Human gene therapy: principles and modern advances

Biotechnology Annual Review

Volumn 3, Issue C, 1997, Pages 59-110

  Reimer, Dorothy L ^a   Bally, Marcel B ^a   Singh, Shiva M ^b  

^a BRITISH COLUMBIA CANCER AGENCY   (Canada)

^b LONDON HEALTH SCIENCES CENTRE   (Canada)

Author keywords

AAT; adeno associated; adenoviral; AIDS; antisense; cancer; CF; FA; FH; gene therapy; gene transfer; herpesvirus; human; lipid based carriers; MD; microencapsulation; oligonucleotides; PKU; receptor mediated; retroviral; SCID; vectors; viral

Indexed keywords

EID: 0009577851     PISSN: 13872656     EISSN: None     Source Type: Book Series    
DOI: 10.1016/S1387-2656(08)70030-9     Document Type: Article

References (305)

1. Capecchi M.R. High efficiency transformation by direct microinjection of DNA into cultured mammalian cells. Cell 22 (1980) 479-488
2. Gordon J.W., Scangos G.A., Plotkin D.J., Barbosa J.A., and Ruddle F.H. Genetic transformation of mouse embryos by microinjection of purified DNA. Proc Natl Acad Sci 77 (1980) 7380-7384
3. Brinster R.L., Chen H.Y., Trumbauer M.E., Yagle M.K., and Palmiter R.D. Factors affecting the efficiency of introducing foreign DNA into mice by microinjecting eggs. Proc Natl Acad Sci 82 (1985) 4438-4442
4. Hansen E., Fernandes K., Goldspink G., Butterworth P., Umeda P.K., and Chang K.C. Strong expression of foreign genes following direct injection into fish muscle. FEBS Lett 290 (1991) 73-76
5. 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 (1991) 474-485
6. Wolff J.A., Malone R.W., Williams P., Chong W., Ascadi G., Jani A., and Felgner P. Direct gene transfer into mouse muscle in vivo. Science 247 (1990) 1465-1468
7. Davis H.L., Demeneix B.A., Quantin B., Coulombe Y., and Whalen R.G. Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal muscle. Hum Gene Ther 4 (1993) 733-740
8. Manthorpe M., Cornefert-Jensen F., Hartikka J., Feigner J., Rundell A., Margalith M., and Dwarki V. Gene therapy by intramuscular injection of plasmid DNA: studies on firefly luciferase gene expression in mice. Hum Gene Ther 4 (1993) 419-431
9. 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 (1992) 21-33
10. Ascadi G., Dickson G., Love D., Jani A., Gurusinghe A., Walsh F.S., Wolff J.A., and Davies K.E. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 352 (1991) 815-818
11. Wolff J.A., Dowry M.E., Jiao S., Repetto G., Berg R.K., Ludtke J.J., Williams P., and Slatutterbak D.B. Expression of naked plasmids by cultured myotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle. J Cell Sci 103 (1992) 1249-1259
12. Ulmer J.B., Donnelly J.J., Parker S.E., Rhodes G.H., Feigner P.L., Dwarki V.J., Gromkowski S.H., Deck R.R., DeWitt C.M., and Friedman A. Heterologous protection against influenza by injection of DNA encoding a viral protein. Science 259 (1993) 1745-1749
13. Sikes M., O'Malley Jr. B.W., Finegold M.J., and Ledley F.D. In vivo gene transfer into rabbit thyroid follicular cells by direct DNA injection. Hum Gene Ther 5 (1994) 837-844
14. Hickman M.A., Malone R.W., Lehmann K., Sih T.R., Knoell D., Szoka Jr. F.C., Walzem R., Carlson D.M., and Powell J.S. Gene expression following direct injection of DNA into liver. Hum Gene Ther 5 (1994) 1477-1483
15. Fraley R., and Papahadjopoulos D. New generation liposomes: The engineering of an efficient vehicle for intracellular delivery of nucleic acids. Trends Biochem Sci 6 (1981) 77-80
