4 Pharmaceutical Perspectives of Nonviral Gene Therapy

Advances in Genetics

Volumn 41, Issue C, 1999, Pages 95-156

  Mahato, Ram I ^a   Smith, Louis C ^b   Rolland, Alain ^b  

^a Inc Cleveland ^*  (United States)

^b Valentis Inc The Woodlands   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DNA VACCINE; RECOMBINANT DNA;

ANIMAL; BIOTECHNOLOGY; CHEMISTRY; CLINICAL TRIAL; DRUG DELIVERY SYSTEM; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HUMAN; LEGAL ASPECT; METHODOLOGY; NEOPLASM; PLASMID; REVIEW; UNITED STATES;

ANIMALS; BIOTECHNOLOGY; CLINICAL TRIALS; DNA, RECOMBINANT; DRUG DELIVERY SYSTEMS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; NEOPLASMS; PLASMIDS; UNITED STATES; VACCINES, DNA;

EID: 0032605317     PISSN: 00652660     EISSN: None     Source Type: Book Series    
DOI: 10.1016/S0065-2660(08)60152-2     Document Type: Chapter

References (245)

1. Abdallah B., Hassan A., Benoist C., Goula D., Behr J.P., and Demeneix B.A. A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: Polyethylenimine. Hum. Gene Ther. 7 (1996) 1947-1954
2. Aldjei A.L., and Gupta P.K. "Inhalation on Delivery of Therapeutic Peptides and Proteins" (1997), Marcel Dekker, Inc, New York
3. Alila H., Coleman M., Nitta H., French M., Anwer K., Liu Q.S., Meyer T., Wang J.J., Mumper R., Oubari D., Long S., Nordstrom J., and Rolland A.P. Expression of biologically active human insulin-like growth factor-I following intramuscular injection of a formulated plasmid in rats. Hum. Gene Ther. 8 (1997) 1785-1795
4. Alton E.W., Middteton P.G., Caplen N.J., Smith S.N., Steel D.M., Munkonge F.M., Jeffery P.K., Geddes D.M., Hart S.L., Williamson R., et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nature Genet. 5 (1993) 135-142
5. Anderson R.G.W., Kamen B.A., Rotherberg K.G., and Lacey S.W. Potocytosis: Sequestration and transport of small molecules by caveolae. Science 255 (1992) 410-411
6. Anwer K., Shi M., French M.F., Muller S.R., Chen W., Liu Q., Proctor B.L., Wang J.J., Mumper R.J., Singhal A., Rolland A.P., and Alila H.W. Systemic effect of human growth hormone after intramuscular injection of a single dose of a muscle-specific gene medicine. Hum. Gene Ther. 9 (1998) 659-670
7. Arfors K.-E., Rutili G., and Svensjo E. Microvascular transport of macromolecules in normal and inflammatory conditions. Acta Physiol. Scad. Suppl. 463 (1979) 93-103
8. Baatz J.E., Bruno M.D., Ciraolo P.J., Glasser S.W., Stripp B.R., Smyth K.L., and Korfhagen T.R. Utilization of modified surfactant-associated protein B for delivery of DNA to airway cells in culture. Proc. Natl. Acad. Sci. USA 91 (1994) 2547-2551
9. Barron L.G., Meyer K.B., and Szoka Jr. F.C. Effects of complement depletion on the pharmacokinetics and gene delivery mediated by cationic lipid-DNA complexes. Hum. Gene Ther. 9 (1998) 315-323
10. Baru M., Axelrod J.H., and Nur I. Liposome-encapsulated DNA-mediated gene transfer and synthesis of human factor IX in mice. Gene 161 (1995) 143-150
11. Behr J.P., Demeneix B., Loeffler J.P., and Perez-Mutul J. Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA. Proc. Natl. Acad. Sci. USA 86 (1989) 6982-6986
12. Bennett M.J., Nantz M.H., Balasubramanian R.P., Gruenert D.C., and Malone R.W. Cholesterol enhances cationic liposome-mediated DNA transfection of human respiratory epithelial cells. Bioscience Reports 15 (1995) 47-53
13. Berg P., and Singer M. "Dealing With Genes: The Language of Heredity" (1992), University Science Books, Mill Valley, California
14. Bloomfield V.A. Condensation of DNA by multivalent cations: Considerations on mechanism. Biopolymers 31 (1991) 1471-1481
15. Bloomfield V.A. DNA condensation. Curr. Opin. Struct. Biol. 6 (1996) 334-341
16. Bos J.L. Ras oncogenes in human cancer. A review. Cancer Res. 49 (1989) 4682-4689
17. Bossart J., and Pearson B. Commercialization of gene therapy-The evolution of licensing and rights issues. Trends Biotechnol. 13 (1995) 290-294
18. Brigham K.L., Meyrick B., Christman B., Magnuson M., King G., and Berry Jr. L.C. Rapid communications: In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am. J. Med. Sci. 298 (1989) 278-281
19. Brigham K.L., Meyrick O.B., Christman B., Conary J.T., King G., Berry Jr. L.C., and Magnuson M.A. Expression of human growth hormone fusion genes in cultured lung endothelial cells and in the lungs of mice. Am. J. Respir. Cell Mol. Biol. 8 (1993) 209-213
20. Boussif O., Lezoualch F., Zenta M.A., Mergny M.D., Scherman D., Demeneix B., and Behr J.-P. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethyleneimine. Proc. Natl. Acad. Sri. USA 92 (1995) 7297-7301
21. Brown T.A. "Gene Cloning: An Introduction". (1990), Chapman & Hall, London
22. Buschle M., Cotten M., Kirlappos H., Mechtler K., Schaffner G., Zauner W., Birnstiel M.L., and Wagner E. Receptor-mediated gene transfer into human T lymphocytes via binding of DNA/CD3 antibody particles to the CD3 T cell receptor complex. Hum. Gene Ther. 6 (1995) 753-761
23. Campbell M.J. Lipofection reagents prepared by a simple ethanol injection technique. Biotechniques 18 (1995) 1027-1032
24. Canonico A.E., Conary J.T., Meyrick B.O., and Brigham K.L. Aerosol and intravenous transfection of human α-antitrypsin gene to the lungs of rabbits. Am. J. Respir. Cell Mol. Biol. 10 (1994) 24-29
25. Canonico A.E., Plitman J.D., Conary J.T., Meyrisk B.O., and Brigham K.L. No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes. J. Appl. Physiol. 77 (1994) 415-419
26. Capecchi M.R. High efficiency transfomation by direct microinection of DNA into cultured mammalian cells. Cell 22 (1980) 479-488
27. Caplen N.J., Alton E.W., Middleton P.G., Dorin J.R., Stevenson B.J., Gao X., Durham S.R., Jeffery P.K., Hodson M.E., Coutelle C., et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med. 1 (1995) 39-46
