbers in vivo. Hum. Gene Tber. (1992) 3:21–33.
  • ACSADI G, JIAO SS, JANI A et at.: Direct gene transfer andexpression into rat heart in vivo. New Biol. (1991) 3:71–81.
  • YOVANDICH J, O'MALLEY B, Jr., SIKES M, LEDLEY FD: Gene transfer to synovial cells by intra-articular ad-ministration of plasmid DNA. Hum. Gene Tber. (1995) 6:603-610. Demonstrates that direct administration of an expression plasmid into the knees of rabbits and rats results in gene expression in synovial tissue (cf. skeletal muscle). DNA was taken up rapidly by non-specific endocytosis and, despite degradation, gene expression persisted for 2–5 days after administration.
  • DAVIS HL, DEMENEIX BA, QUANTIN B, COLTLOMBE J,WHALEN RG: Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal mus-cle. Hum, Gene 7ber. (1993) 4:733–740.
  • WOLFF JA, LUDTKE JJ, ACSADI G, WILLIAMS P, JAN! A:Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. (1992) 1:363–369.
  • WOLFF JA, DOWTY ME, JIA0 S et al.: Expression of naked plasmids by cultured tuyotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle. J. Cell. Sci. (1992) 103:1249–1259.
  • DOWTY ME, WOLFF JA: Possible mechanisms of DNA uptake in skeletal muscle. In: Gene Therapeutics: Methods and Applications of Direct Gene Transfer. Wolff JA (Ed.), Birkhauser, Boston (1994):82–89.
  • LEVY MY, BARRON LG, MEYER KB, SZOKA FC: Characterization of plasmid DNA transfer into mouse skeletal muscle: evaluation of uptake mechanism, expression and secretion of gene products into blood. Gene Ther. (1996) 3:201-211. Demonstrates the potential of direct injection into muscle to achieve therapeutic levels of a secreted protein in vivo.
  • DOWTY ME, WILLIAMS P, ZHANG G, HAGSTROM JE, WOLFF JA: Plasmid DNA entry into postmitotic nuclei of primary rat myotubes. Proc. Natl. Acad. Sci. USA (1995) 92:4572–4576.
  • FURTH PA, SHAMAY A, WALL RJ, HENNIGHAUSEN L: Genetransfer into somatic tissues by jet injection. Anal. Biochem. (1992)205:365–368.
  • DAVIS HL, WHALEN RG, DEMENELX BA: Direct gene transfer into skeletal muscle in vivo: factors affecting efficiency of transfer and stability of expression. Hum. Gene Ther. (1993) 4:151–159.
  • DANKO I, FRITZ JD, BAG S et al.: Pharmacological en-hancement of in vivo foreign gene expression in mus-cle. Gene Ther. (1994) 1:114-121. Describes studies to examine the effects of treating muscle with a range of agents to enhance gene expression following direct injec-tion of naked plasmid DNA. Up to 40-fold enhancement was achieved which persisted for one month.
  • ACSADI G, DICKSON G, LOVE DR et al.: Human dystro-phin expression in mdx mice after intramuscular in-jection of DNA constructs. Nature (1991) 52:815–818.
  • YAO SN, SMITH KJ, KURACIII K: Primary myoblast-me-diated gene transfer: persistent expression of human factor IX in mice. Gene Ther. (1994) 1:99–107.
  • HAMAMORI Y, SAMAL B, TIAN J, KEDES L: Persistenterythropoiesis by myoblast transfer of erythropoietin cDNA. Hum. Gene Ther. (1994) 5:1349–1356.
  • ULMER JB, DONNELLY JJ, PARKER SE et at.: Heterologous protection against influenza by injection of DNA encod-ing a viral protein. Science (1993) 259:1745-1749. Describes the use of a plasmid encoding influenza A nucleoprotein to generate antigen for presentation to the immune system by injection into muscle. Resulted in protection from a subsequent challenge with a heterologous strain of influenza A virus.
  • DAVIS FIL, MICHEL ML, WHALEN RG: DNA-based immu-nization induces continuous secretion of hepatitis B surface antigen and high levels of circulating antibody. Hum. Mol. Genet. (1993) 2:1847–1851.
  • DAVIS HL, MICIIEL ML, WHALEN RG: Use of plasmid DNAfor direct gene transfer and immunization. Ann. NY Acad. Sci. (1995) 772:21–29.
  • ULMER J13, DONNELLY JJ, DECK RR, DEWITT CM, LIU MA:Immunization against viral proteins with naked DNA. Ann. NY Acad. Sci. (1995) 772:117–125.
  • DONNELLY B, FRIEDMAN A, MARTINEZ D et al.: Preclinical efficacy of a prototype DNA vaccine: enhanced protection against antigenic drift in influenza virus. Nat. Med. (1995) 1:583-587. A comparison of a DNA viral vaccine and conventional, clinically licensed vaccines in preclinical animal models. The DNA vaccine elicited antibody responses and protected against challenge with an antigenically distinct virus more effectively than the clinically li-censed vaccine.
  • YANG NS, BURKHOLDER J, ROBERTS B, MARTINELL B,MCCABE D: In vivo and in vitro gene transfer to mam-malian somatic cells by particle bombardment. Proc. Natl. Acad. Sci. USA (1990) 87:9568–9572.
  • QIU P, ZIEGELHOFFER P, SUN J, YANG NS: Gene gundelivery of mRNA in situ results in efficient transgene expression and genetic immunization. Gen. Ther. (1996) 3:262–268.