16. Straubinger R.M., and Papahadjopoulos D. Liposomes as carriers for intracellular delivery of nucleic acids. Meth Enzymol 101 (1983) 512-527
17. Feigner 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 84 (1987) 7413-7417
18. Pinnaduwage R., Schmitt L., and Huang L. Use of a quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells. Biochim Biophys Acta 985 (1989) 33-37
19. 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
20. Zhou X.H., Klibanov A.L., and Huang L. Lipophilic polylysines mediate efficient DNA transfections in mammalian cells. Biochim Biophys Acta 1065 (1991) 8-14
21. 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 (1992) 239-246
22. Barthel F., Remy J.S., Loeffler J.P., and Behr J.P. Gene transfer optimization with lipospermine-coated DNA. DNA Cell Biol 12 (1993) 553-560
23. Zhou X., and Huang L. DNA transfection mediated by cationic liposomes containing lipopofylysine: Characterization and mechanism of action. Biochim Biophys Acta 1189 (1994) 195-203
24. Smith J.G., Walzem R.L., and German J.B. Liposomes as agents of DNA transfer. Biochim Biophys Acta 1154 (1993) 327-340
25. 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 (1989) 429-438
26. Liu D., Huang L., Moore M.A., Anantharamaiah G.M., and Segrest J.P. Interactions of serum proteins with small unilamellar liposomes composed of dioleoylphosphatidylethanolamine and oleic acid: high density lipoprotein, apolipoprotein A1 and amphipathic peptides stabilize liposomes. Biochemistry 29 (1990) 3637-3643
27. Cullis P.R., Hope M.J., and Tilcock C.P. Lipid polymorphism and the roles of lipids in membranes. Chem Phys Lipid 40 (1986) 127-144
28. Rose J.K., Buonocore L., and Whitt M.A. A new cationic liposome reagent mediating nearly quantitative transfection of animal cells. Biotechniques 10 (1991) 520-525
29. Stewart M.J., Plautz G.E., Buono L.D., Yang Z.Y., Xu L., Gao X., Huang L., Nabel E.G., and Nable G.J. Gene transfer in vivo with DNA-liposome complexes: Safety and acute toxicity in mice. Hum Gene Ther 3 (1992) 267-275
30. Wasan E.K., Reimer D.L., and Bally M.B. Plasmid DNA is protected against ultrasonic cavitation-induced damage when complexed to cationic liposomes. J Pharm Sci 85 (1996) 427-433
31. Felgner J.H., Kumar R., Sridhar C.N., Wheeler C.J., Tsai Y.J., Border R., Ramsey P., Martin M., and Feigner P.L. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem 269 (1994) 2550-2561
32. Legendre J.Y., and Szoka Jr. F.C. Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: comparison with cationic liposomes. Pharmacol Res 9 (1992) 1235-1242
33. 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 Molec Biol 7 (1992) 406-413
34. Nicolau C., and Cudd A. Liposomes as carriers of DNA. Crit Rev Ther Drug Carrier Sys 6 (1989) 239-271
35. Farhood H., Gao X., Son K., Yang Y.Y., Lazo J.S., Huang L., Barsoum J., Bottega R., and Epand R.M. Cationic liposomes for direct gene transfer in therapy of cancer and other diseases. Ann NY Acad Sci 716 (1994) 23-34
36. Reimer D.L., Zhang Y., Kong S., Wheeler J.J., Graham R.W., and Bally M.B. Formation of a hydrophobic complexes between cationic lipids and plasmids. Biochemistry 34 (1995) 12877-12883
37. Wong F.M.R., Reimer D.L., and Bally M.B. Cationic lipid binding to DNA: characterization of complex formation. Biochemistry 35 (1996) 5756-5763
