From genes to gene medicines: Recent advances in nonviral gene delivery

Critical Reviews in Therapeutic Drug Carrier Systems

Volumn 15, Issue 2, 1998, Pages 143-198

  Rolland, Alain P ^a  

^a GeneMedicine Inc   (United States)

Author keywords

Cationic lipids; Endosomes; Gene therapy; Liposomes; Nonviral gene delivery; Peptides; Plasmid; Polymers; Receptor mediated endocytosis; Targeting; Viral vectors

Indexed keywords

VIRUS VECTOR;

GENE TARGETING; GENE THERAPY; HUMAN; MEDICAL RESEARCH; PLASMID; REVIEW;

EID: 0031970246     PISSN: 07434863     EISSN: None     Source Type: Journal    
DOI: 10.1615/CritRevTherDrugCarrierSyst.v15.i2.20     Document Type: Review

References (294)

1. Anderson, W. F., Human Gene Therapy, Science, 256, 808, 1992.
2. Ledley, F. D., Are contempory methods for somatic gene therapy suitable for clinical applications?, Clin. Invest. Med., 16, 78, 1993.
3. Ledley, F. D., Hepatic gene therapy: Present and future, Hepatology, 18, 1263, 1993.
4. Ledley, F. D., Development in somatic gene therapy, Exp. Op. Invest. Drugs, 3, 913, 1994.
5. Ledley, F. D., Non-viral gene therapies, Current Opinion in Biotechnology, 5, 626, 1994.
6. Anderson, W. F., Gene Therapy, Scientific American, 273, 124, 1995.
7. Hodgson, C. P., The vector void in gene therapy, Biotechnology, 13, 22, 1995.
8. Miller, A. D., Human gene therapy comes of age, Nature, 357, 455, 1992.
9. Kay, M. A., Rothenberg, S., Landen, C. N., Bellinger, D.A., Leland, F.,Toman, C., Finegold, M., Thompson, A. R., Read, M. S., Brinkhous, K. M., and Woo, S. L. C., In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs, Science, 262, 117, 1993.
10. Culver, K. W. and Blaese, R. M., Gene therapy for adenosine deaminase deficiency and malignant solid tumors, in Gene Therapeutics: Methods and Applications of Direct Gene Transfer, Wolff, J. A., Ed., Boston: Birkhauser, 1994, 263.
11. Glorioso, M.J. C., DeLuca, N. A., Goins, W. F., and Fink, D. J., Development of herpes simplex virus vectors for gene transfer to the central nervous system, in Gene Therapeutics: Methods and Applications of Direct Gene Transfer, Wolff,J. A., Ed., Boston: Birkhauser, 1994, 281.
12. Ledley, F. D., Nonviral gene therapy: The promise of genes as pharmaceutical products, Hum. Gene Ther., 6, 1129, 1995.
13. Knowles,M.R.,Hohneker,K.W.,Zhou,Z., Olsen,J. C.,Noah,T. L.,Hu,P.-C.,Leigh, M.W., Engelhardt,J.F., Edwards, L. J.,Jones, K. R., Grossman, M., Wilson J. M.Johnson, L. G., and Boucher, R. C., A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis, N. Eng. J. Med., 333, 823, 1995.
14. Mendell, J. R., Kissel,J. T., Amato, A. A., King, W., Signore, L., Prior, T. W., Sahenk, Z., Benson, S., McAndrew, P. E., Rice, R., Nagaraja, H., Stephens, R., Lantry, L., Morris, G. E., and Burghes, A. H. M., Myoblast transfer in the treatment of Duchenne's muscular dystrophy, N. Eng. J. Med., 333, 832, 1995.
15. Rolland, A. and Tomlinson, E., Controllable gene therapy using non-viral systems,in Gene Therapy and Artificial Self-Assembling Aystems for Gene Transfer, Felgner, P, Heller, M., Lehn, P., Behr,J.-P., and Szoka, F. C.,Jr., Eds., Washington, DC: ACS Books, 1996, 86.
16. Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher,T., Clerici, M., Shearer, G., Chang, L., Chiang, Y., Tolstoshev, P., GreenblatT, J. J., Rosenberg, S. A., Klein, H., Berger, M., Mullen, C. A., Ramsey, W. J., Muul, L., Morgan, R. A., and Anderson, W. F., T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years, Science, 270, 475, 1995.
17. Grossman,M.,Raper,S.E.,Kozarsky,K.,Stein,E.A.,Engelhardt,J.F.,Muller,D., Lupien, P. J., and Wilson,J. M., Successful ex vivo gene therapy directed to the liver in a patient with familial hypercholesterolemia, Nat. Genet., 6, 335, 1994.
18. Grossman, M., Rader, D.J., Muller, D. W. M., Kolansky, D. M., Kozarsky, K., Clark, B. J., Stein, E. A., Lupien, P. J., Brewer, H. B., Raper, S. E., and Wilson,J. M., A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolemia, Nature Medicine, 1, 1148, 1995.
19. Marshall, E., Gene therapy's growing pains, Science, 269, 1050, 1995.
20. Williams, R. S., Human gene therapy of tortoises and hares, Nature Medicine, 1, 1137, 1995.
21. Nolta, J. A., Crooks, G. M., Overell, R. W., Williams, D. E., and Kohn, D. B., Retroviral vector-mediated gene transfer into primitive human hematopoietic progenitor cells: Effects of mast cell growth factor (MGF) combined with other cytokines, Experimental Hematology, 20, 1065, 1992.
22. Xu,L.,Stahl,S.K.,Dave,H.P.,Schiffmann,R.,Correll,P.H.,Kessler,S., andKarlsson, S., Correction of the enzyme deficiency in hematopoietic cells of Gaucher patients using a clinically acceptable retroviral supernatant transduction protocol, Experimental Hematology, 22, 223, 1994.
23. Ohashi, T., Boggs, S., Robbins, P., Bahnson, A., Patrene, K., Wei, F.-S., Wei,J.-F., Li, J., Lucht, L., Fei, Y., Clark, S., Kimak, M., He, H., Mowery-Rushton, P., and Barranger, J. A.,Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector, Proc. Natl. Acad. Sci. USA, 89, 11332, 1992.
24. Yu, M., Leavitt, M. C., Maruyama, M., Yamada, O., Young, D., Ho, A. D., and Wong-Staal, F., Intracellular immunization of human fetal cord blood stem/progenitor cells with a ribozyme against human immunodeficiency virus type 1, Proc. Natl. Acad. Sci. USA, 92, 699, 1995.
25. Riddell, S. R.,Greenberg, P. D.,Overell,R.W.,Loughran,T. P.,Gilbert, M.J., Lupton, S. D., Agosti,J., Scheeler, S., Coombs, R. W., and Corey, L., Phase I study of cellular adoptive immunotherapy using genetically modified CD8+ HIV-specific T cells for HIV seropositive patients undergoing allogeneic bone marrow transplant, Hum. Gene Ther., 3, 319, 1992.
26. O'Shaughnessy, J. A., Cowan, K. H., Nienhuis, A. W., McDonagh, K. T., Sorrentino, B. P., Dunbar, C. E., Chiang, Y., Wilson, W., Goldspiel, B., Kohler, D., Cottier-Fox, M., Leitman,S.,Gottesman,M.,Pastan,I.,Denicoff,A.,Noone,M.,andGress,R.,Retroviral mediated transfer of the human multidrug resistance gene (MDR-1 ) into hematopoietic stem cells during autologous transplantation after intensive chemotherapy for metastatic breast cancer, Hum. Gene Ther., 5, 891, 1994.
27. Ward, M., Richardson, C., Pioli, P., Smith L., Podda, S., Goff, S., Hesdorffer, C., and Bank, A., Transfer and expression of the human multiple drug resistance gene in human CD34+ cells, Blood 84, 1408, 1994.
28. Boesen,J.J.,Nooter,K., and Valerio,D., Circumvention of chemotherapy-induced myelo-suppression by transfer of the mdr1 gene, Biotherapy, 6,291, 1993.
29. Deisseroth, A. B., Kavanagh,J., and Champlin, R., Use of safety-modified retroviruses to introduce chemotherapy resistance sequences into normal hematopoietic cells for chemoprotection during the therapy of ovarian cancer: A pilot trial, Hum. Gene Ther., 5, 1507, 1994.