  • PERTMER TM, EISENBRAUN MD, MCCABE D et at.: Gene gun-based nucleic acid immunization: elicitation of humoral and cytotoxic T lymphocyte responses follow-ing epidermal delivery of nanogram quantities of DNA. Vaccine (1995) 13:1427-1430, Describes the development and use of a simple, hand-held device capable of delivering plasmid DNA-based antigen expression vectors to the epidermis in vivo and eliciting an immune response.
  • CHENG L, ZIEGELHOFFER PR, YANG NS: In vivo promoter activity and transgene expression in mammalian so-matic tissues evaluated by using particle bombardment. Proc. Natl. Acad. Sci. USA (1993) 90:4455–4459.
  • WILLIAMS RSJOHNSTON SA, RIEDY M et at,: Introductionof foreign genes into tissues of living mice by DNA-coated microprojectiles. Proc. Natl. Acad. Sci. USA (1991) 88:2726–2730.
  • FULLER JT, FULLER DH, MCCABE D, HAYNES JR, WIDERAG: Immune responses to hepatitis B virus surface and core antigens in mice, monkeys, and pigs after Accell particle-mediated DNA immunization. Ann. NY Acad. Sci. (1995) 772:282–284.
  • ROBINSON HL, HUNT LA, WEBSTER RG: Protection against a lethal influenza virus challenge by immuniza-tion with a haeraagglutinin-expressing plasmid DNA. Vaccine (1993) 11:957–960.
  • SUN WH, BURKHOLDER JK, SUN J et al.: In vivo cytokinegene transfer by gene gun reduces tumor growth in mice. Proc. Nail, Acad. Sci. USA (1995) 92:2889–2893.
  • ANDREE C, SWAIN WE, PAGE CF et at.: In vivo transfer and expression of a human epidermal growth factor gene accelerates wound repair. Proc. Natl. Acad. Sci. USA (1994) 91:12188-12192. Demonstrates the possible utility of in vivo gene transfer to enhance epidermal repair. Local delivery of a human EGF expression plasmid to a wound in a porcine model resulted in a 190-fold increase in EGF concentration and more rapid (20%, 2.1 days) healing cf. controls.
  • RIESSEN R, RAHIMIZADEH H, BLESSING E et al.: Arterial gene transfer using pure DNA applied directly to a hydrogel-coated angioplasty balloon. Hum. Gene Ther. (1993) 4:749–758.
  • SIMONS M, EDELMAN ER, DEKEYSER JL, LANGER R,ROSENBERG RD: Antisense c-myb oligonudeotides in-hibit intimal arterial smooth muscle cell accumulation in vivo. Nature (1992) 359:67–70.
  • PHILLIPS SC: Receptor-mediated DNA delivery ap-proaches to human gene therapy. Biologicals (1995) 23:13–16.
  • KUKOWSKA-LATALLO JF, BIELINSICA AU, JOHNSON J et al.:Efficient transfer of genetic material into mammalian cells using starburst polyamidoamine dendrimers. Proc. Natl. Acad. Sci. USA (1996) 93:4897–4902.
  • BOUSSIF O, LEZOUALC'H F, ZANTA MA et al.: A versatilevector for gene and oligonucleotide transfer into cells In culture and in vivo: polyethylenimine. Proc. Nall. Acad. Set USA (1995) 92:7297–7301.
  • FELGNER PL, GADEK TR, HOLM M et al.: Lipofection; a highly efficient, lipid-mediated DNA-transfection procedure. Proc. Natl. Acad. Sci. USA (1987) 84:7413-7417. The first description of a synthetic cationic lipid (DOTMA) for DNA-transfection.
  • MALONE RW, FELGNER PL, VERMA IM: Cationic liposome-mediated RNA transfection. Proc. Natl. Acad. Sci. USA (1989) 86:6077–6081.
  • FELGNER JH, KUMAR R, SRIDHAR CN et al.: Enhanced genedelivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. (1994) 269:2550–2561.
  • JIAO S, ACSADI G, JANI A, FELGNER PL, WOLFF JA: Per-sistence of plasmid DNA and expression in rat brain cells in vivo. Exp. Neurol. (1992) 115:400–413.
  • FARHOOD H, SERBINA N, HUANG L: The role of dioleoyl phosphatidylethanolamine in cationic liposome medi-ated gene transfer. Biochim. Biophys. Acta. (1995) 1235:289-295. Proposes an endocytosis model of DNA delivery by cationic liposomes in which agents such as DOPE can enhance gene expression by destabilising the endosome membrane and promote the release of DNA into the cytosol.
  • PINNADUWAGE P, SCHMITT L, HUANG L: Use of a quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells. Biochim. Biophys. Acta (1989) 985:33–37.
  • GAO XA, HUANG L: A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. (1991) 179:280–285,
  • FARHOOD H, BOlIEGA R, EPAND RM, HUANG L: Effect of cationic cholesterol derivatives on gene transfer and protein kinase C activity. Biochim. Biophys. Acta (1992) 1111:239–246.
  • ZHOU X, HUANG L: DNA transfection mediated by cationic liposomes containing lipopolylysine: charac-terization and mechanism of action. Biochim. Biophys. Acta (1994) 1189:195–203.