38. Zhang Y.-P., Reimer D.L., Zhang G., Lee P.H., and Bally M.B. Self-assembling DNA-lipid particles for gene transfer. J Pharm Sci 14 (1996) 190-196
39. Haensler J., and Szoka Jr. F.C. Polyamidoamine cascade polymers mediate efficient transfection of cells in culture. Bioconjug Chem 4 (1993) 372-379
40. Tomalia D.A. Dendrimer molecules. Sci Am 272 (1995) 62-66
41. Wu G.Y., and Wu C.H. Receptor-mediated gene delivery and expression in vivo. J Biol Chem 263 (1988) 14261-14264
42. Zenke M., Steinlein P., Wagner E., Cotten M., Beug H., and Birnstiel M.L. Receptor-mediated endo-cytosis of transferrin-polycation conjugates: an efficient way to introduce DNA into hematopoetic cells. Proc Natl Acad Sci 87 (1990) 3655-3659
43. Wagner E., Cotten M., Foisner R., and Bernsteil M.L. Transferrin-polycation-DNA complexes: the effect of polycations on the structure of the complex and DNA delivery to cells. Proc Natl Acad Sci 88 (1991) 4255-4259
44. Wu G.Y., Wilson J.M., and Wu C.H. Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J Biol Chem 264 (1989) 16985-16987
45. 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 asialoglycoprotein receptor-mediated endocytosis in vivo. Prolonged persistence in cytoplasmid vesicles after partial hepatectomy. J Biol Chem 268 (1993) 11265-11271
46. 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 by receptor-targeted uptake. Proc Natl Acad Sci 91 (1994) 4086-4090
47. Michael S.I., and Curiel D.T. Strategies to achieve targeted gene delivery via the receptor-mediated endocytosis pathway. Gene Ther 1 (1994) 223-232
48. Huebers H.A., and Finch C.A. The physiology of transferrin and transferrin receptors. Physiol Rev 67 (1987) 520-582
49. Wagner E., Zenke M., Cotten M., Beug H., and Birnsteil M.L. Transferrin-polycation-conjugates as carriers for DNA uptake into cells. Proc Natl Acad Sci 87 (1990) 3410-3414
50. 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 Molec Biol 9 (1993) 441-447
51. 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 91 (1994) 2547-2551
52. 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. Bioconjug Chem 3 (1992) 323-327
53. Rosenkrantz A.A., Yachmenev S.V., Jans D.A., Serebryakova N.V., Marav'ev V.I., Peters R., and Sobolev A.S. Receptor-mediated endocytosis and nuclear transport of a transfecting DNA construct. Exp Cell Res 199 (1992) 323-329
54. Chen J., Gamou S., Takayanagi A., and Shimizu N. A novel gene delivery system using EGF receptor-mediated endocytosis. FEBS Lett 338 (1994) 167-169
55. Barra R.K., Wang-Johanning F., Wagner E., Garver R.I., and Curiel D.T. Receptor-mediated gene delivery employing lectin-binding specificity. Gene Ther 1 (1994) 255-260
56. 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 (1994) 1204-1206
57. Garoff H., Wilschut J., Liljestrom P., Wahylberg J.M., Bron R., Suomalainen M., Smyth J., Salminen A., Barth B.U., and Zhao H. Assembly and entry mechanism of Simliki Forest Virus. Arch Virol 9 Suppl (1994) 329-338
58. 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 90 (1993) 2122-2126
59. Gottschalk S., Cristiano R.J., Smith L.C., and Woo S.L.C. Folate receptor-mediated DNA delivery into tumor cells: protosomal disruption results in enhanced gene expression. Gene Ther 1 (1993) 185-191