28. Caplen N.J., Gao X., Hayes P., Elaswarapu R., Fisher G., Kinrade E., Chakera A., Schorr J., Hughes B., Dorin J.R., Porteous D.J., Alton E.W., Geddes D.M., Coutelle C., Williamson R., Huang L., and Gilchrist C. Gene therapy for cystic fibrosis in humans by liposome-mediated DNA transfer. The production of resources and the regulatory process. Gene Ther. 1 (1994) 139-147
29. Chelsky D., Ralph R., and Jonak G. Sequence requirements for synthetic peptide-mediated translocation to the nucleus. Mol. Cell Biol 9 (1989) 2487-2492
30. Chen J., Gamou S., Takayanagi A., and Shimizu N. A novel gene delivery system using EGF receptor-mediated endocytosis. FEBS Letters 338 (1994) 167-169
31. Chen J., Sticles R.J., and Daichendt K.A. Galactosylated histone-mediated gene transfer and expression. Hum. Gene Ther. 5 (1994) 429-435
32. Chen X., Li Y., Xiong K., Aizicovici S., Xie Y., Zhu Q., Sturtz F., Shulok J., Snodgrass R., Wagner T.E., and Platika D. Cancer gene therapy by direct tumor injections of a nonviral T7 vector encoding a thymidine kinase gene. Hum. Gene Ther. 9 (1998) 729-736
33. Cheng P.-W. Receptor ligand-facilitated gene transfer: Enhancement of liposome-mediated gene transfer and expression by transferrin. Hum. Gene Ther. 7 (1995) 275-282
34. Cheng P.-W., and Yin W. Lectin conjugate-directed gene transfer to airway epithelial cells. Biochem. Biophys. Res. Commun. 205 (1994) 826-833
35. Cherng J.-Y., Van der Wetering P., Talsma H., Crommelin D.J.A., and Hennik W.E. Effect of size and serum proteins on transfection efficiency of poly((2-dimethylamino)ethyl methacrylate)-plasmid nanoparticles. Pharm. Res. 13 (1996) 1038-1042
36. Chowdhury N.R., Wu C.H., Wu G.Y., Yemeni P.C., Bommineni V.R., and Chowdhury J.R. Fate of DNA targeted to the liver by asialqglycoprotein receptor-mediated endocytosis in vivo: Prolonged persistence in cytoplasmic vesicles after partial hepatotectomy. J. Biol. Chem. 268 (1992) 11265-11271
37. Cohen-Haguenauer O. Overview of regulation of gene therapy in Europe: A current statement including reference to U. S. regulation. Hum. Gene Ther. 6 (1995) 773-785
38. Cohen-Haguenauer O. Safety and regulation at the leading edge of biomedical biotechnology. Curr. Opin. Biotechnol. 7 (1996) 265-272
39. Cohen-Haguenauer O. Gene therapy: Regulatory issues and international approaches to regulation. Curr. Opin. Biotechnol. 8 (1997) 361-369
40. Cole N.B., and Lippincott-Schwartz J. Organization of Organelles and membrane trafficking by microtubules. Curr. Opin. Cell Biol. 7 (1995) 55-64
41. Coleman M.E., De Mayo F., Yin K.C., Lee H.M., Geske R., Montgomery C., and Schwartz R.J. Myogenic vector expression of insulin-like growth factor-I stimulates muscle cell differentiation and myofiber hypertrophy in transgenic mice. J. Biol. Chem. 270 12 (1995) 109-112 116
42. Conary J.T., Parker R.E., Christman B.W., Faulks R.D., King G.A., Meyrick B.O., and Brigham K.L. Protection of rabbit lungs from endotoxin injury by in vivo hyperexpression of the prostaglandin G/H synthase gene. J. Clin. Invest. 93 (1994) 1834-1840
43. Conry R.M., Lo Buglio A.F., Loechel F., Moore S.E., Sumerel L.A., Barlow D.L., Pike J., and Curiel D.T. A carcinoembryonic antigen polynudeotide vaccine for human clinical use. Cancer Gene Ther. 2 (1995) 33-38
44. Curiel D.T., Agrawal S., Wagner E., and Cotten M. Adenovirus enhancement of transferrin-polylysine-mediated gene delivery. Proc. Natl. Acad. Sci. USA 88 (1991) 8850-8854
45. Curiel D.T., Pilewski J.M., and Albelda S.M. Gene therapy approaches for inherited and acquired lung diseases. Am. J. Respir. Cell Mol. Biol. 14 (1996) 1-18
46. Dalgleish A.G. Why gene therapy. Gene Ther. 4 (1997) 629-630
47. Davis H.L. Plasmid DNA expression systems for the purpose of immunization. Curr. Opin. Biotechnol. 8 (1997) 635-640
48. Ding Z.M., Cristiano R.J., Roth J.A., Takacs B., and Kuo M.T. Malarial circumsporozoite protein is a novel gene delivery vehicle to primary hepatocyte cultures and cultured cells. J. Biol. Chem. 270 (1995) 3667-3670
49. Donnelly J.J., Ulmer J.B., and Liu M.A. DNA vaccines. Life Sci. 60 (1997) 163-172
50. Doolan D.L., Sedegah M., Hedstrom R.C., Hobart P., Charoenvit Y., and Hoffman S.L. Circumventing genetic restriction of protection against malaria with multigene DNA immunization CD8+ cells, interferron gamma- and nitric oxide-dependent immunity. J. Exp. Med. 183 (1996) 1739-1746
51. Dowty M.E., Williams P., Zhang G., Hagstrom J.E., and Wolff J.A. Plasmid DNA entry into postmitotic nuclei of primary rat myotubes. Proc. Nad. Acad. Sci. USA 92 (1995) 4572-4576
52. Drlika K. "Understanding DNA and Gene Cloning: A Guide for the Curious", 3rd ed. (1996), Wiley, New York
53. Duguid J.G., Li C., Shi M., Logan M.J., Alila H., Rolland A., Tomlinson E., Sparrow J.T., and Smith L.C. A physicochemical approach for predicting the effectiveness of peptidebased gene delivery systems for use in plasmid-based gene therapy. Biophysical J. 74 (1998) 2802-2814
54. Dunlap D.D., Maggi A., Soria M.R., and Monaco L. Nanoscopic structure of DNA condensed for gene delivery. Nucleic Acids Res. 25 (1997) 3095-3101
55. Eastman S.J., Tousignant J.D., Lukason M.J., Murray H., Siegel C.S., Constatino P., Harris D.J., Cheng S.H., and Scheule R.K. Optimization of formulations and conditions for the aerosol delivery of functional cationic lipid: DNA complexes. Hum. Gene Ther. 8 (1997) 313-322
56. Eck S.L., and Wilson J.M. Gene-based therapy. In Goodman and Gilman's. "The Pharmacological Basis of Therapeutics," 9th ed. (1996), McGraw-Hill, New York 77
57. Ellens H., Bentz J., and Szoka F.C. p H-induced destabilization of phosphatidylethanolamine-containing liposomes: Role of bilayer contact. Biochemistry 23 (1984) 1532-1538
58. Elroy-Stein O., and Moss B. Cytoplasmic expression system based on constitutive synthesis of bacteriophage T7 RNA polymerase in mammalian cells. Proc. Nad. Acad. Sci. USA 87 (1990) 6743-6747