  • FELGNER PL, TSAI YJ, SUKHU L et al.: Improved cationic lipid formulations for in vivo gene therapy. Ann. NY Acad. Sci. (1995) 772:126-139. A report outlining the limitations and some recent developments in synthetic cationic lipid-based gene delivery systems.
  • CAPLEN NJ, KINRADE E, SORGI F et al.: In vitro liposome-mediated DNA transfection of epithelial cell lines using the cationic liposome DC-Chol/DOPE. Gene Ther. (1995) 2:603–613.
  • SAN II, YANG ZY, POMPILI VI et al.: Safety and short-termtoxicity of a novel cationic lipid formulation for human gene therapy. Hum. Gene Ther. (1993) 4:781–788.
  • MCLACHLAN G, DAVIDSON DJ, STEVENSON BJ et al.: Evaluation in vitro and in vivo of cationic liposome-ex-pression construct complexes for cystic fibrosis gene therapy. Gene Ther. (1995) 2:614–622.
  • BUCHBERGER B, FERNHOLZ E, ELTZ H, HINZPETER M:DOSPER liposomal transfection reagent: a reagent with unique transfection properties. Biochemica (1994) 11(2):7–10.
  • CICCARONE V, ANDERSON A, LAN J, SCHIFFERLI K, JESSEEJ: DMRIE-C reagent for transfection of suspension cells and for RNA transfections. FOCUS (1995) 17(3):84–86.
  • MCDONALD AS, SCHIFFERLI K, ANDERSON D, JESSEE J, C1CCARONE V: A simple method for cationic lipid re-agent selection for transfection. FOCUS (1996) 18(1)4–6.
  • BEHR JP, DEMENEIX B, LOEFFLER JP, PEREZ-MUTUL J: Efficient gene transfer into mammalian primary endo-crine cells with lipopolyamlne-coated DNA. Proc. Natl. Acad. Sci. USA (1989) 86:6982–6986.
  • TSUKAMOTO M, OCHIYA T, YOSHIDA S, SUGIMURA T,TERADA M: Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice. Nature Genetics (1995) 9:243–248.
  • BENNETT MJ, NANTZ MH, BALASUBRAMANIAM RP, GRUENERT DC, MALONE RW: Cholesterol enhances cat-ionic liposome-mediated DNA transfection of human respiratory epithelial cells. Biosci. Rep. (1995) 15:47–53.
  • ABERLE AM, BENNETT MJ, MALONE RW, NANTZ MH: Thecounterion influence on cationic lipid-mediated trans-fection of plasmid DNA. Biochim. Biophys. Acta (19%) 1299:281–283.
  • GUSTAFSSON J, ARVIDSON G, 'CARLSSON G, ALMGREN M:Complexes between cationic liposomes and DNA visu-alized by cryo-TEM. Biochim. Biophys. Acta. (1995) 1235:305–312.
  • STERNBERG B, SORGI FL, HUANG L: New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron mi-croscopy. FEBS Lett. (1994) 356:361–366.
  • ALLEN TM: Long-circulating (sterically-stabilised) liposomes for targeted drug delivery. TiPS (1994) 15:215–220.
  • GAO X, HLTANG L: Potentiation of cationic liposome-me-diated gene delivery by polycations. Biochemistry (19%) 35:1027-1036.
  • KICHLER A, REMY JS, BOUSSIF O et al.: Efficient gene delivery with neutral complexes of lipospermine and thiol-reactive phospholipids. Biochem. Biophys. Res, Commun. (1995) 209:444-450. Describes the use of thiol-reactive phospholipid derivatives with electrically neutral lipospermine/DNA complexes to enhance trans-fection efficiency in vitro (100-fold). It is proposed that the com-plexes become covalently anchored to the cells and are then endocytosed.
  • ALLEN TM, BRANDEIS E, HANSEN CB, ICAO GY, ZALIRSKY S: A new strategy for attachment of antibodies to steri-cally stabilized liposomes resulting in effident target-ing to cancer cells. Biochim. Biophys. Acta (1995) 1237:99–108.
  • MARUYAMA K, TAKIZAWA T, YUDA T et al.: Targetability of novel immunoliposomes modified with amphipa-thic poly(ethylene gitycol)s conjugated at their distal terminals to monoclonal antibodies. Biochim. Biophys. Acta. (1995) 1234:74-80. Presents a simple means of conjugating antibodies via PEG to liposomes for targeted drug and gene delivery. Modest targeting was observed, and PEG prolonged the circulation time and reduced reticulo-endothelial system uptake.
  • CHONN A, CULLIS PR: Recent advances in liposomaldrug-delivery systems. Curr. Opin. Biotechnol. (1995) 6:698–708.
  • PHILLIPS NC, DAHMAN J: Immunogenicity of immuno-liposomes: reactivity against species-specific IgG and liposomal phospholipids. Immunol. Lett. (1995) 45:149–152.
  • IIARA T, ARAMAKI Y, TAKADA S, KOIKE K, TSUCHIYA S: •Receptor-mediated transfer of pSV2CAT DNA to a human hepatoblastoma cell line HepG2 using asialofetuin-labeled cationic liposomes. Gene (1995) 159:167-174. Describes the use of asialofetuin-labelled liposomes to deliver a reporter gene to FlepG2 cells. Uptake was competitively inhibited by the addition of free asialofetuin and cytochalasin B, and reporter gene expression was enhanced relative to non-labelled control liposomes.