60. Wagner E., Zatloukal K., Cotten M., Kirlappos H., Mechtler K., Curiel D.T., and Birnsteil M.L. Coupling of adenovirus to transferrin-porylysine-DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc Natl Acad Sci. 89 (1992) 6099-6103
61. Cristiano R.J., Smith L.C., Kay M.A., Brinkley B.R., and Woo S.L. Hepatic gene therapy: efficient gene deliver and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex. Proc Natl Acad Sci 90 (1993) 11548-11552
62. Wagner E., Plank C., Zatloukal K., Cotten M., and Birnsteil M.L. Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: Toward a synthetic virus-like gene-transfer vehicle. Proc Natl Acad Sci. 89 (1992) 7934-7938
63. Furth P.A., Shamay A., Wall R.J., and Hennighausen L. Gene transfer into somatic tissues by jet injection. Anal Biochem 20 (1992) 365-368
64. Davis H.L., Michel M.L., Mancini M., Schleef M., and Whalen R.G. Direct gene transfer in skeletal muscle: plasmid DNA-based immunization against the hepatitis B virus surface antigen. Vaccine 12 (1994) 1503-1509
65. Vahlsing H.L., Yankauckas M.A., Sawdey M., Gromkowsky S.H., and Manthorpe M. Immunization with plasmid DNA using a pneumatic gun. J Immunol Meth 175 (1994) 11-22
66. Yang N.S., Burkholder J., Roberts B., Martinell B., and McCabe D. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci 87 (1990) 9568-9572
67. Cheng L., Ziegelhoffer P.R., and Yang N.S. In vivo promoter activity and transgene expression in mammalian somatic tissues evaluated by using particle bombardment. Proc Natl Acad Sci 90 (1993) 4455-4459
68. Thompson T.A., Gould M.N., Burkholder J.K., and Yang N.S. Transient promoter activity in primary rat mammary epithelial cells evaluated using particle bombardment gene transfer. In Vitro Cell Dev Biol 29A (1993) 165-170
69. Burkholder J.K., Decker J., and Yang N.S. Rapid transgene expression in lymphocyte and macrophage primary cultures after particle bombardment-mediated gene transfer. J Immunol Meth 165 (1993) 149-156
70. Williams R.S., Johnston S.A., Reidy M., Devit M.J., Mclligot S.G., and Sanford J.C. Introduction of foreign genes into tissues of living mice by DNA-coated microprojectiles. Proc Natl Acad Sci 88 (1991) 2726-2730
71. Reilly E.B., Antognetti G., Weslowski Jr. J.S., and Sakorafas P. The use of microcapsules for high density growth of human tumour infiltrating lymphocytes and other immune reactive T cells. J Immunol Meth 126 (1990) 273-279
72. Chang P.L., Shen N., and Westcott A.J. Delivery of recombinant gene products with microencapsulated cells in vivo. Hum Gene Ther 4 (1993) 433-440
73. Liu H.W., Ofosu F.A., and Chang P.L. Expression of human factor IX by microencapsulated recombinant fibroblasts. Hum Gene Ther 4 (1993) 291-301
74. Hughes M., Vassilakos A., Andrews D.W., Hortelamo G., Belmont J.W., and Chang P.L. Delivery of a secretable adenosine deaminase through microcapsules-a novel approach to somatic gene therapy. Hum Gene Ther 5 (1994) 1445-1455
75. al-Hendy A., Hortelano G., Tannenbaum G.S., and Chang P.L. Correction of growth defect in dwarf mice with nonautologous microencapsulated myoblasts-an alternate approach to somatic gene therapy. Hum Gene Ther 6 (1995) 165-175
76. Choppin P.W., and Scheid A. The role of viral glycoproteins in adsorption, penetration, and pathogenicity of viruses. Rev Infect Dis 2 (1980) 40-61
77. Kielian M., and Jungerwirth S. Mechanisms of enveloped virus entry into cells. Molec Biol Med 7 (1990) 17-31