59. Erbacher P., Roche A.C., Monsigny M., and Midoux P. Glycosylated polylysine/DNA complexes: Gene transfer efficiency in relation with the size and the sugar substitution level of glycosylated polylysines and with the plasmid size. Bioconjugate Chem. 6 (1995) 401-410
60. Erbacher P., Bousser M.T., Raimond J., Monsigny M., Midoux P., and Roche A.C. Gene transfer by DNA/glycosylated polylysine complexes into human blood monocyte-derived macrophages. Hum. Gene Ther. 7 (1996) 721-729
61. Farhood H., Serbina N., and Huang L. The role of dioleoylphosphatidylethanolamine in cationic liposome-mediated gene transfer. Biochim. Biophys. Acta 1235 (1995) 289-295
62. Fasbender A., Marshall J., Moninger T.O., Grunst T., Cheng S., and Welsh M.J. Effect of co-lipids in enhancing cationic lipid-mediated gene transfer in vitro and in vivo. Gene Ther. 4 (1997) 716-725
63. Felgner P.L., Gadek T.R., Holm M., Roman R., Chan H.W., Wenz M., Northorp J.P., Ringold G.M., and Danielsen M. Lipofectin: A highly efficient, lipid-mediated DNA transfection procedure. Proc. Natl. Acad. Sci. USA 84 (1987) 7413-7417
64. Felgner J.H., Kumar R., Shridhar C.N., Wheeler C.J., Tsai Y.J., Border R., Ramsey P., Martin M., and Felgner P.L. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. 269 (1994) 2550-2561
65. Ferkol T., Kaetzel C.S., and Davis P.B. Gene transfer into respiratory epithelial cells by targeting the polymeric immunoglobulin receptor. J. Clin. Invest. 92 (1993) 2394-2400
66. Ferkol T., Perales J.C., Mularo F., and Hansen R.W. Receptor-mediated gene transfer into macrophages. Proc. Natl. Acad. Set. USA 93 (1996) 101-105
67. Findeis M.A., Wu C.H., and Wu G.Y. Ligand-based carrier systems for delivery of DNA to hepatocytes. Methods Enzymol. 247 (1994) 341-351
68. Fraley R.T., Subramanii S., Berg P., and Papahadjopoulos D. Introduction of liposome-encapsulated SV40 DNA in cells. J. Biol. Chem. 255 10 (1980) 431-10,435
69. Friend D.S., Papahadjopoulos D., and Debs R.J. Endocytosis and intracellular processing accompanying transfection mediated by cationic liposomes. Biochim. Biophys. Acta 1278 (1996) 41-50
70. Fynan E.F., Webster R.G., Fuller D.H., Haynes J.R., Santoro J.C., and Robinson H.L. DNA vaccines: Protective immunizations by parenteral, mucosal and gene-gun inoculations. Proc. Nad. Acad. Sci. USA 90 (1993) 11,478-11,482
71. Gajewski T.F., Renauld J.-C., Van Pel A., and Boon T. Costimulation with B7-1, IL-6 and IL-12 is sufficient for primary generation of murine antitumor cytotoxic T lymphocytes in vitro. J Immunol. 154 (1995) 5637-5648
72. Gao X.A., and Huang L. A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. 179 (1991) 280-285
73. Gao X., and Huang L. Potentiation of cationic liposome mediated gene delivery by polycations. Biochemistry 35 (1996) 1027-1036
74. Garcia-Bustos J., Heitman J., and Hall M.N. Nuclear protein localization. Biochim. Biophys. Acta 1071 (1991) 83-101
75. Gill D.R., Southern K.W., Mofford K.A., Seddon T., Huang L., Sorgi F., Thomson A., Mac Vinish L.J., Ratcliff R., Bilton D., Lane D.J., Littlewood J.M., Webb A.K., Middleton P.G., College W.H., Cuthbert A.W., Evans M.J., Higgins C.F., and Hyde S.C. A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 4 (1997) 199-209
76. Goodwin E.C., and Rottman F.M. The 3′ flanking sequence of the bovine growth hormone gene contains novel elements required for efficient and accurate polyadenylation. J. Biol. Chem. 267 (1992) 16,330-16,334
77. Gossen M., Freundlieb S., Bender G., Muller G., Hillen W., and Bujard H. Transcriptional activation by tetracyclines in mammalian cells. Science 268 (1995) 1766-1769
78. Gottschalk S., Cristiano R.J., Smith L.C., and Woo S.L. Folate receptor-mediated DNA delivery into tumor cells: Potosomal disruption results in enhanced gene expression. Gene Ther. 1 (1994) 185-191
79. Gottschalk S., Sparrow J.T., Hauer J., Mims M.P., Leland F.E., Woo S.L.C., and Smith L.C. A novel DNA-peptide complex for efficient gene transfer and expression in mammalian cells. Gene Ther. 3 (1996) 48-57
80. Gould-Fogerite S., Mazurkiewics J.E., Raska Jr. K., Voelkerding K., Lehman J.M., and Mannino R.J. Chimerasome-mediated gene transfer in vitro and in vivo. Gene 84 (1989) 429-438
81. Grabbe S., Beissert S., Schwarz T., and Granstein R.D. Dendritic cells as initiators of tumor immune response: A possible strategy for tumor immunotherapy. Immunol. Today 16 (1995) 117-121
82. Gribaudo G., Ravaglia S., Gaboli M., Gariglio M., Cavallo R., and Landolfo S. Interferon-α inhibits the murine cytomegalovirus immediate-early gene expression by down-regulating NF-k B activity. Virology 211 (1995) 251-260
83. Haensler J., and Szoka Jr. F.C. Polyamidoamine cascade polymers mediate efficient transfection of cells in culture. Bioconjugate Chem. 4 (1993) 372-379
84. Hara T., Aramaki Y., Takada S., Koike K., and Tsuchiya S. Receptor-mediated transfer of p SV2CAT DNA to mouse liver cells using asialofetuin-labeled liposomes. Gene Ther. 2 (1995) 784-788
85. Hart S.L., Harbottle R.P., Cooper R., Miller A., Williamson R., and Couttele C. Gene delivery and expression mediated by an integrin-binding peptide. Gene Ther. 2 (1995) 552-554
86. Hartikka J., Sawdey M., Cornefert-Jansen F., Margalith M., Barnhart K., Nolasco M., Vahlsing H.L., Meek J., Marquet M., Hobart P., Norman J., and Manthorpe M. An improved plasmid DNA expression vector for direct injection into skeletal muscle. Hum. Gene Ther. 7 (1996) 1205-1217
87. Hawley-Nelson P., Cicarone V., Gebeyehu G., Jessee J., and Felgner P.L. Lipofectamine reagent: A new, highly efficient polycationic liposome transfection reagent. Focus 15 (1993) 73-79
88. Hayward L.J., and Schwartz R.J. Sequential expression of chicken actin gene during myogenesis. J. Cell Biol. 102 (1986) 1485-1493