  • WANG S, LEE RJ, CAUCHON G, GORENSTEIN DG, LOW PS: Delivery of antisense oligodeoxyribonucleotides against the human epidermal growth factor receptor into cultured KB cells with liposomes conjugated to folate via polyethylene glycoL Proc. Natl. Acad. Sci. USA (1995) 92:3318–3322.
  • REMY JS, KICHLER A, MORDVINOV V, SCHUBER F, BEHR JP: Targeted gene transfer into hepatoma cells withlipopolyamine-condensed DNA partides presenting galactose ligands: a stage toward artificial viruses. Proc. Natl. Acad. Sci. USA (1995) 92:1744-1748. Outlines the development of a modular transfection system based on lipid-coated DNA particles to which other synthetic lipids with targeting moieties, e.g., a triantennary galactosyl residue which targets the complex to the asialoglycoprotein receptor of HepG2 cells, or key viral properties are hydrophobically adsorbed.
  • KATO K, KANEDA Y, SAKURAI M, NAKANISHI M, OKADA •Y: Direct injection of hepatitis B virus DNA into liver Induced hepatitis in adult rats. J. Biol. Chem. (1991) 266:22071-22074. Establishment and use of an efficient method for gene transfer in vivo using liposomes incorporating the haemagglutinating virus of Japan (HVJ) to mediate membrane fusion.
  • SCHOFIELD JP, CASKEY CT: Non-viral approaches to gene therapy. Br. Med. Bull. (1995) 51(1):56–71.
  • MORISHITA R, GIBBONS GLI, KANEDA Y, OGIIIARA T, DZAU VJ: Novel in vitro gene transfer method for study of local modulators in vascular smooth muscle cells. Hypertension (1993) 21:894–899.
  • TOMITA N, MORISHITA R, HIGAKI J et al.: Transient decrease in high blood pressure by in vivo transfer of antisense oligodeoxynudeotides against rat angiotensi-nogen. Hypertension (1995) 26:131-136. Demonstrates that in vivo transfection of antisense oligonucleotides against rat angiotensinogen can elicit a transient (up to 7 days post-injection) decrease in the blood pressure of spontaneously hypertensive rats.
  • MORISHITA R, GIBBONS GH, HORIUCHI M et al.: A gene therapy strategy using a transcription factor decoy of the E 2F binding site inhibits smooth muscle proliferation in vivo. Proc. Natl. Acad. Sci. USA (1995) 92:5855-5859. An elegant demonstration of enhanced liposome-mediated delivery of a transcription factor decoy which can modulate gene expression and inhibit smooth muscle proliferation and vascular lesion forma-tion in vivo.
  • KATO K, YONEDA Y, OKADA Y, KIYAMA H, SHIOSAKA S:Gene transfer and. the expression of a foreign gene in vivo in post-mitotic neurons of the adult rat brain using the hemagglutinating virus of the Japanliposome method. Brain Res. Mol. Brain Res. (1994) 25:359–363.
  • SCHREIER H, GONZALEZ-ROTHI RJ, STECENKO AA: Pul-monary delivery of liposomes. J. Contr. Release (1993) 24:209–223.
  • NABEL GJ, YANG ZY, NABEL EG et al.: Direct gene transferfor treatment of human cancer. Ann. NY Acad. Sci. (1995) 772:227–231.
  • CONARY Jef, PARKER RE, CHRISTMAN BW et al.: Protectionof rabbit lungs from endotoxin injury by in vivo hyper-expression of the prostaglandin G/H synthase gene. J. Clin. Invest. (1994) 93:1834–1840.
  • CAPLEN NJ, ALTON EW, MIDDLETON PG et al.: Liposomemediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med. (1995) 1:39-46. First placebo-controlled clinical trial of cationic liposome-mediated CFTR cDNA delivery to CF patients. No adverse clinical effects were seen, however, transfection efficiency and the duration of expression was insufficient for therapeutic benefit.
  • ZABNER J, FASBENDER AJ, MONINGER T, POELLINGER KA, WELSH MJ: Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. (1995) 270:18997-19007. The results of this comprehensive study provide an insight into the physical limitations to cationic lipid-mediated gene transfer, and indicates that improvements in the cellular processing of the com-plexes may further improve the efficiency of functional gene deliv-ery.
  • WROBEL I, COLLINS D: Fusion of cationic liposomes with mammalian cells occurs after endocytosis. Biochim. Biophys. Acta (1995) 1235:296-304. The data presented demonstrates that binding of cationic liposomes to the cell surface is insufficient for cationic liposome-cell fusion, and that uptake into the endocytic pathway is required for fusion to °COM%
  • WU GY, WU CH: Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J. Biol. Chem. (1987) 262:4429–4432.
  • WU GY, WU CH: Receptor-mediated gene delivery and expression in vivo. J. Biol. Chem. (1988) 263:14621–14624.
  • WAGNER E, ZENKE M, COT1EN M, BEUG II, BIRNSTIEL ML: Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. USA (1990) 87:3410–3414.
  • ROSENKRANZ AA, YACHMENEV SV, JANS DA et al.: Recep-tor-mediated endocytosis and nuclear transport of a transfecting DNA construct. Exp. Cell. Res. (1992) 199:323–329.
  • GOTTSCHALK S, CHRISTIAN° RJ, SMITE LC, WOO SL: Folate receptor-mediated DNA delivery into tumor cells: potosomal disruption results in enhanced gene expres-sion. Gene Ther. (1993) 1:185-191. It has been suggested that folate-mediated uptake of DNA may differ to that of peptide ligands in that it is routed through the potosome, rather than the endosome.