78. Miller N., and Vile R. Targeted vectors for gene therapy. FASEB J 9 (1995) 190-199
79. Baichwal V.R., and Sugden B. Vectors for gene transfer derived from animal DNA viruses: Transient and stable expression of transferred genes. In: Kucherlapati R. (Ed). Gene Transfer (1986), Plenum Press, New York 117
80. Crystal R. Transfer of genes to human: Early lessons and obstacles to success. Science 270 (1995) 404-410
81. Miller A.D. Retroviral vectors. Curr Top Micro Immunol 158 (1992) 1-24
82. Bandyopadhyay P.K., and Temin H.M. Expression of complete chicken thymidine kinase gene inserted in a retrovirus vector. Molec Cell Biol 4 (1984) 749-754
83. Mann R., and Baltimore D. Varying the position of a retrovirus packaging sequence results in the encapsidation of both unspliced and spliced RNAs. J Virol 54 (1985) 401-407
84. Page K.A., Landau N.R., and Littman D.R. Construction and use of a human immunodeficiency virus vector for analysis of virus infectivity. J Virol 64 (1990) 5270-5276
85. + cells by a recombinant human immunodeficiency virus retroviral vector. J Clin Invest 88 (1991) 1043-1047
86. Buchschacher Jr. G.L., and Panganiban A.T. Human immunodeficiency virus vectors for inducible expression of foreign genes. J Virol 66 (1992) 2731-2739
87. Anderson W.F. Human gene therapy. Science 256 (1992) 808-813
88. Miller A.D. Human gene therapy comes of age. Nature 357 (1992) 455-460
89. Miller A.D., and Rosman G.J. Improved retroviral vectors for gene transfer and expression. Biotechniques 7 (1989) 980-990
90. McLauchlin J.R., Cornetta K., Eglitis M.A., and Anderson W.F. Retroviral-mediated gene transfer. Prog Nucl Acid Res 38 (1990) 91-135
91. Mulligan R.C. The basic science of gene therapy. Science 260 (1993) 926-932
92. Temin H.M. Safety considerations in somatic gene therapy of human diseases with retroviral vectors. Hum Gene Ther 1 (1990) 111-123
93. Miller D.G., Adam M.A., and Miller A.D. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Molec Cell Biol 10 (1990) 4239-4242
94. Horwitz M.S. Adenoviruses. In: Fields B.N., and Knipe D.M. (Eds). Fields Virology. 2nd edn. (1990), Raven Press, New York 1723-1740
95. Berkner K.L. Development of adenovirus vectors for the expression of heterologous genes. Biotechniques 6 (1988) 616-629
96. Kozarsky K.F., and Wilson J.M. Gene therapy: adenovirus vectors. Curr Opin Genet Devel 3 (1993) 499-503
97. Ginsberg H.S., Moldawar L.L., Sehgal P.B., Redington M., Kilian P.L., Chanock R.M., and Prince G.P. A mouse model for investigating the molecular pathogenesis of adenovirus pneumonia. Proc Natl Acad Sci 88 (1991) 1651-1655
98. Wickham T.J., Mathias P., Cheresh D.A., and Nemerow G.R. Integrins alpha v beta 3 and alpha v beta 5 promote adenovirus internalization but not virus attachment. Cell 73 (1993) 309-319
99. Berkner K.L. Expression of heterologous sequences in adenoviral vectors. Curr Top Microbiol Immunol 158 (1992) 39-66
100. Rosenfeld M.A., Yoshimura K., Trapnell B.C., Yoneyama K., Rosenthal E.R., Dalemans W., Fukayama M., Bargon J., Stier L.E., and Stratford-Perricaudet L. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68 (1992) 143-155
101. Yoshimura K., Rosenfeld M.A., Nakamura H., Scherer E.M., Pavirani A., Lecocq J.-P., and Crystal R.G. Expression of the human cystic transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer. Nucl Acid Res. 20 (1992) 3233-3240