30. Brenner, M. K., Gene transfer into human hematopoietic progenitor cells: A review of current clinical protocols, J. Hem., 2, 7, 1993.
31. Rosefeld, M. A.,Yoshimura, K., Trapnell, B.,Yoneyama, K., Rosenthal, E.,Dalemans, W.,Fukayama, M., StierJ., Stratford-Perricaudet, L. D., Perricaudet, M., Guggino, W., Pavirani, A.,Lecocq, P-P., and Crystal, R. G., In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium, Cell, 68, 143, 1992.
32. Latchman, D. S., Herpes simplex virus vectors for gene therapy, Mol-Biotechnol., 2, 179, 1994.
33. Blaese,M., Blankenstein,T., Brenner, M.,Cohen-Haguenauer, O., Gansbacher,B.,Russel, S., Sorentino, B., and Velu, T., Vectors in cancer therapy: How will they deliver?, Cancer Gene Ther., 2, 291, 1995.
34. Schnierle, B. S., and Groner, B., Retroviral targeted delivery, Gene Ther., 3, 1069, 1996.
35. Miller, A. D., Retrovirus packaging cells, Hum. Gene Ther., 1, 5, 1990.
36. Rother, R. P., Squinto, S. P., Mason,J.M.,and Rollins, S. A., Protection of retroviral vector particles in human blood through complement inhibition, Hum. Gene Ther., 6, 429, 1995.
37. Rother, R. P., Fodor, W. L., Springhorn, J. P., Birks, C. W., Setter, E., Sandrin, M. S., Squinto, S. P., and Rollins, S. A., A novel mechanism of retro virus inactivation in human serum mediated by anti-alpha-galactosyl natural antibody, J. Exp. Med., 182, 1345, 1995.
38. Temin, H. M., Safety considerations in somatic gene therapy of human disease with retrovirus vectors, Hum. Gene Ther., 1, 111, 1990.
39. Vanin, E. F., Kaloss, M., Broscius, G., and Nienhuis, A. W., Characterization of replication-competent retroviruses from nonhuman primates with virus-induced T-cell lymphomas and observations regarding the mechanism of oncogenesis, J. Vir., 68, 4241, 1994.
40. Gornetta,K.,Morgan,R. A.,and Anderson,W.F., Safety issues related to retroviral-mediated gene transfer in humans, Hum. Gene Ther., 2, 5, 1991.
41. Cornetta, K., Safety aspects of gene therapy, Br. J. Haem., 80, 421, 1992.
42. Uckert, W. and Walther, W., Retrovirus-mediated gene tramsfer in cancer therapy, Pharmac. Ther., 63, 323, 1994
43. Jolly, D., Viral vector systems for gene therapy, Gene Ther., 1, 31, 1994.
44. Vile, R. G., Tuszynski, A., and Castleden, S., Retroviral vectors. From laboratory tools to molecular medicine, Mol-Biotechnol., 5, 139, 1996.
45. Trapnell, B. C., Adenoviral vectors for gene transfer, Adv. Drug Del. Rev., 12, 185, 1993.
46. Addison, G. L., Braciak, T., Ralston, R., Muller, W.J., Gauldie, J., and Graham, F. L., Intratumoral injection of an adenovirus expressing interleukin 2 induces regression and immunity in a murine breast cancer model, Proc. Natl Acad. Sci. USA, 92, 8522, 1995.
47. Chen, S. H., Shine, H. D., Goodman,J. C., Grossman, R. G., and Woo, S. L., Gene therapy for brain tumors: Regression of experimental gliomas by adenovirus-mediated gene transfer in vivo, Proc. Natl. Acad. Sci. USA, 91, 3054, 1994.
48. Chen, S. H., Chen,X. H.,Wang,Y.,Kosai, K., Finegold, M.J., Rich, S. S.,and Woo, S. L., Combination gene therapy for liver metastasis of colon carcinoma in vivo, Proc. Natl. Acad. Sci. USA, 92, 2577, 1995.
49. Clayman, G.L.,el-Naggar,A.K.,Roth,J.A.,Zhang,W.W.,Goepfert,H.,Taylor,D.L., and Liu,T.J., In vivo molecular therapy with p53 adenovirus for microscopic residual head and neck squamous carcinoma, Cancer Research, 55, 1, 1995.
50. Eissa, N. T., Chu, C. S., Danel, C., and Crystal, R. G., Evaluation of the respiratory epithelium of normals and individuals with cystic fibrosis for the presence of adenovirus E1a sequences relevant to the use of EIa-adenovirus vectors for gene therapy for the respiratory manifestations of cystic fibrosis, Hum. Gene Ther., 5, 1105, 1994.
51. Fang, B., Eisensmith, R. G.,Wang ,H., Kay, M. A., Cross, R.E., Landen, C.N., Gordon, G., Bellinger, D. A., Read, M. S., Hu, P. C., Brinkhous, K. M., and Woo, S. L. C., Gene therapy for hemophilia B: Host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression, Hum. Gene Ther., 6, 1039, 1995.
52. Viola,J.J., Ram, Z., Walbridge, S., Oshiro, E. M., Trapnell, B., Tao-Cheng,J. H., and Oldfield, E. H., Adenovirally mediated gene transfer into experimental solid brain tumors and leptomeningeal cancer cells, J. Neurosurg, 82, 70, 1995.
53. Yei, S., Mittereder, N., Tang, K., O'Sullivan, G., and Trapnell, B. C., Adenovirus-mediated gene transfer for cystic fibrosis: Quantitative evaluation of repeated in vivo vector administration to the lung, Gene Ther., 1, 192, 1994.
54. Smith, A. E., Viral vectors in gene therapy, Annu. Rev. Microbiol., 49, 807, 1995.
55. Dai, Y., Schwarz, E.M., Gu, D., Zhang, W-W., Sarvetnick, N., and Verma, I.M., Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression,Proc. Natl. Acad. Sci. USA, 92, 1401, 1995.
56. Kremer, E.J. and Perricaudet, M., Adenovirus and adeno-associated virus mediated gene transfer, Med. Bull., 51, 31, 1995.
57. Yei, S., Mittereder, N., Wert, S., Whitsett,J. A., Wilmott, R. W., and Trapnell, B. C., In vivo evaluation of the safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lung, Hum. Gene Ther., 5, 731, 1994.
58. Brody, S. L., Metzger, M., Danel, G., Rosenfeld, M. A., and Crystal, R. G., Acute responses of non-human primates to airway delivery of an adenovirus vector containing the human cystic fibrosis transmembrane conductance regulator cDNA, Hum. Gene Ther., 5, 821, 1994.
59. Zabner, J., Petersen, D. M., Puga, A. P., Graham, S. M., Couture, L. A., Keyes, L. D., Lukason, M. J., St George, J. A., Gregory, R. J., Smith, A. E., and Welsh, M. J., Safety and efficacy of repetitive adenovirus-mediated transfer of CFTR cDNA to airway epithelia of primates and cotton rats, Nature Genet., 6, 7, 1994.
60. Yang, Y., Ertl, H. C., and Wilson,J. M., MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses, Immunity, 1, 433, 1994.
61. Yang, Y., Li, Q., Ertl, H. G., and Wilson,J. M., Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses, J. Virol., 69, 2004, 1995.
62. Yang, Y., Nunes, F. A., Berencsi, K., Gonczol, E., Engelhardt, J. F., and Wilson, J. M., Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis, Nature Genetics, 7, 362, 1994.
63. Kotin, R. M., Prospects for the use of adeno-associate virus as a vector for human gene therapy, Hum. Gene Ther., 5, 793, 1994.
64. Podsakoff, G., Wong, K. K.,Jr., and Chatterjee, S., Efficient gene transfer into nondivicling cells by adeno-associated virus-based vectors, J. Virol., 68, 5656, 1994.
65. Russell, D. W., Miller, A. D., and Alexander, I. E., Adeno-associated virus vectors preferentially transduce cells in S phase, Proc. Natl. Acad. Sci. USA, 91, 8915, 1994.
66. Flotte, T. R., Barraza-Ortiz, X., Solow R., Afione, S. A., Carter, B.J., and Guggino, W. B., An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction, Gene Ther. 2, 29, 1995.
67. Rolling, F. and Samulski, R.J., AAV as a viral vector for human gene therapy, Generation of recombinant virus, Molecular Biotechnology, 3, 9, 1995.