89. Hermann F. Clinical application of gene transfer. J. Mol. Med. 74 (1996) 213-221
90. Holter W., Fordis C.M., and Howard B.H. Efficient gene transfer by sequential treatment of mammalian cells with DEAE-dextran and deoxynbonucleic acid. Exp. Cell Res. 184 (1989) 546-551
91. Huang M.T., and Gorman C. Intervening sequences increase efficiency of RNA 3′ processing and accumulation of cytoplasmic RNA. Nucleic Acids Res. 18 (1990) 937-947
92. Huckett B., Ariatti M., and Hawtrey A.O. Evidence for targeted gene transfer by receptor-mediated endocytosis: Stable expression following insulin-directed entry of NEO in Hep G2 cells. Biochem. Pharmacol. 40 (1990) 253-263
93. Hui S.W., Langner M., Zhao Y.-L., Ross P., Hurley E., and Chan K. The role of helper lipids in cationic liposome-mediated gene transfer. Biophys. J. 71 (1996) 590-599
94. Hyde S.C., Gill D.R., Higgins C.F., Trezise A.E., Macvinish L.J., Cuthbert A.W., Ratcliff R., Evans M.J., and Colledge W.H. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 362 (1993) 250-255
95. Jain R.K. Delivery of novel therapeutic agents in tumors: Physiological barriers and strategies. J. Not. Cancer. Inst. 81 (1989) 570-576
96. Jain R.K. Barriers to drug delivery in solid tumors. Sci. Am. 271 (1994) 58-65
97. Johnston S.A., and Tang D.C. Gene gune transfection of animal cells and genetic immunization. Methods Cell Biol. 43 (1994) 353-365
98. Kabanov A.V., Astafeva I.V., Chikindas M.L., Rosfnblat G.F., Kiselev V.I., Severin E.S., and Kabanov V.A. DNA interpolyelectrolyte complexes as a tool for efficient cell transformation. Biopolymers 31 (1991) 1437-1443
99. Kabanov A.V., and Kabanov V.A. DNA complexes with polycations for the delivery of genetic material into cells. Biocon jugate Chem. 6 (1995) 7-20
100. Kakutani T., Yamaoka Y., Hashida M., and Sezaki H. A new method for assessment of drug disposition in muscle: Application of statistical moment theory to local perfusion systems. J. Pharmacokinet. Biopharm. 13 (1985) 609-631
101. Kawabata K., Takakura Y., and Hashida M. The fate of plasmid DNA after intravenous injection in mice: Involvement of scavenger receptors in its hepatic uptake Pharm. Res. 12 (1995) 825-830
102. Kessler D.A., Siegel J.P., Noguchi P.D., Zoon K.C., Feiden K.L., and Woodcock J. Regulation of somaticcell therapy and gene therapy by the Food and Drug Administration. New England J. Med. 329 (1993) 1169-1173
103. Kircheis R., Kichler A., Wallner G., Kursa M., Orgis M., Felzmann T., Buchberger M., and Wagner E. Coupling of cell-binding ligands to polyethylenimine for targeted gene therapy. Gene Ther. 4 (1997) 409-418
104. Kozak M. Structural features in eukaryotic m RNAs that modulate the initiation of translation. J. Biol. Chem. 266 (1991) 19,867-19,870
105. Kozak M. Regulation of translation in eukaryotic systems. Annu. Rev. Cell. Biol. 8 (1992) 197-225
106. Kukowska-Latallo J.F., Bielinska A.U., Johnson J., Spindler R., Tomalia D.A., and Baker Jr. J.R. Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine. Proc. Nad. Acad. Sri. USA 93 (1996) 4897-4902
107. Labat-Moleur F., Steffan A.-M., Brisson C., Perron H., Feugeas O., Furstenberger P., Oberling F., Brambilla E., and Behr J.-P. An electron microscopy study into the mechanism of gene transfer with lipopolyamines. Gene Ther. 3 (1996) 1010-1017
108. Lai W.C., Bennett M., Johnston S.A., Barry M.A., and Pakes S.P. Protection against Mycoplasma pulmonis infection by genetic vaccination. DNA Cell Biol. 14 (1995) 643-651
109. Lanford R.E., and Butel J.S. Construction and characterization of an SV40 mutant defective in nuclear transport of T-antigen. Cell 37 (1984) 801-813
110. Lanier L.L., O'Fallon S., Somoza C., Phillips J.H., Linsely P.S., Okumura K., Ito D., and Azuma M. CD80 (B7) CD86 (B70) provide similar costimulatory signals for T cell proliferation, cytokine production and gene ration of CTL. J. Immunol. 154 (1995) 97-105
111. Lasic D.D., and Templeton N.S. Liposomes in gene therapy. Adv. Drug. Del. Rev. 20 (1996) 221-266
112. Lear J.D., and De Grado W.F. Membrane binding and conformational properties of peptides representing the NH2 terminus of influenza HA-2. J. Biol. Chem. 262 (1987) 6500-6505
113. Ledley F.D. Clinical considerations in the design of protocols for somatic gene therapy. Hum. Gene Ther. 2 (1991) 77-83
114. Ledley F.D. After gene therapy: Issues in long-term clinical follow-up and care. Adv. Genetics 32 (1995) 1-15
115. Ledley F.D. Pharmaceutical approaches to somatic gene therapy. Pharm. Res. 13 (1996) 1595-1614
116. Lee E.R., Marshall J., Siegel C.S., Jiang C., Yew N.S., Nichols M.R., Nietupski J.B., Ziegler R.J., Lane M.B., Wang K.X., Wan N.C., Scheule R.K., Harris D.J., Smith A., and Cheng S.H.E. Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. Hum. Gene Ther. 7 (1996) 1701-1717
117. Lee R.J., and Huang L. Lipidic vector systems for gene transfer. Crit. Rev. Ther. Drug Carrier Syst. 14 (1997) 173-206
118. Legendre J.Y., and Szoka Jr. F.C. Delivery of plasmid DNA into mammalian cell lines using p H-sensitive liposomes: Comparison with cationic liposomes. Pharm. Res. 9 (1992) 1235-1242
119. Lesoon-Wood L.A., Kim W.H., Kleinman H.K., Weintraub B.D., and Mixon A.J. Systemic gene therapy with p53 reduces growth and metastases of a malignant human breast cancer in nude mice. Hum. Gene Ther. 6 (1995) 395-405
120. Lew D., Parker S.E., Latimer T., Abal A.M., Kuwahara-Rundell A., Doh S.G., Yang Z.-Y., Laface D., Gromkowski S.H., Nabel G.J., Manthorpe M., and Norman J. Cancer gene therapy using plasmid DNA: Pharmacokinetic study of DNA following rejection in mice. Hum. Gene Ther. 6 (1995) 553-564
121. Li S., and Huang L. Lipidic supramolecular assemblies for gene transfer. J. Liposome Res. 6 (1996) 589-608