102. Welsh MJ RAC Report 9212-036, 1995.
103. Rosenfeld M.A., Siegfried W., Yoshimura K., Yoneyama K., Fukayama M., Stier L.E., Paakko P.K., Gilardi P., Stratford-Perricaudet L., and Perricaudet M. Adenovirus-mediated transfer of a recombinant α-1 antitrypsin gene to the lung epithelium in vivo. Science 252 (1991) 431-434
104. Yang Y., Nunes F.A., Berencsi K., Furth E.E., Gonczol E., and Wilson J.M. Cellular immunity to viral antigens limits E1 deleted adenoviruses for gene therapy. Proc Natl Acad Sci 91 (1994) 4407-4411
105. Yang Y., Li Q., Erte H.C.J., and Wilson J.M. Cellular and humoral responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 69 (1995) 2004-2015
106. Samulski R.J., Zhu X., Xiao X., Brook J.D., Housman D.E., Epstein N., and Hunter L.A. Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 10 (1991) 3941-3950
107. 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 adenoassociated virus vector. Proc Natl Acad Sci 90 (1993) 10613-10617
108. Carter B.J. The growth cycle of adenoassociated virus. In: Tjissen P. (Ed). Handbook of Parvoviruses 1 (1990), CRC Press, Boca Raton, Florida 155-168
109. Samulski R.J. Adeno-associated virus: integration at a specific chromosomal locus. Curr Opin Genet Devel 3 (1993) 74-80
110. Kearns W., Afione S., and Cutting G. AAV-CFTR vectors integrate at multiple chromosomal sites in a CF bronchial epithelial cell line. Pediatr Pulmonol 9S (1993) 242
111. Freese A., Geller A.I., and Neve R. HSV-1 vector mediated neuronal gene delivery. Strategies for molecular neuroscience and neurology. Biochem Pharmacol 40 (1990) 2189-2199
112. Fink D.J., Sternberg L.R., Weber P.C., Mata M., Goins W.F., and Glorioso J.C. In vivo expression of beta-galactosidase in hippocampal neurons by HSV-mediated gene transfer. Hum Gene Ther 3 (1992) 11-19
113. Margolskee R.F. Epstein-Barr virus based expression vectors. Curr Top Microbiol Immunol 158 (1992) 67-95
114. Suhr S.T., and Gage F.H. Gene therapy for neurologic disease. Neurol Rev 50 (1993) 1252-1268
115. Breakefield X.O., and DeLuca N.A. Herpes simplex virus for gene delivery to neurons. New Biol 3 (1991) 203-218
116. Roizman B., and Sears A.E. Herpes viruses and their replication. In: Fields B.N., and Knipe D.M. (Eds). Virology. 2nd edn. (1990), Raven Press, New York 1795-1842
117. Barker D.E., and Roizman B. Identification of three genes nonessential for growth in cell culture near the right terminus of the unique sequences of long component of herpes simplex virus I. J Virol. 177 (1990) 684-691
118. Longnecker R., Roizman B., and Meignier B. Herpes simplex viruses as vectors: Properties of a prototype vaccine strain suitable for use as a vector. In: Gluzman Y., and Hughes S.H. (Eds). Viral Vectors (1988), Cold Spring Harbor Lab. Press, Cold Spring Harbor 68
119. Tiberghien P. Use of suicide genes in gene therapy. J Leuk Biol 56 (1994) 203-209
120. Oldfield E.H., Ram Z., Culver K.W., Blaese R.M., and DeVroom H.L. Gene therapy for the treatment of brain tumors using intra-tumoral transduction with the thymidine kinase gene and intravenous ganciclovir. Hum Gene Ther 4 (1993) 39-69
121. Freeman S.M., McCune C., Angel C., Abraham G.N., and Abboud C.N. Treatment of ovarian cancer using HSV-TK gene modified vaccine-regulatory issues. Hum Gene Ther 3 (1992) 342-349
122. Dobson A.T., Margolis T.P., Sedarati F., Stevens J.G., and Feldman L.T. A latent nonpathogenic HSV-1-derived vector stably expresses β-galactosidase in mouse neurons. Neuron 5 (1990) 353-360
123. Wolfe J.H., Deshmane S.L., and Fraser N.W. Herpesvirus vector gene transfer and expression of B-glucouronidase in the CNS of MPS VII mice. Nature Genet 1 (1992) 379-384
124. Orkin SH, Motulsky AG, Report and recommendations of the panel to assess the NIH investment in research on gene therapy. 1995; http://www.nih.gov/news/panelrep.html.