68. Flotte, T. R., Afione, S. A., Conrad, C., McGrath, S. A., Solow, R., Oka, H., Zeitlin, P. L., Guggino, W. B., and Carter, B. J., Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector, Proc. Natl. Acad. Sci. USA, 90, 10613, 1993.
69. Guriel, 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, 1, 1996.
70. Tomlinson, E. and Rolland, A., Controllable gene therapy: Pharmaceutics of non-viral gene delivery systems, J. Control. Rel., 39, 357, 1996.
71. Tomlinson, E., Theory and practice of site-specific drug delivery, Adv. Drug Del. Revs., 1, 87, 1987.
72. Rolland, A., Pharmaceutical Particulate Carriers: Therapeutic Applications, New York: Marcel Dekker, 1993.
73. Lew, D., Parker,S.E.,Latimer,T.,Abai,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 injection in mice, Hum. Gene Ther., 6, 553, 1995.
74. Liu, Y., Liggitt, D., Zhong, W., Tu, G., Gaensler, K., and Debs, R., Cationic liposome-mediated intravenous gene delivery, J. Biol. Chem., 270, 24864, 1995.
75. Thierry, A. R., Lunardi-Iskandar, Y., Bryant, J. L., Rabinovich, P., Gallo, R. C., and Mahan, L. C., Systemic gene therapy: Biodistribution and long-term expression of a transgene in mice, Proc. Natl. Acad. Sci. USA, 92, 9742, 1995.
76. Ledley,T. A. S. and Ledley, F. D., A multicompartment, numerical model of cellular events in the pharmacokinetics of gene therapies, Hum. Gene Ther., 5, 679, 1994.
77. Zabner, J., Fasbender Al,J., Moninger, T., Poellinger, K. A., and Welsh, M.J., Cellular and molecular barriers to gene transfer by a cationic lipid, J. Biol. Chem., 270, 18997, 1995.
78. Sellheyer,K.,Bickenbach,J.R.,Rothnagel,J.A.,Bundman,D.,Longley,M.A., Krieg,T., Roche, N. S., Roberts, A. B., and Roop, D. R., Inhibition of skin development by overexpression of transforming growth factor β1 in the epidermis of transgenic mice, Proc. Natl. Acad. Sci. USA, 90, 5237, 1993.
79. Coleman, M. E., Yin, K. C., DeMayo, F. J., and Schwartz, R. J., Regulatory elements of the chicken α-skeletal actin gene direct high level and tissue specific expression of a hIGFI cDNA in cultured myogenic cells and transgenic mice resulting in enhanced myogenesis and pronounced muscle hypertrophy, Keystone Symposium on Molecular Biology of Muscle Development, J. Cell Biochem., W25, 1994.
80. Anwer, K., Shi, M., French, M., Muller, S., Chen,W.,Liu, Q.,Thompson, B.,Wang,J., Mumper, R., Rolland, A., and Alila, H., Systemic effect of human growth hormone after intramuscular injection of a single dose of a muscle-specific gene medicine, Gene Therapy, 9, 5, 1998.
81. Schwartz, R. J., DeMayo, F. J., and O'Malley, B. W., Myogenic Vector Systems, U. S. Patent 5, 298, 422, 1994.
82. Wang, Y., O'Malley, B. W., Jr, Tsai, S. Y., and O'Malley, B. W., A regulatory system for use in gene transfer, Proc. Natl. Acad. Sci. USA, 91, 8180, 1994.
83. Wang, Y., DeMayo, F. J., Tsai, S. Y., and O'Malley, B., Ligand-inducible and liver-specific target gene expression in transgenic mice, Nature Biotech., 15, 239, 1997.
84. 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, 1068, 1983.
85. Soriano, P., Dijkstra,J., Legrand, A., Spanjer, H., Londos-Gagliardi, D., Roerdink, F., Scherphof, G., and Nicolau, G., Targeted and nontargeted liposomes for in vivo transfer to rat liver cells of a plasmid containing the preproinsulin I gene, Proc. Natl. Acad. Sci. USA, 80, 7128, 1983.
86. Nicolau, C. and Gudd, A., Liposomes as carriers of DNA, Crit Rev. Ther. Drug Carrier Syst., 6, 239, 1989.
87. Alino, S. F., Garcia-Sanz, M.,Irruairizaga, A., Alfaro,J.,and Hernandez,J., High encapsulation efficiencies in sized liposomes produced by extrusion of dehydration-rehydration vesicles, J. Microencaps., 7, 497, 1990.
88. Alino, S. F., Bobadilla, M., Unda, F.J., Garcia-Sanz, M., Hilario, E., and Lejarreta, M., Recombinant cDNA encapsulation in small liposomes with hepatocyte access ability, J. Microencaps., 10, 163, 1993.
89. Fraley, R. T., Subramanii, S., Berg, P., and Papahadjopoulos, D., Introduction of liposomes encapsulated SV40 DNA into cells, J. Biol. Chem., 255, 10431, 1980.
90. Alino,S.F.,Crespo,J.,Bobadilla,M.,Lejaireta,M.,Blaya,C.,and Crespo,A.,Expression of human α1-antitrypsin in mouse after in vivo gene transfer to hepatocytes by small liposomes, Biochem. Biophys. Res. Comm., 204, 1023, 1994.
91. Baru, M., Axelrod, J. H., and Nur, I., Liposome-encapsulated DNA-mediated gene transfer and synthesis of human factor IX in mice, Gene, 161, 143, 1995.
92. Wang, C. Y and Huang, L., pH-sensitive immunoliposomes mediate target-cell-specific delivery and controlled expression of a foreign gene in mouse, Proc. Natl. Acad. Sci. USA, 84, 7851, 1987.
93. Legendre, J. Y. and Szoka, F. C., Jr., Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: Comparison with cationic liposomes, Pharm. Res., 9, 1235, 1992.
94. Kaneda, Y., Iwai, K., and Uchida, T., Increased expression of DNA cointroduced with nuclear protein in adult rat liver, Science, 243, 375, 1989.
95. Kato, K., Nakanishi, M., Kaneda, Y., Uchida, T., and Okada, Y., Expression of hepatitis B virus surface antigen in adult rat liver. Co-introduction of DNA and nuclear protein by a simplified liposome method, J. Biol. Chem., 266, 3361, 1991.
96. Wang, C. Y. and Huang, L., Highly efficient DNA delivery mediated by pH-sensitive immunoliposomes, Biochem., 28, 9508, 1989.
97. Zhou,X. and Huang, L., Targeted delivery of DNA by liposomes and polymers, J. Control. Rel., 19, 269, 1992.
98. 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 proteoliposomes, Gene, 63, 321, 1988.
99. Gould-Fogerite, S., Mazurkiewicz, J.E., Raska, K., Voelkerding, K.,Lehman, J.M., and Mannino, R. J., Chimerasome-mediated gene transfer in vitro and in vivo, Gene, 84, 429, 1989.
100. Kato, K., Kaneda, Y., Sakurai, M., Nakanishi, M.,and Okada, Y., Direct injection of hepatitis B virus DNA into liver induced hepatitis in adult rats,J. Biol., Chem., 266, 22071, 1991.
101. Von der Leyen, H. E., Gibbons, G. H., Morishita, R., Lewis, N. P., Zhang, L., Nakajima, M., Kaneda, Y., Cooke,J. P.,and Dzau, V.J., Gene therapy inhibiting neointimal vascular lesion: In vitro transfer of endothelial cell nitric oxide synthase gene, Proc. Natl. Acad. Sci. USA, 92, 1137, 1995.
102. Felgner,P.L. and Ringold,G.M., Cationic liposome-mediated transfection, Nature, 337, 387, 1989.
103. Felgner, P. L., Gadek,T. R., Holm, M., Roman, R., Chan, H. W.,Wenz, M.,Northrop, J. P., Ringold, G. M., and Danielsen, M., Lipofection; A highly efficient, lipid-mediated DNA-transfection procedure, Proc. Natl. Acad. Sci. USA, 84, 7413, 1987.
104. Rose,J. K., Buonocore, L., and Whitt, M. A., A new cationic liposome reagent mediating nearly quantitative transfection of animal cells, Biotechniques, 10, 520, 1991.
105. Pinnaduwage, P., Schmitt, L., and Huang, L., Use of quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells, Biochim. Biophys. Acta., 985, 33, 1989.
106. Zhou, X., Klibanov, A. L., and Huang, L., Lipophilic polylysines mediate efficient DNA transfection in mammalian cells, Biochim. Biophys. Acta., 1065, 8, 1991.