122. Liu Y., Liggitt D., Zhong W., Tu G., Gaensler K., and Debs R. Cationic liposome-mediated intravenous gene delivery. J. Biol. Chem. 270 (1995) 24,864-24,870
123. Liu Y., Mounkes L.C., Liggitt H.D., Brown C.S., Solodin I., Heath T.D., and Debs R.J. Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nature Biotechnol. 15 (1997) 167-173
124. Logan J.J., Bebok Z., Walker L.C., Peng S., Felgner P.L., Siegel G.P., Frizzell R.A., Dong J., Howard Matalon M., et al. Cationic lipids for reporter gene and CFTR transfer to rat pulmonary epithelium. Gene Ther. 2 (1995) 38-49
125. Lowrie D.B., Tascon R.E., Colston M.J., and Silva C.L. Towards a DNA vaccine against tuberculosis. Vaccine 12 (1994) 1537-1540
126. Mahato R.I., Kawabata K., Takakura Y., and Hashida M. In vivo disposition characteristics of plasmid DNA complexed with cationic liposomes. J. Drug Target. 3 (1995) 149-157
127. Mahato R.I., Kawabata K., Nomura T., Takakura Y., and Hashida M. Physicochemical and pharmacokinetic characteristics of plasmid/cationic liposome complexes. J. Pharm. Sci. 84 (1995) 1267-1271
128. Mahato R.I., Takakura Y., and Hashida M. Nonviral vectors for in vivo gene therapy: Physicochemical and pharmacokinetic considerations. Crit. Rev. Ther. Drug Carrier Syst. 14 (1997) 133-172
129. Mahato R.I., Rolland A., and Tomlinson E. Cationic lipid-based gene delivery systems: Pharmaceutical perspectives. Pharm. Res. 14 (1997) 853-859
130. Mahato R.I., Anwer K., Tagliaferri F., Meaney C., Leonard P., French M., Wadhwa M.S., and Rolland A. Biodistribution and gene expression of plasmid/lipid complexes after systemic administration in mice. Hum. Gene Ther. 9 (1998) 2083-2099
131. Mahato R.J., Rolland A., and Tomlinson E. Gene medicines for controllable in vivo production of therapeutic proteins. In: Vincent Lee H.L. (Ed). "Peptide and Protein Drug Delivery," 2nd ed. (1999), Marcel Dekker (in press)
132. R. J. Mannino S. Gould-Fogerite (1996). Cochleate phospholipids in drug delivery. PCT WO96, 25,942
133. Marcel T., and Grausz J.D. The TMC worldwide gene therapy enrollment report, end 1996. Hum. Gene Ther. 8 (1997) 775-800
134. Martin P.A., and Thomas S.M. The commercial development of gene therapy in Europe and the USA. Hum. Gene Ther. 9 (1998) 87-114
135. McLachlan G., Davidson H., Davison D., Dickinson P., Dorin J., and Porteous D. DOTAP as a vehicle for efficient gene delivery in vitro and in vivo. Biochemistry 11 (1994) 19-21
136. McLean J.W., Fox E.A., Baluk P., Bolton P.B., Haskell A., Pearlman R., Thurston G., Umemato E.Y., and McDold D.M. Organ-specific endothelial cell uptake of cationic liposome-DNA complexes in mice. Am. J. Physiol. 273 (1997) H387-H404 (Heart Circ. Physiol. 42)
137. Meyer K.B., Thompson M.M., Levy M.Y., Barron L.G., and Szoka Jr. F.C. Intratracheal gene delivery to the mouse airway: Characterization of plasmid DNA expression and pharmacokinetics. Gene Ther. 2 (1995) 450-460
138. K. B. Meyer L. S. Uyechi F. C. Szoka (1997). Manipulating the intracellular trafficking of nucleic acids. In"Gene Therapy for Diseases of the Lung" (K. L. Brigham), Marcel Dekker, pp 135-180
139. Middleton P.G., Caplen N.J., Gao X., Huang L., Gaya H., Gedes D.M., and Alton E.W. Nasal application of the cationic liposome DC-Chol: DOPE does not alter ion transfer, lung function or bacterial growth. Eur. Respir. J. 7 (1994) 442-445
140. Miller N., and Vile R. Targeted vectors for gene therapy. FASEB J. 9 (1995) 190-199
141. Mumper R.J., Wang J., Claspell J.M., and Rolland A.P. Novel polymeric condensing carriers for gene delivery. Proceed. Intern. Symp. Control. Rel. Bioact. Mater. 22 (1995) 178-179
142. Mumper R.J., Duguid J.G., Anwer K., Barron M.K., Nitta H., and Rolland A.P. Polyvinyl derivatives for controlled gene delivery to muscle. Pharm. Res. 13 (1996) 701-709
143. Mumper R.J., Wang J., Klakamp S.L., Nitta H., Anwer K., Tagliaferri F., and Rolland A.P. Protective interactive noncondensing (PINC) polymers for enhanced plasmid distribution and expression in rat skeletal muscle. J. Control. Ret. 52 (1998) 191-203
144. Nabel E.G., Gordon D., Yang Z.-Y., Xu L., San H., Plautz G.E., Wu B.-Y., Gao X., and Huang Nabel G.J. Gene transfer vitro with DNA-liposome complexes: Lack of autoimmunity and gonadal localization. Hum. Gene. Ther. 3 (1992) 649-656
145. Nabel G.J., Chang A.E., Nabel E.G., Plautz G.E., Ensminger W., Fox B.A., Feigner P., Shu S., and Cho K. Immunotherapy for cancer by direct gene transfer into tumors. Hum. Gene Ther. 5 (1994) 57-77
146. Nakanishi A., Clever J., Yamada M., Li P.P., and Kasamatsu H. Association with capsid proteins promotes nuclear targeting of simian virus40 DNA. Proc. Natl. Acad. Sci. USA 93 (1996) 96-100
147. Nicolau C., Le Pape A., Soriano P., Fargette F., and Juhel M.F. In vivo expression of rat insulin after intravenous administration of the liposome-entrapped gene for rat insulin I. Proc. Natl. Acad. Sci. USA 80 (1983) 1068-1072
148. No D., Yao T.-P., and Evans R.M. Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc. Natl. Acad. Sci. USA 93 (1996) 3346-3351
149. Nomura T., Nakajima S., Kawabata K., Yamashita F., Takakura Y., and Hashida M. Intratumoral pharmacokinetics and in vivo gene expression of naked plasmid DNA and its cationic liposome complexes after direct gene transfer. Cancer Res. 57 (1997) 2681-2686
150. Osaka G., Carey K., Cuthbertson A., Godowski P., Patapoff T., Ryan A., Godek T., and Mordent J. Pharmacokinetics, tissue distribution and expression efficiency of plasmid [33P]DNA following intravenous administration of DNA/cationic lipid complexes in mice: Use of a novel radionucleotide approach. J. Pharm. Sci. 85 (1996) 612-618
151. Parmiani G., Colombo M.P., Melani C., and Arienti F. Cytokine gene transduction in the immunotherapy of cancer. Adv. Pharmacol. 40 (1997) 259-307