  • TRUBETSKOY VS, TORCIIILIN VP, KENNEL SJ, HUANG L:Use of N-terminal modified poly(L-lysine)-antibody conjugate as a carrier for targeted gene delivery in mouse lung endothelial cells. Bioconjug. Chem. (1992) 3: 32 3–327.
  • CIIEN J, GAMOU S, TAKAYANAGI A, SHIMIZU N: A novelgene delivery system using EGF receptor-mediated en-docytosis. FESS Lett. (1994) 338:167–169.
  • FERKOL T, PERALES JC, ECKMAN E et al.: Gene transferInto the airway epithelium of animals by targeting the polymeric ircununog,lobulin receptor. J. Clin. Invest. (1995) 95:493–502.
  • THURNHER M, WAGNER E, CLAUSEN H et al.: Carbohy-drate receptor-mediated gene transfer to human T leukaemic cells. Glycobiology (1994) 4:429–435.
  • FERKOL T, PERALES JC, AMULARO F, HANSON RW: Receptor-mediated gene transfer into macrophages. Proc. Natl. Acad. Sci. USA (1996) 93:101-105. Use of a synthetic molecular conjugate, consisting of mannosylated polylysine that exploits endocytosis via the macrophage mannose receptor, is described. Reporter gene expression in macrophages residing in the spleen and liver of adult mice was maximal at 4 days after transfection and decreased to lower levels by 16 days.
  • WADHWA MS, RICE KG: Receptor mediated glycotargeting. J. Drug Target. (1995) 3:111-127. This review compares different types of glycotargeting agents and the lectins which have been successfully employed to treat both model and human diseases. The major applications are in antiviral therapy, immuno-activation, enzyme replacement therapy and gene therapy.
  • FERKOL T, LINDBERG GL, CHEN J et al.: Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer. FASE 8 J. (1993) 7:1081-1091. A synthetic neoglycoprotein carrier was prepared and used to deliver a reporter gene to rat liver (via the asialoglycoprotein receptor) after partial hepatectomy - reporter gene expression was detected up to 30 days after delivery.
  • LONGLEY C, AXELROD H, MIDHA S et al.: Conjugates ofglycosylated steroids and polyarnines as novel nonviral gene delivery systems. Ann. NY Acad. Sci. (1995) 772:268–270.
  • PLANK C, ZATLOUKAL K, CO1 LEN M, MECH'FLER K, WAG-NER E: Gene transfer into hepatocytes using as ialoglyco-protein receptor mediated endocytosis of DNA completed with an artificial tetra-antennary galactose ligand. Bioconjug. Chem. (1992) 3:533–539.
  • HART SL, HARBOTTLE RP, COOPER R et al.: Gene deliveryand expression mediated by an integrin-binding pep-tide. Gene Ther. (1995) 2:552–554.
  • WAGNER E, COTTEN M, FOISNER R, BIRNSTIEL ML: Trans-ferrin-polycation-DNA complexes: the effect of polyca-dons on the structure of the complex and DNA delivery to cells. Proc. Natl. Acad. Sci. USA (1991) 88:4255–4259.
  • BOTTGER M, VOGEL F, PLATZER M et al.: Condensationof vector DNA by the chromosomal protein HMG1 results in efficient transfection. Biochim. Biophys. Acta (1988) 950:221–228.
  • COIIEN M, LANGLE-ROUAULT F, KIRLAPPOS H et al.: Transferrin-polycation-mediated introduction of DNA into human leukemic cells: stimulation by agents that affect the survival of transfected DNA or modulate transferrin receptor levels. Proc. Natl. Acad. Sci. USA (1990) 87:4033–4037.
  • WAGNER E, COI LEN M, MECHTLER K, KIRLAPPOS H,BIRNSTIEL ML: DNA-binding transferrin conjugates as functional gene-delivery agents: synthesis by linkage of polylysine or ethidium homodimer to the transferrin carbohydrate moiety. Bioconjug. Chem. (1991) 2:226–231.
  • HAENSLER J, SZOKA FC, Jr.: Synthesis and charac-terization of a trigalactosylated bisacridine compound to target DNA to hepatocytes. Bioconjug. Chem. (1993) 4:85–93.
  • NXTU GY, WILSON JM, SHALABY F et al.: Receptor-mediated gene delivery in vivo. Partial correction of genetic analbuminemia in Nagase rats. J. Biol. Chem. (1991) 266:14338–14342.
  • WILSON JM, GROSSMAN M, WU CII et al.: Hepatocyte-di-rected gene transfer in vivo leads to transient improve-ment of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits. J. Biol. Chem. (1992) 267:963–967.
  • STANKOVICS J, CRANE AM, ANDREWS E et al.: Overex-pression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycopro-tein/polylysine/DNA complexes. Hum. Gene Ther. (1994) 5:1095–1104.
  • PERALES JC, FERKOL T, BERGEN H, RATNOFF OD, HAN- •SON RW: Gene transfer in vivo: sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc. Natl. Acad. Sci. USA (1994) 91:4086-4090. A chimeric gene containing the phosphoenolpyruvate carboxy-kinase gene promoter linked to the structural gene for human factor LX was condensed with galactosylated poly(L-lysine). Following injection into rats, delivery was targeted to the liver, and rnRNA and functional protein were detected up to 140 days after administration.