107. Trubetskoy, V. S., Torchilin, V. P., Kennel, S.J., and Huang, L., Use of N-terminal modified poly(L-lysine)-antibody conjugates as a carrier for targeted gene delivery in mouse lung endothelial cells, Bioconj. Chem., 3, 323, 1992.
108. Jiao, S., Williams, P., Berg, R. K., Hodgeman, B. A., Liu, L., Repetto, G., and Wolff, J. A., Direct gene transfer into nonhuman primate myofibers in vivo, Hum. Gene Ther., 3, 21, 1992.
109. Campbell, M. J., Lipofection reagents prepared by a simple ethanol injection technique, Biotechniques, 18, 1027, 1995.
110. Lewis, J. G., Lin, K.-Y., Kothavale, A., Flanagan, M., Matteucci, M. D., DePrince, R. B., Mook, R. A., Jr., Hendren, R. W., and Wagner, R. W., A serum-resistant cyofectin for cellular delivery of antisense oligodeoxynucleotides and plasmid DNA, Proc. Natl. Acad. Sci. USA, 93, 3176, 1996.
111. Puyal, G., Maurin, L., Miquel, G., Bienvenue, A., and Philippot, J., Design of a short membrane-destabilizing peptide covalently bound to liposomes, Biochim. Biophys. Acta., 1195, 259, 1994.
112. Solodin, I., Brown, C. S., Bruno, M. S., Chow, C.-Y., Jang, E.-H., Debs, R. J., and Heath, T. D., A novel series of amphiphilic imidazolinium compounds for in vitro and in vivo gene delivery, Biochem., 34, 13537, 1995.
113. Cheng, P.-W., Receptor ligand-facilitated gene transfer: Enhancement of liposome-mediated gene transfer and expression by transferrin, Hum. Gene Ther., 7, 275, 1996.
114. Walker, S., Sofia, M.J., Kakarla, R., Kogan, N. A., Wierichs, L., Longley, G. B., Bruker, K., Axelrod, H. R., Midha, S., Babu, S., and Kahne, D., Cationic facial amphiphiles: A promising class of transfection agents, Proc. Natl. Acad. Sci. USA, 93, 1585, 1996.
115. 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, Biochem., 11, 19, 1994.
116. Felgner,J. H., Kumar, R., Sridhar, 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, 2550, 1994.
117. Harrison, G.S., Wang, Y., Tomczak,J.,Hogan, C., Shpall, E.J., Curiel,T.J.,and Felgner, P. L., Optimization of gene transfer using cationic lipids in cell lines and primary human CD4+ and CD34+ hematopoietic cells, Biotechniques, 19, 816, 1995.
118. Wheeler, C.J., Sukhu, L., Yang, G., Tsai, Y., Bustamente, C., Feigner, P., Norman, J., and Manthorpe, M., Converting an alcohol to an amine in a cationic lipid dramatically alters the co-lipid requirement, cellular transfection activity and the ultrastructure of DNA-cytofectin complexes, Biochim. Biophys. Acta., 1280, 1, 1996.
119. Zhou, X. and Huang, L., DNA transfection mediated by cationic liposomes containing lipopolylysine: Characterization and mechanism of action, Biochim. Biophys. Acta., 1189, 195, 1994.
120. Behr, J.-P., DNA strongly binds to micelles and vesicles containing lipopolyamines or lipointercalants, Tetrahedron Lett., 27, 5861, 1986.
121. Behr,J.-P., Demeinex, 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, 6982, 1989.
122. Loeffler, J. Ph., Barthel, F., Feltz, P., Behr, J. P., Sassone-Corsi, P., and Feltz, A., Lipopolyamine-mediated transfection allows gene expression studies in primary neuronal cells, J. Neurochem., 54, 1812, 1990.
123. Behr, J.-P., Gene transfer with synthetic cationic amphiphiles: Prospects for gene therapy, Bioconj. Chem., 5, 382, 1994.
124. Mack, K. D., Walzem, R., and Zeldis, J. B., Cationic lipid enhances in vitro receptor-mediated transfection, Am. J. Med. Sci., 307, 138, 1994.
125. Remy, J. S., Sirlin, C., Vierling, P., and Behr, J. P., Gene transfer with a series of lipophilic DNA-binding molecules, Bioconj. Chem., 5, 647, 1994.
126. 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 stage toward artificial viruses, Proc. Natl. Acad. Sci. USA, 92, 1744, 1995.
127. Staedel, C., Remy, J. S., Hua, Z., Broker, T. R., Chow, L. T., and Behr, J. P., High-efficiency transfection of primary human keratinocytes with positively charged lipopolyamine:DNA complexes, J. Invest. Dermatol., 102, 768, 1994.
128. Gao, X. A. and Huang, L., A novel cationic liposome reagent for efficient transfection of mammalian cells, Biochem. Biophys. Res. Commun., 179, 280, 1991.
129. Gao, X. and Huang, L., Cationic liposome-mediated gene transfer, Gene Ther., 2, 710, 1995.
130. Farhood, H., Bottega, R., Epand, R. M., and Huang, L., Effect of cationic cholesterol derivatives on gene transfer and protein kinase C activity, Biochim. Biophys. Acta., 1111, 239, 1992.
131. Balasubramaniam, R. P., Bennett, M.J., Aberle, A. M., Malone, J. G., Nantz, M. H., and Malone, R. W., Structural and functional analysis of cationic transfection lipids: The hydrophobic domain, Gene Ther., 3, 163, 1996.
132. Aberle, A. M., Bennett, M. J., Malone, R. W., and Nantz, M. H., The counterion influence on cationic lipid-mediated transfection of plasmid DNA, Biochim. Biophys. Acta., 1299, 281, 1996.
133. Bennett, M. J., Nantz, M. H., Balasubramaniam, R. P., Gruenert, D. C., and Malone, R. W., Cholesterol enhances cationic liposome-mediated DNA transfection of human respiratory epithelial cells, Bioscience Reports, 15, 47, 1995.
134. Farhood, H., Serbina, N., and Huang, L., The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer, Biochim. Biophys. Acta., 1235, 289, 1995.
135. Gustafsson,J., Arvidson, G., Karlsson, G., and Almgren, M., Complexes between cationic liposomes and DNA visualized by cryo-TEM, Biochim. Biophys. Acta., 2, 305, 1995.
136. Felgner, P. L., Tsai, Y. J., Sukhu, L., Wheeler, C. J., Manthorpe, M., Marshall, J., and Cheng, S. H., Improved cationic lipid formulations for in vivo gene therapy, Annals New York Academy of Sciences, 126, 1996.
137. 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, 361, 1994.
138. Gershon, H., Ghirlando, R., Guttman, S. B., and Minsky, A., Mode of formation and structural features of DNA-cationic liposome complexes used for transfection, Biochem., 32, 7143, 1993.
139. Smith, J. G., Walzem, R. L., and German, J. B., Liposomes as agents of DNA transfer, Biochim. Biophys. Acta., 1154, 327, 1993.
140. Wrobel, I. and Collins, D., Fusion of cationic liposomes with mammalian cells occur after endocytosis, Biochim. Biophys. Acta., 1235, 296, 1995.
141. Van der Woude, I., Visser, H. W., Ter Beest, M. B. A., Wagenaar, A., Ruiters, M. H. J., Engberts, J. B. F. N., and Hoekstra, D., Parameters influencing the introduction of plasmid DNA into cells by the use of synthetic amphiphiles as a carrier system, Biochim. Biophys. Acta., 1240, 35, 1995.
142. Fasbender, A. J., Zabner, J., and Welsh, M. J., Optimization of cationic lipid-mediated gene transfer to airway epithelia, Am. J. Physiol., 269, L45, 1995.
143. Gapecchi, M. R., High efficiency transformation by direct microinjection of DNA into cultured mammalian cells, Cell, 22, 479, 1980.
144. Xu, Y. and Szoka, F. C.,Jr., Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection, Biochem., 35, 5616, 1996.
145. Rolland, A., Duguid, J., Barron, M., Gong, L., Levin, J., and Eastman, E., Characterization of non-viral gene medicines, Proceed. Intern. Symp. Control. Rel. Bioact. Mater., 21, 240, 1994.
146. Gong, L., Claspell, J., and Rolland, A., The effect of Lipofectin™ complexation on the characteristics and stability of DNA expression vectors, Pharm. Res., 11, S-77, 1994.