152. Perales J.C., Ferkol T., Beegan H., Ratnoff O.D., and Hanson R.W. Gene transfer in vivo: Sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc. Natl. Acad. Sci. USA 91 (1994) 4086-4090
153. Perales J.C., Ferkol T., Molas M., and Hanson R.W. An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. Eur. J. Biochem. 226 (1994) 255-266
154. Persidis A., and Tomczyk M.S. Critical issues in gene therapy commercialization. Nature Biotechnol. 15 (1997) 689-690
155. Petropoulos C.J., Rosenberg M.P., Jenkins N.A., Copeland N.G., and Hughes S.H. The chicken skeletal muscle α-actin promoter is tissue-specific in transgenic mice. Mol. Cell Biol. 9 (1989) 3785-3792
156. Plank C., Oberhauser B., Mechtler K., Koch C., and Wagner E. The influence of endosome-destructive peptides on gene transfer using synthetic virus-like gene transfer systems. J. Biol. Chem. 269 (1994) 12,918-12,924
157. Plank C., Mechtler K., Szoka Jr. F.C., and Wagner E. Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery. Hum. Gene Ther. 7 (1996) 1437-1446
158. Plautz G.E., Yang Z.Y., Wu B.Y., Gao X., and Huang Nabel G.J. Immunotherapy of malignancy by in vivo gene transfer into tumors. Proc. Nad. Acad. Sci. USA 90 (1993) 4645-4649
159. Plautz G.E., Nabel E.G., Fox B., Yang Z.Y., Jaffe M., Gordon D., Chang A., and Nabel G.J. Direct gene transfer for the understanding and treatment of human disease. Ann. N. Y. Acad. Sci. 716 (1994) 144-153
160. Porteous D.J., Dorin J.R., McLachlan G., Davidson-Smith H., Davidson H., Stevenson B.J., Carothers A.D., Wallace W.H.C., Moralee S., Hoenes C., Kallmeyer G., Michaelis U., Naujkos K., Ho L.-P., Samways J.M., Imrie M., Greening A.P., and Innes J.A. Evidence for safety and efficacy of DOTAP cationic liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 4 (1997) 210-218
161. Qin L., Ding Y., Pahud D.R., Chang E., Imperiale M.J., and Bromberg J.S. Promoter attenuation in gene therapy: Interferon-γ and tumor necrosis factor-α inhibit transgene expression. Hum. Gene Ther. 8 (1997) 2019-2029
162. Rabinovich N.R., Mclnnes P., Klein D.L., and Hall B.F. Vaccine technologies: View to the future. Science 265 (1994) 1401-1404
163. Rafalski M., Ortiz A., Rockwell A., van Ginkel L.C., Lear J.D., De Grado W.F., and Wilschut J. Membrane fusion activity of the influenza virus hemagglutinin interaction of HA2 N-terminal peptides with phospholipid vesicles. Biochemistry 30 (1991) 10,211-10,220
164. Remy J.S., Sirlin C., Vierling P., and Behr J.P. Gene transfer with a series of lipophilic DNA-binding molecules. Bioconjugate Chem. 5 (1994) 647-654
165. Remy J.S., Kichler A., Mordvinov V., Schuber F., and Behr J.P. Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: A statue toward artificial viruses. Proc. Natl. Acad. Sci. USA 92 (1995) 1744-1748
166. Richardson S., Kolbe H.V.J., and Duncan R. Evaluation of highly purified chitosan as a potential gene delivery vector. Proc. Int. Symp. Control. Rel. Broact. Mater. 24 (1997) 649-650
167. Rolland A. Controlling gene therapy: Recent advances in non-viral gene delivery. In: Gregoriadis, and McCormack (Eds). "Targeting of Drugs Vol. 5: Strategies for Oligonucleotide and Gene Delivery in Therapy" (1996), Plenum Press, New York 79-95
168. Rolland A., and Felgner P. Preface: Non-viral gene delivery systems. Adv. Drug Del. Rev. 30 (1998) 1-3
169. Ross G.F., Morris R.E., Ciraolo G., Huelsman K., Bruno M., Whitsett J.A., Baatz J.E., and Korfthagen T.R. Surfactant protein A-polylysine conjugates for delivery of DNA to airway cells in culture. Hum. Gene Ther. 6 (1995) 31-40
170. Ross G., Erickson R., Knorr D., Motulsky A.G., Parkman R., Samulski J., Straus S.E., and Smith B.R. Gene therapy in the United States: A five-year status report. Hum. Gene Ther. 7 (1996) 1781-1790
171. Roth J.A., Nguyen D., Lawrence D.D., Kemp B.L., Carrasco C.H., Ferson D.Z., Hong W.K., Komaki R., Lee J.J., Nesbitt J.C., Pisters K.M.W., Putnam J.B., Schea R., et al. Retrovirus-mediated wild-type p53 gene transfer to tumors of patients with lung cancer. Nat. Med. 2 (1996) 985-991
172. Rothberg K.G., Ying Y., Kolhouse J.F., Kamen B.A., and Anderson R.G.W. The glycophospholipid-linked folate receptor internalizes folate without entering the clathrin-coated pit endocytic pathway. Cell Biol. 110 (1990) 637-649
173. Ryu W.S., and Mertz J.E. Simian virus 40 late transcripts lacking excisable intervening sequences are defective in both stability in the nucleus and transport to the cytoplasm. J. Virol. 63 (1989) 4386-4394
174. San H., Yang Z.-Y., Pompili V.J., Jaffe M.L., Plautz G.E., Xu L., Feigner J.H., Wheeler C.J., Feigner P.L., Gao X., Huang L., Gordon D., Nabel G.J., and Nabel E.G. Safety and short-term toxicity of a novel cationic lipid formulation for human gene therapy. Hum. Gene Ther. 4 (1993) 781-788
175. Scheule R.K., and Cheng S.H. Gene transfer into mammalian cells using synthetic cationic lipids. In: Feigner P.L., Heller M.J., Lehr P., Behr J.P., and Szoka Jr. F.C. (Eds). "Artificial Self-Assembling Systems for Gene Delivery" (1996), ACS Books, Washington 177-190
176. Schneider H., Huse K., Birkenmeier G., Otto A., and Scholz G.H. Gene transfer mediated by "α2-macroglobulin. Nucleic Acids Res. 24 (1996) 3873-3874
177. Schwarz L.A., Johnson J.L., Black M., Cheng S.H., Hogan M.E., and Waldrep J.C. Delivery of DNA-cationic liposome complexes by small-particle aerosol. Hum. Gene Ther. 7 (1996) 731-741
178. Schwarzenberger P., Spence S.E., Gooya J.M., Michiel D., Curiel D.T., Ruscetti F.W., and Keller J.R. Targeted gene transfer to human hematopoietic progenitor cell lines through the c-kit receptor. Blood 87 (1996) 472-478
179. Sharp K.A., and Honig B. Salt effects on nucleic acids. Curr. Opin. Struct. Biol. 5 (1995) 323-328
180. Shaw P., Bovey R., Tardy S., Sahli R., Sordat B., and Costa J. Induction of apoptosis by wild-type p53 in a human colon tumor-derived cell line. Proc. Natl. Acad. Sci. USA 89 (1992) 4495-4499