  • PERALES JC, FERKOL T, MOLAS M, HANSON RW: An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. Eur. J. Biochem. (1994) 226:255-266. A review which discusses the critical steps in the preparation of the DNA-ligand complex, and the factors involved in the delivery and regulated expression of a transgene in animal tissues.
  • CURIEL DT, AGARWAL S, WAGNER E, COTTEN M: Adenovirus enhancement of transferrin-polylysine-medi-ated gene delivery. Proc. Natl. Acad. Sci. USA (1991) 88:8850-8854. The first use of AdV particles to augment delivery of transferrin-polylysine conjugates to cells, via endosome disruption, and en-hance gene expression (2000-fold above baseline).
  • WAGNER E, ZATLOUKAL K, CO El EN NI et al.: Coupling of Adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc. Natl. Acad. Sci. USA (1992) 89:6099–6103.
  • CURIEL DT, WAGNER E, COI lEN M et al.: High-efficiency gene transfer mediated by Adenovirus coupled to DNA-polylysine complexes. Hum. Gene Ther. (1992) 3:147–154.
  • CURIEL DT: High-effietency gene transfer employing adenovirus-polylysine-DNA complexes. Nat. Imm. (1994) 13:141–164.
  • COTTEN M, WAGNER E, ZATLOUKAL K et al.: High-effi-ciency receptor-mediated delivery of small and large (48 kilobase) gene constructs using the endosome-dis-ruption activity of defective or chemically inactivated adenovirus particles. Proc. Natl. Acad. Sci. USA (1992) 89:6094–6098•
  • COTFEN M, SALTIK M, KURSA M et al.: Psoralen treatment of Adenovirus particles eliminates virus replication and transcription while maintaining the endosomolytic activity of the virus capsid. Virology (1994) 205:254-261. Describes the chemical inactivation of replication-incompetent AdV to allow deliver of DNA to target cells without interference from AdV gene expression or replication.
  • PRCHLA E, PLANK C, WAGNER E, BLAAS D, FUCHS R: •Virus-mediated release of endosomal content in vitro: different behavior of adenovirus and rhinovirus sero-type 2.J. Cell. Biol. (1995) 131:111-123. The data presented support the model of a specific pore-forming mechanism for human rhinovirus serotype 2 (HRV2) uncoating which is in contrast to the membrane-disrupting mechanism of AdV.
  • CO II LN M, WAGNER E, ZATLOUKAL K, BIRNSTIEL ML: Chicken adenovirus (CELO virus) particles augment receptor-mediated DNA delivery to mammalian cells and yield exceptional levels of stable transformants. J. Virol. (1993) 67:3777-3785. Presents the potential to exploit the endosomolytic activity of non-human viruses, specifically the chicken AdV, which have no natural tropism for human cells. Such viruses can be used to augment receptor-mediated gene delivery by coupling to molecular conju-gates or could be engineered to have a targeted human tropism.
  • MICHAEL SI, HUANG CH, ROMER MU et al.: Binding-in-competent adenovirus facilitates molecular conjugate-mediated gene transfer by the receptor-mediated endocytosis pathway. J. Biol. Chem. (1993) 268:6866–6869.
  • CO'rTEN M, WEBER JM: The adenovirus protease is required for virus entry into host cells. Virology (1995) 213:494–502.
  • MICHAEL SI, HONG JS, CURIEL DT, ENGLER JA: Addition of a short peptide ligand to the adenovirus fiber protein. Gene Ther. (1995) 2:660–668.
  • ZEIGLER ST, KERBY JD, CURIEL DT, DIETHELM AG, THOMPSON JA: Molecular conjugate-mediated gene transfer into isolated human kidneys. Transplantation (1996) 61:812-817. Describes the first genetic transfection of isolated human kidneys under conditions of organ preservation with a reporter gene con-struct using Ad-polylysine-DNA complexes. Gene delivery and ex-pression were localised in proximal tubular epithelial cells. Demonstrates the potential for genetic intervention in chronic rejec-tion.
  • GAO L, WAGNER E, COTTEN M et al.: Direct in vivo gene transfer to airway epithelium employing adenovirus-polylysine-DNA complexes. Hum. Gene Ther. (1993) 4:17–24.
  • ZATLOUKAL K, COHEN M, BERGER M et al.: In vivo production of human factor VII in mice after intras-plenic implantation of primary fibroblasts transfected by receptor-mediated, adenovirus-augmented gene de-livery. Proc. Natl. Acad. Sci. USA (1994) 91:5148–5152.
  • ZATLOUKAL K, SCHNEEBERGER A, BERGER M et al.: Elici-tation of a systemic and protective anti-melanoma im-mune response by an 11-2-based vaccine. Assessment of critical cellular and molecular parameters. J. Immu-nol. (1995) 154:3406–3419.
  • MERTELSMANN R, LINDEMANN A, BOEHM T et al.: Pilot study for the evaluation of T-cell-mediated tumour immunotherapy by cytokine gene transfer in patients with malignant tumours. J. Mol. Med. (1995) 73(4):205–206
  • YANG Y, NUNES FA, BERENCSI K et al.: Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA (1994) 91:4407–4411.
  • PARENTE RA, NADASDI L, SUBBARAO NK, SZOKA FC, Jr : Association of a p11-sensitive peptide with membrane vesicles: role of amino add sequence. Biochemistry (1990) 29:8713–8719.