147. Bertling, W. M., Gareis, M., Paspaleeva,V., Zimmer, A., Kreuter,J., Nurnberg, E., and Harrer, P., Use of liposomes, viral capsids, and nanoparticles as DNA carriers, Biotech. Appl. Biochem., 13, 390, 1991.
148. Brigham, K. L., Meyrick, B., Christman, B., Magnuson, M., King, G., and Berry, L. C., Jr., In vivo transfection of murine lungs with a functioning prokaiyotic gene using a liposome vehicle, Am. J. Med. Sci., 298, 278, 1989.
149. Brigham, K. L., Meyrick, B., Christman, B., Conary, J. T., King, G., Berry, L. C. Jr., 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, 209, 1993.
150. Canonico, A. E., Conary, J. T., Meyrick, B. O., and Brigham, K. L., Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits, Am. J. Respir. Cell Mol. Biol, 10, 24, 1994.
151. Alton, E. W., Middleton, 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., Fasold, K. I., Miller, A. D., Dickinson, P., Stevenson, B. J., MacLachlan, G., Dorin, J. R., and Porteous, D. J., Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice, Nat. Genet., 5, 135, 1993.
152. 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, 250, 1993.
153. Geisert, E. E., Jr., Del Mar, N. A., Owens, J. L., Holmberg, E. G., Logan, J. J., Bebok, Z., Walker, L. C., Peng, S., Feigner, P. L., Siegal, G. P., Frizzell, RA., Dong, J., Howard, M., Matalon, S., Lindsey, J.R., DuVall, M., and Sorscher, E.J., Cationiclipids for reporter gene and CFTR transfer to rat pulmonary epithelium, Gene Ther., 2, 38, 1995.
154. Stribling, R., Brunette, E., Liggit, D., Gaensler, K., and Debs, R., Aerosol gene delivery in vivo, Proc. Natl. Acad. Sci. USA, 89, 11277, 1992.
155. 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 gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer, Nucl. Acids Res., 20, 3233, 1992.
156. Logan, J. J., Bebok, Z., Walker, L. C., Peng, S., Feigner, P. L., Siegal, G. P., Frizzell, R. A., Dong, J., Howard, M., Matalon, S., Lindsey, J. R., DuVall, M., and Sorscher, E. J., Cationic lipids for reporter gene and CFTR transfer to rat pulmonary epithelium, Gene Ther., 2, 38, 1995.
157. 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. J., Gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: A phase I trial of safety and efficacy in the nasal airway, Hum. Gene Ther., 5, 1259, 1994.
158. 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, 731, 1996.
159. Nabel, E. G., Plautz, G., and Nabel, G.J., Transduction of a foreign histocompatibility gene into the arterial wall induces vasculitis, Proc. Natl. Acad. Sci. USA, 89, 5157, 1992.
160. Nabel, E. G., Shum, L., Pompili, V.J., Yang, Z. Y., San, H., Shu, H. B., Liptay, S., Gold, L., Gordon, D., and Derynck, R., Direct transfer of transforming growth factor beta I gene into arteries stimulates fibrocellular hyperplasia, Proc. Natl. Acad. Sci. USA, 90, 10759, 1993.
161. Nabel, E. G., Yang, Z., Liptay, S., San, H., Gordon, D., Haudenschild, C. C., and Nabel, G.J., Recombinant platelet-derived growth factor B gene expression in porcine arteries induce intimal hyperplasia in vivo, J. Clin. Invest., 91, 1822, 1993.
162. Nabel, E. G., Yang, Z. Y., Plautz, G., Forough, R., Zhan, X., Haudenschild, C. C., Maciag, T., and Nabel, G. J., Recombinant fibroblast growth factor-1 promotes intimai hyperplasia and angiogenesis in arteries in vivo, Nature, 362, 844, 1993.
163. 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, 1834, 1994.
164. Zhu, N., Liggitt, D., Liu, Y., and Debs, R., Systemic gene expression after intravenous DNA delivery into adult mice, Science, 261, 209, 1993.
165. Plautz, G.E., Yang, Z.Y., Wu, B.Y., Gao, X., Huang, L.,and Nabel, G.J., Immunotherapy of malignancy by in vivo gene transfer into tumors, Proc. Natl Acad. Sci. USA, 90, 4645, 1993.
166. Nabel, G.J., Nabel, E.G., Yang, Z.Y., Fox, B.A., Plautz, G.E., Gao, X., Huang, L., Shu, S., Gordon, D., and Chang, A. E., Direct gene transfer with DNA-liposome complexes in melanoma: Expression, biologic activity, and lack of toxicity in humans, Proc. Natl. Acad. Sci. USA, 90, 11307, 1993.
167. Lesoon-Wood, L.A., Kim, W. H., Kleiman, H. K., Weintraub, B. D., and Mixson, A.J., Systemic gene therapy with p53 reduces growth and mestatases of a malignant human breast cancer in nude mice, Hum. Gene Ther., 6, 395, 1995.
168. Raz, E., Carson, D.A., Parker, S.E., Parr, T.B., Abai, A.M., Aichinger, G., Gromkowski, S. H., Singh, M., Lew, D., Yankauckas, M. A., Baird, A. M., and Rhodes, G. H., Intradermal gene immunization: The possible role of DNA uptake in the induction of cellular immunity to viruses, Proc. Natl. Acad. Sci. USA, 91, 9519, 1994.
169. Ono, T., Fujino, Y., Tsuchiya, T., and Tsuda, M., Plasmid DNAs directly injected into mouse brain with Lipofectin can be incorporated and expressed by brain cells, Neurosci. Letters, 117, 259, 1990.
170. Wattiaux, R., Jadot, M., Dubois, F., Misquith, S.,and Wattiaux-De Coninck, S., Uptake of exogenous DNA by rat liver: Effect of cationic lipids, Biochim. Biophys. Res. Commun., 213, 81, 1995.
171. Hazinski, T. A., Ladd, P. A., and Dematteo, G. A., Localization and induced expression of fusion genes in the rat lung, Am. J. Respir. Cell Mol. Biol., 4, 206, 1991.
172. Debs, R., Pian, M., Gaensler, K., Clements, J., Friend, D. S., and Dobbs, L., Prolonged transgene expression in rodent lung cells, Am. J. Respir. Cell Mol. Biol., 7, 406, 1992.
173. Bout, A., Valerio, D., and Scholte, B. J., In vivo transfer and expression of the lacZ gene in the mouse lung, Exp. Lung Res., 19, 193, 1993.
174. Middleton, P. G., Gaplen, N.J.,Gao,X., Huang,L.,Gaya, H.,Geddes,D. M.,and Alton, E. W., Nasal application of the cationic liposome DC-Chol:DOPE does not alter ion transport, lung function or bacterial growth, Eur. Respir. J., 7, 442, 1994.
175. Tsan, M-F., White, J. E., and Shepard, B., Lung-specific direct in vivo gene transfer with recombinant plasmid DNA, Am. J. Physiol., 268 (Lung Cell. Mol. Physiol. 12), L1052-L1056, 1995.
176. Fortunati, E., Bout, A., Zanta, M. A., Valerio, D., and Scarpa, M., In vitro and in vivo gene transfer to pulmonary cells mediated by cationic liposomes, Biochim. Biophys. Acta., 1306,55,1996.
177. 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., Huang, L., Porteous, D. J., Williamson, R., and Geddes, D. M., Liposome mediated GFTR gene transfer to the nasal epithelium of patients with cystic fibrosis, Nature Medicine, 1, 39, 1995.
178. Schreier, H. and Sawyer, S. M., Liposomal DNA vectors for cystic fibrosis gene therapy. Current applications, limitations, and future directions, Adv. Drug Deliv. Rev., 19, 73, 1996.
179. Brigham, K.L., Canonico, A.E.,Conary, J.T., Meyrick, B.O., and Schreier, H., Potential for gene therapy in the treatment of sepsis, in Sepsis: Current Perspectives in Pathophysiology and Therapy, Berlin: Springer-Verlag, 1994, 528.
180. Canonico, A. E., Plitman, J. D., Conary, J. T., Meyrisk, B. O., and Brighman, K. L., No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes, J. Appl. Physiol., 77, 415, 1994.
181. 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 vive with DNA-liposome complexes: Safety and acute toxicity in mice, Hum. Gene Ther., 3, 267, 1992.