181. Sikora K. Scope and limitatons of gene therapy. In: Lemoine N.R., and Cooper D.N. (Eds). "Gene Therapy" (1996), Bios Scientific Publishers, Oxford, UK 1-10
182. Simionescu N. Cellular aspects of transcapillary exchange. Physiol. Rev. 63 (1983) 1536-1579
183. Smith L.C., Duguid J., Wadhwa M.S., Logan M.J., Tung C.-H., Edwards V., and Sparrow J.T. Synthetic peptide-based DNA complexes for nonviral gene delivery. Adv. Drug. Del. Rev. 38 (1998) 115-131
184. Soriano P., Dijkstra J., Legrand A., Spanjer H., Londos-Gagliard D., Roerdink F., Scherphof G., and Nicolau C. Targeted and non-targeted liposomes for in vivo transfer to rat liver cells of a plasmid containing the preproinsulin I gene. Proc. Natl. Acad. Sci. USA 80 (1983) 7128-7131
185. Sorscher E.J., Logan J.J., Frizzell R.A., Lyrene R.K., Bebok Z., Dong J.Y., Duvall M.D., Felgner P.L., Matalon S., Walker L., and Wiatrak B.R. Gene therapy for cystic fibrosis using cationic liposomes mediated gene transfer. A Phase I trial of safety and efficacy in the nasal airway. Hum. Gene Ther. 5 (1994) 1259-1277
186. Spooner R.A., Deonarian M.P., and Epenetos A.A. DNA vaccination for cancer treatment. Gene Ther. 2 (1995) 173-180
187. Stankovics J., Crane A.M., Andrews E., Wu C.H., Wu G.Y., and Ledley F.D. Overexpression of human methylmalonyl Co A mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine/DNA complexes. Hum. Gene Ther. 5 (1994) 1095-1104
188. Stein J., Volk H.D., Liebenthal C., Kruger D.H., and Prosch S. Tumor necrosis factor α stimulates the activity of the human cytomegalovirus major immediate early enhancer/promoter in immature monocytic cells. J. Gen. Virol. 74 (1993) 2333-2338
189. Sternberg B., Sorgi F.L., and Huang L. New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron microscopy. FEBS Lett. 356 (1994) 361-366
190. Stewart M.J., Plautz G.E., Del Buono L., Yang Z.-Y., Xu L., Gao X., Huang L., Nabel E.G., and Nabel G.J. Gene transfer in vivo with DNA-liposome complexes: Safety and acute toxicity in mice. Hum. Gene Ther. 3 (1992) 267-275
191. Stribling R., Brunette E., Liggitt D., Gannsler K., and Debs R. Aerosol gene delivery in vivo. Proc. Natl. Acad. Sci. USA 89 (1992) 11,277-11,281
192. Takai T., and Ohmori H. DNA transfection of mouse lymphoid cells by the combination of DEAE-dextran-mediated DNA uptake and osmotic shock procedure. Biochim. Biophys. Acta. 1048 (1990) 105-109
193. Takakura Y., and Hashida M. Macromolecular drug carrier systems in cancer chemotherapy: Macromolecular prodrugs. Crit. Rev. Oncol. Hematol. 18 (1995) 207-231
194. Tang D.-C., Shi Z., and Curiel D.T. Vaccination onto bare skin. Nature 388 (1997) 729-730
195. Tang M.X., and Szoka F.C. The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther. 4 (1997) 823-832
196. Tang M.X., and Szoka Jr. F.C. Biophysical and colloidal aspects of DNA complexation. In: Kabanov A.V., Seymour L.W., and Felgner P.L. (Eds). "Self-Assembling Complexes for Gene Delivery: From Chemistry to Clinical Trial" (1997), Wiley, New York 2-67
197. Tatsumi T., Takehara T., Katayama K., Mochizuki K., Yamamoto M., Kanto T., Sasaki Y., Kasahara A., and Hayashi N. Expression of costimulatory molecules B7-1(CD80) and B7-2 (CD86) on human hepatocellular carcinoma. Hepatology 25 (1997) 1108-1114
198. Taylor A., and Granger D.N. Exchange of macromolecules across the microcirculation. In: Renkin E.M., and Michael C.C. (Eds). "Handbook of Physiology, Section 2: The Cardiovascular System" (1984), American Physiology Society, Bethesda, Maryland 492-494
199. Templeton N.S., Lasic D.D., Frederik P.M., Strey H.H., Roberts D.D., and Pavlakis G.N. Improved DNA:liposome complexes for increased systemic delivery and gene expression. Nature Biotechnol. 15 (1997) 647-652
200. Tepper R.I., and Mule J.J. Experimental and clinical studies of cytokine gene-modified tumor cells. Hum. Gene Ther. 5 (1994) 153-164
201. Thurnher M., Wagner E., Clausen H., Mechtler K., Rusconi S., Dinter A., Birnstiel M.L., Berger E.G., and Cotten M. Carbohydrate receptor-mediated gene transfer to human T leukaemic cells. Glycobiology 4 (1994) 429-435
202. Tikchonenko T.I., Glushakova S.E., Kislina O.S., Grodnitskaya N.A., Manykin A.A., and Naroditsky B.S. Transfer of condensed viral DNA into eukaryotic cells using proteogliposomes. Gene 83 (1988) 321-330
203. Tomalia D.A., Naylor A.M., and Goddard W.A. Starburst cascade polymers: Molecular level control of size, shape, surface chemistry, topology and flexibility from atoms to macroscopic matter. Angew. Chem. Int. Ed. Engl. 29 (1990) 138-175
204. Tomlinson E. Theory and practice of site-specific drug delivery. Adv. Drug Del. Rev. 1 (1987) 87-198
205. Tomlinson E. Biological dispersion and the design of site-specific protein therapeutic systems. In: Gregoriadis G., Allison A.C., and Poste G. (Eds). "Targeting of Drugs 2" (1990), Plenum Press, New York 1-20
206. Tomlinson E. Impact of the new biologies on the medical and pharmaceutical sciences. J. Pharm. Pharmacol. 44 suppl. 1 (1992) 147-159
207. Tomlinson E., and Rolland A. Controllable gene therapy: Pharmaceutics of non-viral gene delivery systems. J. Control. Rel. 39 (1996) 357-372
208. Townsend S.E., and Allison J.P. Tumor rejection after direct co-stimulation of CD8+ T cells by B7-transfected melanoma cells. Science 259 (1993) 368-370
209. Tsan M.-F., White J.E., and Shepard B. Lung-specific direct in vivo gene transfer with recombinant plasmid DNA. Am. J. Physiol. 268 (1995) L1052-L1056
210. Tzen C.-Y., and Scott R.E. Induction of SV40 early transcription by type I interferon. Exp. Cell Res. 205 (1993) 246-250
211. Vega M.A. Gene Targeting (1995), CRC Press, Boca Raton, Florida 212
212. Venkatachalam M.A., and Rennke H.G. The structural and molecular basis of glomerular filtration. Circ. Res. 43 (1978) 337-347