  • PARENTE RA, NIR S, SZOKA FC, Jr.: Mechanism of leakage of phospholipid vesicle contents induced by the pep-tide GAIA. Biochemistry (1990) 29:8720–8728.
  • WAGNER E, PLANK C, ZATLOUKAL K, COTTEN M, BIRN- •SHEL ML: Influenza virus hernagghninin HA-2 N-termi-nal f-usogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: toward a syn-thetic virus-like gene-transfer vehicle. Proc. Natl. Acad. Sci. USA (1992) 89:7934-7938. The first demonstration that the N-terminal sequence of the influenza virus haemagglutinin subunit HA-2 can enhance receptor-mediated gene transfer.
  • STEINHALTER DA, WHARTON SA, SKEHEL JJ, WILEY DC: •Studies of the membrane fusion activities of fusion peptide mutants of influenza virus hemagglutinin. J. Virol. (1995) 69:6643-6651. A study of the amino acid sequence requirements for a functional fusion peptide by determining the fusion capacities of site-specific mutant HAs and of synthetic peptide analogues.
  • PLANK C, OBERHAUSER B, MECHTLER K, KOCH C, WAG-NER E: The influence of endosome-disruptive peptides on gene transfer using synthetic virus-like gene trans-fer systems. J. Biol. Chem. (1994) 269:12918–12924.
  • FATTAL E, NIR S, PARENTE RA, SZOKA FC, Jr.: Pore-form-ing peptides induce rapid phospholipid flip-flop in membranes. Biochemistry (1994) 33:6721–6731.
  • ZAUNER W, BLAAS D, KUECHLER E, WAGNER E: Rhinovirus-mediated endosomal release of transfection complexes. J. Virol. (1995) 69:1085-1092. Describes the use of human rhinovirus (HRV) serotypes to enhance receptor-mediated gene transfer, and a peptide mimetic derived from the N terminus of VP1 of HRV2. The peptide possesses pH-depend-ent membrane-disrupting activity and enhances gene transfer to cells in vitro.
  • GOTTSCHALK S, TWETEN RK, SMITH LC, WOO SL: Effi-cient gene delivery and expression in mammalian cells using DNA coupled with perfringolysin. Gene Ther. (1995) 2:498–503.
  • LEDLEY FD: Phartnacoltinetic considerations in the use of genes as pharmaceuticals. In: Direct Gene Therapy. Wolff JA (Ed.), Birkhauser, New York (1994).
  • AFIONE SA, CONRAD CK, FLO I IL TR: Gene therapy vectors as drug delivery systems. Clin. Pharrnacokinet. (1995) 28(3):181–189.
  • GORLICH D, VOGEL F, MILLS AD, HARTMANN E, LASKEY RA: Distinct functions for the two importin subunits in nuclear protein import Nature (1995) 377:246–248.
  • GORLICH D, MATTAJ IW: Nucleocytoplasmic transport. Science (1996) 271:1513–1518.
  • ROBBINS J, DILWORTH SM, LASKEY RA, DINGWALL C: Two interdependent basic domains in nucleoplasnain nuclear targeting sequence: identification of a class of bipartite nuclear targeting sequence. Cell (1991) 64:615–623.
  • SIOMI II, DREYFUSS G: A nuclear localization domain in the litiRNP Al protein. J. Cell. Biol. (1995) 129:551–560.
  • KIRCHNER J, CONNOLLY CM, SANDMEYER SB: Require-ment of RNA polymerase III transcription factors for in vitro position-specific integration of a retrovirus-like element. Science (1995) 267:1488–1491.
  • BUSHMAN FD: Tethering human immunodeficiency virus 1 integrase to a DNA site directs integration to nearby sequences. Proc. Natl. Acad. Sci. USA (1994) 91:9233-9237. Demonstrates the use of a hybrid integrase composed of HIV-1 integrase and lambda repressor protein for directed integration, and suggests that such a strategy may be useful for directing integration to specific sequences in vivo.
  • METZGER D, CLIFFORD J, CHIBA H, CHAMBON P: Condi-tional site-specific recombination in mammalian cells using a ligand-dependent chimeric Cre recombinase. Proc. Natl. Acad. Sci. USA (1995) 92:6991–6995.
  • SHELLING AN, SMITH MG: Targeted integration of trans-fected and infected adeno-associated virus vectors con-taining the neomycin resistance gene. Gen. Ther. (1994) 1:165–169.
  • PHILIP R, BRUNE IF E, KILINSK1 L et al.: Efficient and sustained gene expression in primary T lymphocytes and primary and cultured tumor cells mediated by adeno-associated virus plasmid DNA complexed to cat-ionic liposomes. Mol. Cell. Biol. (1994) 14:2411–2418.
  • VIEWEG J, BOCZKOWSKI D, ROBERSON KM et al.: Efficknt gene transfer with adeno-associated virus-based plasnilds complexed to cationic liposomes for gene therapy of human prostate cancer. Cancer Res. (1995) 5585:2366-2372. Describes the use of liposome-DNA complexes containing the AAV inverted terminal repeats to introduce and express the IL-2 gene in a prostatic tumour cell line and in short-term cultures of primary human prostatic tumour cells. Expression levels were 3- to 10-fold higher than liposome complexes containing non-AAV containing plasmids.