182. Nabel, E. G., Gordon, D., Yang, Z.-Y., Xu, L., San, H., Plautz, G. E.,. Wu, B.-Y., Gao, X., Huang, L., and Nabel, G. J., Gene transfer in vivo with DNA-liposome complexes: Lack of autoimmunity and gonadal localization, Hum. Gene. Ther., 3, 649, 1992.
183. San,H., Yang, Z.-Y., Pompili, V.J., Jaffe, M. L., Plautz, G.E., Xu, L., Felgner, J.H., Wheeler, C.J.,Felgner,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, 781, 1993.
184. Parker, S.E., Vahlsing, H.L., Serfilippi, L.M., Franklin, C.L., Doh, S.G. ,Gromkowski, S.H., Lew, D., Manthorpe, M., and Norman, J., Cancer gene therapy using plasmid DNA: Safety evaluation in rodents and non-human primates, Hum. Gene Ther., 6, 575, 1995.
185. Senior, J. H.,Trimble, K. R., and Maskiewicz, R., Interaction of positively-charged liposomes with blood: Implications for their application in vivo, Biochim. Biophys. Acta., 1070, 173, 1991.
186. Gagne, L., Conary, J. T., Canonico, A. E., Brigham, K. L., and Schreier, H., Liposomemediated gene delivery: II. In vitro toxicity of cationic liposome DNA plasmid complexes, Proceed. Intern. Symp. Control. Rel. Bioact. Mat., 21, 236, 1994.
187. Bottega, R. and Epand, R. M., Inhibition of protein kinase C by cationic amphiphiles, Biochem., 31, 9025, 1992.
188. Toyofuku,. T., Koyama, S., Kobayashi, T., Kusama, S., and Ueda, G., Effects of polycations on pulmonary vascular permeability in conscious sheep, J. Clin. Invest., 83, 2063, 1989.
189. Plank, C., Mechtler, K., Szoka, F. C.,Jr., and Wagner, E., Activation of the human complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery, Hum. Gene Ther., 7, 1437, 1996.
190. 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. F. W., Geddes, D. M., Coutelle, C., Williamson, R., Huang, L., and Gilchrist, G., Gene therapy for cystic fibrosis in humans by liposome-mediated DNA transfer: The production of resources and the regulatory process, Gene Ther., 1, 139, 1994.
191. Ishikawa, Y. and Homey, C. J., High efficiency gene transfer into mammalian cells by a double transfection protocol, Nucleic Acids Res., 20, 4367, 1992.
192. Kawai, S. and Nishizawa, M., New procedure for DNA transfection with polycation and dimethyl sulfoxide, Mol. Gel Biol., 4, 1172, 1984.
193. Kabanov, A. V. and Kabanov, V. A., DNA complexes with polycations for the delivery of genetic material into cells, Bioconj. Chem., 6,7,1995.
194. Haensler, J. and Szoka, F. C.,Jr., Polyamidoamine cascade polymers mediate efficient transfection of cells in culture, Bioconj. Chem., 4, 372, 1993.
195. Kukowska-Latallo, J. F., Bielinska, A. U.,Johnson, J., Spindler, R.,Tomalia, D. A., and Baker,J. R.,Jr., Efficient transfer ofgenetic material into mammalian cells using Starburst polyamidoamine dendrimers, Proc. Natl. Acad. Sci. USA, 93, 4897, 1996.
196. Boussif, O, Lezoualc'h, F, Zanta, 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. Sci. USA, 92, 7297, 1995.
197. 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, 138, 1990.
198. 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, 178, 1995.
199. Parente, R. A., Nadasdi, L., Subbarao, N. K., and Szoka, F. C., Jr., Association of a pH-sensitive peptide with membrane vesicles: Role of amino acid sequence, Biochemistry, 29, 8713, 1990.
200. Parente, R. A., Nir, S., and Szoka, F. C.,Jr., Mechanism of leakage of phospholipid vesicle contents induced by the peptide GALA, Biochemistry, 29, 8720, 1990.
201. Hirano, S., Tobetto, K., Hasegawa, M., and Matsuda, N., Permeability properties of gels and membranes derived from chitosan, J. Biomed. Mater. Res., 14, 477, 1980.
202. Chandy, T. and Sharma, C. P., Chitosan as a biomaterial, Biomat., Art. Cells, Art. Org., 18, 1, 1990.
203. Kim, K. Y. and Min, D. S., Wound covering materials from polyelectrolyte complexes of chitosan with sulfonated chitosan, Trans. Soc. Biomat., 11, 558, 1988.
204. Ouchi, T. and Banba, T., Fixation of 5-fluorouracil to chitosan and its antitumor activity, Trans. Soc. Biomat., 11, 232, 1988.
205. Israel, M. and Murray, R. J., Adiamycin analogues: Preparation and antitumor evaluation, J. Med. Chem., 25, 24, 1982.
206. Kristl, J., Smid-Korbar, J., Struc, E., Schara, M.,and Rupprecht, H., Hydrocolloids and gels of chitosan as drug carriers, Int. J. Pharm., 99, 13, 1993.
207. Shiraishi, S., Imai, T., and Otagiri, M., Controlled release of indomethacin by chitosan-polyelectrolyte complex: Optimization and in vivo/in vitro evaluation, J. Contr. Rel., 25, 217, 1993.
208. Hirano, S., Seino, H., Akiyama, Y., and Nonaka, I., Biocompatibility of chitosan by oral and intravenous administration, Polym. Eng. Sci., 59, 897, 1988.
209. 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, 1465, 1990.
210. Wolff, J. A., Williams, P., Acsadi, G.,Jiao, S.,Jani, A., and Chong, W., Conditions affecting direct gene transfer into rodent muscle in vivo, Biotechniques, 11, 474, 1991.
211. Wolff, J. A., Dowty, M. E., Jiao, S., Repetto, G., Berg, R. K., Ludtke, J.J., and Williams, P., Expression of naked plasmids by cultured myotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle, J. Cell Sci., 103, 1249, 1992a.
212. 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, 363, 1992.
213. Acsadi, G., Jiao, S.S., Jani, A., Duke, D.,Williams, P.,Chong, W.,and Wolff, J.A., Direct gene transfer and expression into rat heart in vivo, NewBiol., 3, 71, 1991.
214. Acsadi, G., Dickson, G., Love, D. R.Jani, A., Walsh, F. S., Gurusinghe, A., Wolff,J. A., and Davies, K. E., Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs, Nature, 352, 815, 1991.
215. Manthorpe, M., Cornefert-Jensen, F., Hartikka, J., Felgner,J., Rundell, A., Margalith, N., and Dwarki, V., Gene therapy by intramuscular injection of plasmid DNA: Studies on firefly luciferase gene expression in mice, Hum. Gene Ther., 4, 419, 1993.
216. Lin, H., Parmacek, M. S., Morle, G., Boiling, S., and Leiden, J. M., Expression of recombinant genes in myocardium in vivo after direct injection of DNA, Circulation, 82, 2217, 1990.
217. Buttrick, P. M., Kass, A., Kitsis, R. N., Kaplan, M. L., and Leinwand, L. A., Behavior of genes directly injected into the rat heart in vivo, Circ. Res., 70, 193, 1992.
218. Gal, D., Weir, L., Leclerc, G., Pickering, J.G., Hogan, J.,and Isner,J. M., Direct myocardial transfection in two animal models. Evaluation of parameters affecting gene expression and percutaneous gene delivery, Laboratory Investigation, 68, 18, 1993.
219. Hickman, M. A., Malone, R. W., Lehmann-Bruinsma, K., Sih,T. R., Knoell, D., Szoka, F. C.,Jr., Walzem, R., Carlson, D. M., and Powell,J. S., Gene expression following direct injection of DNA into liver, Hum. Gene Ther., 5, 1477, 1994.
220. 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, 825, 1995.
221. Riessen, R., Rahimizadeh, H., Blessing, S., Takeshita, S., Barry,J.J., and Isner, J. M., Arterial gene transfer using pure DNA applied directly to a hydro gel-coated angioplasty balloon, Hum. Gene Ther. 4, 749, 1993.
222. Li, L. and Hoffman, R. M., The feasibility of targeted selective gene therapy of the hair follicle, Nature Med., 1, 705, 1995.