213. Vieira J., and Messing J. The p UC plasmids, an M13 mp7-derived system from insertion mutagenesis and sequencing with synthetic universal primers. Gene 19 (1982) 259-268
214. Wadhwa M.S., Collard W.T., Adami R.C., McKenzie D.L., and Rice K.G. Peptidebased gene delivery: Influence of peptide structure on gene expression. Bioconj. Chem. 8 (1997) 81-88
215. Wagner E., Zenke M., Cotten M., Beug H., and Birnstiel M.L. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. USA. 87 (1990) 3410-3414
216. Wagner E., Plank C., Zatloukal K., Cotten M., and Birnstiel M.L. Influenza virus hemagglutinin HA-2N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: Towards a synthetic virus-like gene transfer vehicle. Proc. Natl. Acad. Sci. USA 89 (1992) 7934-7938
217. Wang C.Y., and Huang L. Plasmid DNA adsorbed to p H-sensitive liposomes efficiently transforms the target cells. Biochem. Biophys. Res. Commun. 147 (1987) 980-985
218. Wang C.Y., and Huang L. p H-sensitive immunoliposomes mediate target-cell-specific delivery and controlled expression of a foreign gene in mouse. Proc. Natl. Acad. Sci. USA 84 (1987) 7851-7855
219. Wang Y., O'Malley Jr. B.W., Tsai S.Y., and O'Malley B.W. A regulatory system for use in gene transfer. Proc. Natl. Acad. Sci. USA 91 (1994) 8180-8184
220. Wang Y., O'Malley B.W., and Tsai S.Y. Inducible system designed for future gene therapy. Methods. Mol. Biol. 69 (1997) 401-413
221. Wang Y., De Mayo F.J., Tsai S.Y., and O'Malley B.W. Ligand-inducible and liver-specific target gene expression in transgenic mice. Nature Biotechnol. 15 (1997) 239-243
222. Whartenby K.A., Abboud C.N., Marrogi A.J., Ramesh R., and Freeman S.M. The biology of cancer gene therapy. Lab. Invest. 72 (1995) 131-145
223. White J.M. Membrane fusion. Science 258 (1992) 917-924
224. Wilson J.M., Grossman M., Wu C.H., Chowdhury N.R., Wu G.Y., and Chowdhury J.R. Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesteromia in low density lipoprotein receptor-deficient rabbits. J. Biol. Chem. 267 (1992) 963-967
225. Wilson R.W., and Bloomfield V.A. Counterion-induced condensation of deoxyribonucleic acid, a light scattering study. Biochemistry 18 (1979) 2192-2196
226. Wimley W.C., and Thompson T.E. Phosphatidylethanolamine enhances the concentration-dependent exchange of phospholipids between bilayers. Biochemistry 30 (1991) 4200-4204
227. Wolfert M.A., Schacht E.H., Toncheva V., Ulbnch K., Nazarova O., and Seymour L.W. Characterization of vectors for gene therapy formed by self-assembly of DNA with synthetic block co-polymers. Hum. Gene Ther. 7 (1996) 2123-2133
228. Wolff J.A., Malone R.W., Williams P., Chong W., Acsadi G., Jani A., and Felgner P.L. Direct gene transfer into mouse muscle in vivo. Science 247 (1990) 1465-1468
229. Wolff J.A., Ludtke J.J., Acsadi G., Williams P., and Jani A. Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1 (1992) 363-369
230. Woodley J.F. Enzymatic barriers for GI peptide and protein delivery. Crit. Rev. Ther. Drug Carrier Syst. 11 (1994) 61-95
231. Wu C.H., Wilson J.M., and Wu G.Y. Targeting of genes: Delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J. Biol. Chem. 264 (1989) 16,985-16,987
232. Wu G.Y., and Wu C.H. Receptor-mediated gene delivery and expression in vivo. J. Biol. Chem. 263 (1988) 14,621-14,624
233. Wu G.Y., Wilson J.M., Shalaby F., Grossman M., Shafritz D.A., and Wu C.H. Receptor-mediated gene delivery in vivo: Partial correlation of genetic analbuminemia in nagase rats. J. Biol. Chem. 266 (1991) 14,338-14,342
234. Xu M., Kumar D., Shrinivas S., Detolla L.J., Yu S.F., Stass S.A., and Mixon A.J. Parenteral gene therapy with p53 inhibits breast tumors in vivo through a bystander mechanism without evidence of toxicity. Hum. Gene Ther. 8 (1997) 177-185
235. Xu Y., and Szoka Jr. F.C. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry 35 (1996) 5616-5623
236. Yang W., Waine G.J., and McManus D.P. Antibodies to Schistosoma japonicum (Asian bloodfluke) paramyosin induced by nucleic acid vaccination. Biochem. Biophys. Res. Commun. III (1995) 1029-1039
237. Yew N.S., Wysokenski D.M., Wang K.X., Ziegler R.J., Marshall J., McNeilly D., Cherry M., Osburn W., and Cheng S.H. Optimization of plasmid vectors for high-level expression in lung endothelial cells. Hum. Gene Ther. 8 (1997) 575-584
238. Yokoyama M., Zhang J., and Whitton J.L. DNA immunization confers protection against lethal lymphatic choriomeningitis virus infection. J. Virol. 69 (1995) 2684-2688
239. Yoshida M., Mahato R.I., Kawabata K., Takakura Y., and Hashida M. Disposition characteristics of plasmid DNA in the single-pass rat liver perfusion system. Pharm. Res. 13 (1996) 599-603
240. Yoshimura K., Rosenfeld M.A., Nakamura H., Scherer E.M., Pavirani A., Lecocq J.P., and Crystal R.G. Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer. Nucleic Acids Res. 20 (1992) 3233-3240
241. Zabner J., Fasbender A.J., Moninger T., Poellinger K.A., and Welsh M.J. Cellular to molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. 270 (1995) 18,997-19,007
242. Zalipsky S., Brandeis E., Newman M.S., and Woodle M.C. Long circulating, cationic liposomes containing amino-PEG-phosphatidylethanolamine. FEBS Letters 353 (1994) 71-74
243. Zelphati O., Uyechi L.S., Barron L.G., and Szoka Jr. F.C. Effect of serum components on the physico-chemical properties of cationic lipid/oligonucleotide complexes and on their interactions with cells. Biochim. Biophys. Acta 1390 (1998) 119-133
244. Zhou X., and Huang L. DNA transfection mediated by cationic liposomes containing lipopolylysines: Characterization and mechanism of action. Biochim. Biophys. Acta 1189 (1994) 195-203
245. Zhu N., Liggitt D., Liu Y., and Debs R. Systemic gene expression after intravenous DNA delivery into adult mice. Science 261 (1993) 209-211