  • WU P, DE FIEBRE CM, MILLARD WJ et al.: An AAV pro-moter-driven neuropeptide Y gene delivery system us-ing Sendai virosomes for neuritis and rat brain. Gene Ther. (1996) 3:246–235.
  • HUXLEY C: Mammalian artificial chromosomes: a new tool for gene therapy. Gene Ther. (1994) 1:7–12.
  • FARR CJ, BAYNE RA, KIPLING D et al.: Generation of a human X-derived minichromosome using telomere-as-sociated chromosome fragmentation. EMBO J. (1995) 14:5444–5454.
  • KEMPKES B, PICH D, ZEIDLER R, HAMMERSCHMIDT W:
  • Immortalization of human primary B lymphocytes invitro with DNA. Proc. Natl. Acad. Sci. USA (1995) 92:5875-5879. Describes the generation of a mini-EBV plasmid which encodes all the essential genes of EBV. The mini-EBV plasmid can yield immor-talised B-cells upon transfer of its naked DNA into human primary B-lymphocytes, and cell lines carrying the recombinant vector DNA do not support virus production.
  • COLLIS P, ANTONIOU M, GROSVELD F: Definition of the minimal requirements within the human beta-globin gene and the dominant control region for high level expression. EMBO J. (1990) 9:233–240.
  • DILLON N: Regulating gene expression in gene therapy. Trends Biotechnol. (1993) 11:167–173.
  • WANG Y, O'MALLEY BW, Jr., TSAI SY, O'MALLEY BW: A regulatory system for use in gene transfer. Proc. Natl.Acad. Sci. USA. (1994) 91:8180-8184. Describes the construction of a chimeric regulator comprising the ligand-binding domain of a mutant human progesterone receptor, which binds RU 486 (Mifepristone) but not progesterone, fused to the yeast transcriptional activator GAL4 DNA-binding domain and the HSV protein VP16 activation domain. The chimeric regulator activates target genes containing the GAL4-binding site in response to RU 486 administration.
  • HELLEN CU, WIMMER E: Translation of encephalomyo-carditis virus RNA by internal ribosomal entry. Curr. Top. Micro biol. Immunol. (1995) 203:31–63.
  • SUGIMOTO Y, AKSENTIJEVICH I, MURRAY Gj et al.:
  • •Retroviral co-expression of a multidrug resistance gene (MDR1) and human alpha-galactosidase A for gene therapy of Fabry disease. Hum. Gene Then (1995) 6:905-915. Presents the use of a retroviral vector system that enables co-expres-sion of drug-selectable markers with a second non-selectable gene as part of a bicistronic message using the internal ribosomal entry site from ECMV.
  • SOKOLIC RA, SEKHSARIA S, SUGIMOTO Y et al.: A bids-ironic retrovirus vector containing a picornavirus in-ternal ribosome entry site allows for correction of X-linked CGD by selection for MDR1 expression. Blood (1996) 87:42–50.
  • DENG H, WOLFF JA: Seff-amplifying expression from the T7 promoter in 3T3 mouse fibroblasts. Gene (1994) 143:245–249.
  • HERWEIJER H, LATENDRESSE JS, WILLIAMS P et al.: A plasmid-based self-amplifying Sindbis virus vector. Hum. Gene Ther. (1995) 6:1161-1167. Describes the use of a recombinant cDNA genome of Sindbis virus under the transcriptional control of a Rous sarcoma virus (RSV) LTR promoter as a self-amplifying eukaryotic expression vector. High-level expression of a reporter gene in vitro and in vivo was observed - up to 200 times higher than obtained with a conventional RSV expression vector.
  • JOHANNING FW, CONRY RM, LOBUGLIO AF et al.: A Sindbis virus mRNA polynucleotide vector achieves prolonged and high level heterologous gene expres-sion in vivo. Nucleic Acids Res. (1995) 23:1495–1501.
  • TAPSCOTT SJ, MILLER AD, OLSON JM et al.: Gene therapy of rat 9L gliosarcoma tumors by transduction with selectable genes does not require drug selection. Proc. Natl. Acad. Sci. USA (1994) 91:8185–8189.
  • HILL A, JUGOVIC P, YORK I et al.: Herpes simplex virus turns off the TAP to evade host immunity. Nature (1995) 375:411–415.
  • FRUH K, AHN K, DJABALLAH Fl et al.: A viral inhibitor of peptide transporters for antigen presentation. Nature (1995) 375:415–418.
  • LEVITSKAYA J, CORAM M, LEVTTSKY V et al.: Inhibition of antigen processing by the internal repeat region ofthe Epstein-Barr virus nuclear antigen-1. Nature (1995) 375:685-688. Demonstrates that presentation of protein sequences linked to a Gly-Ala internal repeat of the EBNA-1 protein to the immune system is impaired, hence the potential to mask foreign proteins.
  • CAPLEN NJ, GAO X, HAYES P et al.: Gene therapy for cystic fibrosis in humans by liposome-mediated DNA trans-fer: the production of resources and the regulatory process. Gene Ther. (1994) 1:139–147.
  • SCHORR J, MORITZ P, SEDDON T, SCHLEEF M: Plasmid DNA for human gene therapy and DNA vaccines. Pro-duction and quality assurance. Ann. NY Acad. Sci. (1995) 772:271–273.
  • SMITII KT, SHEPHERD AJ, BOYD JE, LEES GM: Gene delivery systems for use in gene therapy: an overview of quality assurance and safety issues. Gen. Ther. (1996) 3:190–200.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.