223. Ulmer, J. B., Donnelly, J.J., Parker, S. E., Rhodes, G. H., Felgner, P. L., Dwarki, V.J., Gromkowski, S. H., Deck, R. R., Dewitt, G. M., Friedman, A., Hawe, L. A., Leander, K. R., Martinez, D., Perry, H. G., Shiver, J. W., Montgomery, D. L., and Liu, M. A., Heterologous protection against influenza by injection of DNA encoding a viral protein, Science, 259, 1745, 1993.
224. Davis, H. L., Demeneix, B. A., Quantin, B., Coulombe, J., and Whalen, R. G., Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal muscle, Hum. Gene Ther., 4, 733, 1993.
225. Davis, H. L., Michel, M. L., and Whalen, R. G., DNA-based immunization induces continuous secretion of hepatitis B surface antigen and high levels of circulating antibody, Hum. Mol. Genet., 2, 1847, 1993.
226. Raz, E., Watanabe, A., Baird, S. M., Eisenberg, R. A., Parr, T. B., Lotz, M., Kipps, T.J., and Carson, D. A., Systemic immunological effects of cytokine genes injected into skeletal muscle, Proc. Natl Acad. Sci. USA, 90, 4523, 1993.
227. Levy, M. Y., Meyer, K. B., Barron, L., and Szoka, F. C., Jr., Mechanism of gene uptake and expression in adult mouse skeletal muscle, Pharm. Res., 11, 317, 1994.
228. Levy, M. Y., Barron, L. G., Meyer, K. B., and Szoka, F. C.,Jr., Characterization of plasmid DNA transfer into mouse skeletal muscle: Evaluation of uptake mechanism, expression and secretion of gene products into blood, Gene Ther., 3, 201, 1996.
229. Davis, H. L., Whalen, R. G., and Demeneix, B. A., Direct gene transfer into skeletal muscle in vivo: Factors affecting efficiency of transfer and stability of expression, Hum. Gene Ther., 4, 151, 1993.
230. Danko, I., Fritz, J. D., Jiao, S., Hogan, K., Latendresse, J. S., and Wolff, J. A., Pharmacological enhancement of in vivo foreign gene expression in muscle, Gene Ther., 114, 1994.
231. Wells, D. J., Improved gene transfer by direct plasmid injection associated with regeneration in mouse skeletal muscle, FEBS Lett, 332, 179, 1993.
232. Mumper, R.J., Barron, M. K., Anwer, K., Lessard, R. L., Liu, Q., Nitta, H., Alila, H., and Rolland, A. P., Interactive polymeric gene delivery systems for enhanced muscle expression, Pharm. Res., 12, 80, 1995.
233. Mumper, R. J., Duguid, J. G., Anwer, K., Barron, M. K., Nitta, H., and Rolland, A. P., Polyvinyl derivatives as novel interactive polymers for controlled gene delivery to muscle, Pharm. Res., 13, 701, 1996.
234. Blecher, L., Lorenz, D. H., Lowd, H. L., Wood, A. S., and Wyman, D. P., Polyvinylpyrrolidone, in Handbook of Water Soluble Gums and Resins, Davidson, R. L., Ed., New York: McGraw Hill, 1980, Chapter 21.
235. Robinson, B. V., F. M., Sullivan, J. F., Borzelleca, and Schwartz., S. L., PVP: A Critical Review of the Kinetics and Toxicology of Polyvinylpyrrolidone (Povidone), Lewis Publishers, Michigan, 1990.
236. Kamen, B., Wang, M.-T., Streckfuss, A.J., Peryea, X.,and Anderson,R. G.W., Delivery of folates to the cytoplasm of MA104 cells is mediated by a surface membrane receptor that recycles, J. Biol. Chem., 263, 13602, 1988.
237. Rothberg, K. G., Ying, Y. S., Kolhouse, J. F., Kamen, B. A., and Anderson, R. G., The glycophospholipid-linked folate receptor internalizes folate without entering the clathrincoated pit endocytic pathway, J. Cell Biol., 110, 637, 1990.
238. Dowty, M.E., Williams, P., Zhang, G., Hagstrom into postmitotic nuclei of primary rat myotubes, Proc. Natl Acad. Sci. USA, 92, 4572, 1995.
239. Feldherr, C. M. and Akin, D., Role of nuclear trafficking in regulating cellular activity, Int. Rev. Cytol, 151, 183, 1994.
240. Gorlich, D. and Mattaj, I. W., Nucleocytoplasmic transport, Science, 271, 1513, 1996.
241. Sikes, M., O'Malley, B. W., Jr., Finegold, M. J., and Ledley, F. D., In vivo gene transfer into rabbit thyroid follicular cells by direct DNA injection,Hum. Gene Ther., 5, 837, 1994.
242. Meyer, K. B., Thompson, M. M., Levy, M. Y., Barron, L. G., and Szoka, F. G., Jr., Intertracheal gene delivery to the mouse airway: Characterization of plasmid DNA expression and pharmacokinetics, Gene Ther., 2, 450, 1995.
243. Nead, M. A. and McCance, D.J., Poly-L-ornithine-mediated transfection of human keratinocytes, J. Invest. Dermatol., 105, 668, 1995.
244. Wu, G. Y., Wilson, J. M., Shalaby, F., Grossman, M., Shafritz, D. A., and Wu, C. H., Receptor-mediated gene delivery in vivo: Partial correction of genetic analbuminemia in Nagase rats, J. Biol. Chem., 266, 14338, 1991.
245. Wagner, E., Cotten, M., Foisner, R., and Birnstiel, M. L., Transferrin-polycation-DXA complexes: The effect of polycations on the structure of the complex and DNA delivery to cells, Proc. Natl. Acad. Sci. USA, 88, 4255, 1991.
246. Wilson, J. M., Grossman, M., Cabrera, J. A., Wu, C. H., and Wu, G. Y., A novel mechanism for achieving transgene persistence in vivo after somatic gene transfer into hepatocytes, J. Biol. Chem., 267, 11483, 1992.
247. 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 hypercholesterolemia in low density lipoprotein receptor-deficient rabbits, J. Biol. Chem. 267, 963, 1992.
248. Midoux, P., Mendes, C., Legrand, A., Raimond, J., Mayer, R., Monsigny, M., and Roche, A. C., Specific gene transfer mediated by lactosylated poly-L-lysine into hepatoma cells, Nucl. Acids Res., 21, 871, 1993.
249. Gottschalk, S., Cristiano, R. J., Smith, L. C., and Woo, S. L. C., Folate receptor mediated DNA delivery into tumor cells: Potosomal disruption results in enhanced gene expression, Gene Ther., 1, 185, 1994.
250. Perales, J. C., Ferkol, T., Beegen, H., Ratnoff, O. D., and Hanson, R. W., Gene transfer in vivo: Sustained expression and regulation of genes introduced into the liver receptor-targeted uptake, Proc. Natl. Acad. Sci. USA, 91, 4086, 1994.
251. 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, 255, 1994.
252. Baatz, J. E., Bruno, M. D., Ciraolo, P.J., Classer, 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, 2547, 1994.
253. Ross ,G. F., Morris, R. E., Ciraolo, G., Huelsman, K., Bruno, M., Whitsett, J.A., Baatz, J. E.,and Korfhagen, T. R., Surfactant protein A-polylysine conjugates for delivery of DNA to airway cells in culture, Hum. Gene Ther., 6, 31, 1995.
254. 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 monocy te-derived macrophages, Hum. Gene Ther., 7, 721, 1996.
255. Ding, Z-M., Cristiano, R. J., Roth,J. A., Takacs, B., and Kuo, M. T., Malarial circumsporozoite protein is a novel gene delivey vehicle to primary hepatocyte cultures and cultured cells, J. Biol. Chem., 270, 3667, 1995.
256. Ferkol, T., Perales, J. C., Mularo, F., and Hanson, R. W., Receptor-mediated gene transfer into macrophages, Proc. Natl. Acad. Sci. USA, 93, 101, 1996.
257. Buschle, M., Gotten, M., Kirlappos, H., Mechtler, K., Schaffher, 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, 753, 1995.
258. Plank, C., Zatloukal, K., Gotten, M., Mechter, K., and Wagner, E., Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand, Bioconj. Chem., 3, 533 1992.
259. Chiou, H. C., Tangco, M. V., Levine, S. M., Robertson, D., Kormis, K., Wu, C. H., and Wu, G. Y., Enhanced resistance to nuclease degradation of nucleic acids complexed to asialo-glycoprotein-polylysine carriers, Nucl. Acids Res., 22, 5439, 1994.
260. McKee, T. D., DeRome, M. E., Wu, G. Y., and Findeis, M. A., Preparation of asialoorosomucoid-pylysine conjugates, Bioconj. Chem., 5, 306, 1994.
261. Wu, G. Y. and Wu, C. H., Receptor-mediated gene delivery and expression in vivo, J. Biol. Chem., 263, 14621, 1988.
262. Chowdhury, N. R., Wu, C. H., Wu, G. Y., Yerneni, P. C., Bommineni, V. R., and Chowdhury, J. R., Fate of DNA targeted to the liver by asialoglycoprotein receptor-mediated endocytosis in vivo, Prolonged persistence in cytoplasmic vesicles after partial hepatectomy, J. Biol. Chem., 268, 11265, 1993.
263. Stankovics, J., Crane, A. M., Andrews, E., Wu, C. H., Wu, G. Y., and Ledley, F. D., Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine/plasmid complexes, Hum. Gene Ther., 5, 1095, 1994.
264. Wu, C. H., Wilson, J. M., and Wu, G. Y., Targeting genes: Delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo, J. Biol. Chem., 264, 16985, 1989.
265. Wu, G.Y. and Wu, C.H., Liver-directed gene delivery, Ad. Drug Del. Rev., 12, 159, 1993.
266. Frese, J., Jr., Wu, C. H., and Wu, G. Y., Targeting of genes to the liver with glycoprotein carriers, Adv. Drug Delivery Rev., 14, 137, 1994.
267. 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, 3410, 1990.
268. Wagner, E., Curiel, D., and Gotten, M., Delivery of drugs, proteins and genes into cells using transferrin as a ligand for receptor-mediated endocytosis, Adv. Drug Del. Rev., 14, 113, 1994.
269. Zenke, M., Steinlein, P., Wagner, E., Cotten, M., Beug, H., and Birnstiel, M.L., Receptormediated endocytosis of transferrin-poly cation conjugates: An efficient way to introduce DNA into hematopoietic cells, Proc. Natl. Acad. Sci. USA, 87, 3655, 1990.
270. Harris, C.E., Agarwal, S., Hu, P., Wagner, E., and Curiel, D.T., Receptor-mediated gene transfer to airway epithelial cells in primary culture, Am. J. Respir. Cell Mol. Biol., 9, 441, 1993.
271. Rojanasakul, Y., Wang, L. Y., Malanga, C. J., Ma., J. K. H., and Liaw, J., Targeted gene delivery to alveolar macrophages via Fc receptor-mediated endocytosis, Pharm. Res., 11, 1731, 1994.
272. Gottschalk, S., Gristiano, R. J., Smith, L. C., and Woo, S. L. C., Folate receptor mediated DNA delivery into tumor cells: Potosomal disruption results in enhanced gene expression, Gene Ther., 1, 185, 1993.
273. Curiel, D. T., Agarwal, S., Wagner, E., and Gotten, M., Adenovirus enhancement of transferrin-polylysine-mediated gene delivery, Proc. Natl. Acad. Sci. USA, 88, 8850, 1991.
274. Guriel, D. T., Wagner, E., Gotten, M., Birnstiel, M. L., Agarwal, S., Li, C. M., Loechel, S., and Hu, P. C., High-efficiency gene transfer mediated by adenovirus coupled to DNA-polylysine complexes, Hum. Gene Ther., 3, 147, 1992.
275. Cotten, M., Wagner, E., Zatloukal, K., Phillips, S., Curiel, D. T., and Birnstiel, M. L., High-efficiency receptor-mediated delivery of small and large 48 kilobase gene constructs using the endosome-disruption activity of defective or chemically inactivated adenovirus particles, Proc. Natl. Acad. Sci. USA, 89, 6094, 1992.
276. Cotten, M., Wagner, E., Zatloukal, K., and Birnstiel, M.L., Chicken adenovirus (CELO virus) particles augment receptor-mediated DNA delivery to mammalian cells and yield exceptional levels of stable transformants, J. Virol., 67, 3777, 1993a.
277. Wagner, E., Zatloukal, K., Cotten, M., Kirlappos, H., Mechtler, K., Guriel, D. T., and Birnstiel, M. L., Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes, Proc. Natl. Acad. Sci. USA, 89, 6099, 1992.
278. Cristiano, R. J., Smith, L. C., Kay, M. A., Brinkley, B. R., and Woo, S. L. C., Hepatic gene therapy: Efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus/DNA complex, Proc. Natl. Acad. Sci. USA, 90, 11548, 1993.
279. Cristiano, R.J., Smith, L. C., and Woo, S. L., Hepatic gene therapy: Adenovirus enhancement of receptor-mediated gene delivery and expression in primary hepatocytes, Proc. Natl. Acad. Sci. USA, 90, 2122, 1993.
280. Wu, G. Y., Zhan, P., Sze, L. L., Rosenberg, A. R., and Wu, C. H., Incorporation of adenovinis into a ligand-based DNA carrier system results in retention of original receptor specificity and enhances targeted gene experession, J. Biol. Chem., 269, 11542, 1994.
281. Seth, P., Adenovirus-dependent release of choline from plasma membrane vesicles at an acidic pH is mediated by the penton base protein, J. Virol., 68, 1204, 1994.
282. Prchla, E., Plank, C., Wagner, E., Blaas, D.,and Fuchs, R., Virus-mediated release of endosomal content in vitro: Different behavior of adenovirus and rhinovirus serotype 2, J. Cell Biol, 131, 111, 1995.
283. Wagner, E., Plank, C., Zatloukal, K., Cotten, M., and Birnstiel, M. L., Influenza virus hemagglutinin HA-2 N-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, 7934, 1992.
284. Plank, C., Oberhauser, B., Mechtler, K., Koch, C., and Wagner, E., The influence of endosome-disruptive peptides on gene transfer using synthetic virus-like gene transfer systems, J. Biol. Chem., 269, 12918, 1994.
285. Cotten, M., Wagner, E., and Birnstiel, M. L., Receptor-mediated transport of DNA into eukaryotic cells, in Methods in Enzymology, San Diego: Academic Press, 1993, 618.
286. Wadhwa, M. S., Knoell, D. L., Young, A. P., and Rice, K. G., Targeted gene delivery with a low molecular weight glycopeptide carrier, Bioconj. Chem., 6, 283, 1995.
287. Monsigny, M., Roche, A.-C., Midoux, P., and Mayer, R., Glycoconjugates as carriers for specific delivery of therapeutic drugs and genes, Adv. Drug Del. Rev., 14, 1, 1994.
288. Cristiano, R. J., Smith, L. C., and Woo, S. L., Hepatic gene therapy: Adenovirus enhancement of receptor-mediated gene delivery and expression in primary hepatocytes, Proc. Natl. Acad. Sci. USA, 90, 2122, 1993.
289. Wu, G. Y. and Wu, C. H., Receptor-mediated in vitro gene transformation by a soluble DNA carrier system, J. Biol Chem., 262, 4429, 1987.
290. Chowdhury, N. R., Hays, R. M., Bomminei, V. R., Franki, N., Chowdhury, JR., Wu, C. H., and Wu, G. Y., Microtubular disruption prolongs die expression of human bilirubin uridinediphosphoglucuronate-glucuronosyltransferase-1 gene transferred into Gunn rat livers, J. Biol Chem., 271, 2341, 1996.
291. Yamamoto, M., Hayashi, N., Miyamoto, Y., Takehara, T., Mita, E., Seki, M., Fusamoto, H., and Kamada, T., In vivo transfection of hepatitis C virus complementary DNA into rodent liver by asialoglycoprotein receptor mediated gene delivery, Hepatol., 22, 847, 1995.
292. Gottschalk, S., Sparrow, J. T., Hauer, J., Mims, M. P., Leland, F. E., Woo, S. L. G., and Smith, L. C., A novel DNA/peptide complex for efficient gene transfer and expression in mammalian cells, Gene Ther., 3, 448, 1996.
293. Wisse, E. and De Leeuw, A. M., in Microspheres and Drug Therapy: Pharmaceutical, Immunological and Medical Aspects, Amsterdam: Elsevier Science Publishers B. V., 1984, 1.
294. Duguid, J. G., Li, C. L., 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 peptide-based gene delivery systems for use in plasmid-based gene therapy, Biophys. J., in press, 1998.