125. Giblett E.R., Anderson J.E., Cohen F., Pollara B., and Meuwissen H.J. Adenosine deaminase deficiency in two patients with severely impaired cellular immunity. Lancet 2 (1972) 1067-1069
126. Thompson L.F., and Seegmiller J.E. Adenosine deaminase deficiency and severe combined immunodeficiency disease. In: Meister A. (Ed). Advances in Enzymology 51 (1980), Academic Press, New York 156-210
127. Cohen A., Hirschhorn R., Horowitz S.D., Rubinstein A., Polmar S.H., Hong R., and Martin Jr. D.W. Deoxyadenosine triphosphate as a potentially toxic metabolite in adenosine deaminase deficiency. Proc Natl Acad Sci 75 (1978) 472-476
128. Hirschhorn R., Martiniuk F., Roegner-Maniscalco V., Ellenbogen A., Perignon J.L., and Jenkins T. Genetic heterogeneity in partial adenosine deaminase deficiency. J Clin Invest 71 (1983) 1887-1892
129. Akeson A.L., Wiginton D.A., and Hutton J.J. Normal and mutant adenosine deaminase genes. J Cell Biochem 39 (1989) 217-228
130. Santisteban I., Arredonto-Vega F.X., Kelly S., Mary A., Fischer A., Hummell D.S., Lawton A., Sorenson R.U., Steihm E.R., and Uribe L. Novel splicing, missence and deletion mutations in seven adenosine deaminase deficient patients with late/delayed onset combined immunodeficiency disease. J Clin Invest. 92 (1993) 2291-2302
131. Reisner Y., Kapoor N., Kirkpatrick D., Pollack M.S., Cunningham-Rundles S., Dupont B., Hoder M.Z., Good R.A., and O'Reilly R.J. Transplantation for severe combined immunodeficiency with HLA-A, B, D, DR incompatible parental marrow cells fractionated by soybean agglutinin and sheep red blood cells. Blood. 61 (1983) 341-348
132. O'Reilly R.J., Keever C.A., Small T.N., and Brochstein J. The use of HLA non-identical T-cell-depleted marrow transplants for correction of severe combined immunodeficiency disease. Immunodef Rev 1 (1989) 273-309
133. Hershfield M.S., Buckley R.H., Greenberg M.L., Melton A.L., Schiff R., Hatem C., Kurtzberg J., Markert M.L., Kobayashi R.H., and Kobayashi A.L. Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase. N Engl J Med 316 (1987) 589-596
134. Levy Y., Hershfield M., Fernandez-Mejia C., Polmar S., Scudiery D., Berger M., and Sorensen R.U. Adenosine deaminase deficiency with late onset of recurrent infections: Response to treatment with polyethylene glycol-modified adenosine deaminase. J Pediatr 113 (1988) 312-317
135. Blaese M.R., Culver K.W., Miller D.A., 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 (1995) 475-480
136. Hershfield M.S. The role of polyethylene glycol adenosine deaminase in the evolution of therapy for adenosine deaminase deficiency. In: Gupta S., and Griscelli C. (Eds). New Concepts in Immunodeficiency Diseases (1993), Wiley, Chichester 417-426
137. Bordignon C., Notarangelo L.D., Nobili N., Ferrari G., Casorati G., Panina P., Mazzolari E., Maggioni D., Rossi C., Servida P., Ugazio A.G., and Mavilio F. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Science 270 (1995) 470-474
138. Kohn D.B., Anderson W.F., and Blease R.M. Gene therapy for genetic diseases. Cancer Invest 7 (1989) 179-192
139. Kohn D.B., Mitsuya A., Ballow M., Selegue J.E., Barankiewicz J., Cohen A., Gelfand E., Anderson W.F., and Blaese R.M. Establishment and characterization of adenosine deaminase deficient human T cells. J Immunol 142 (1989) 3971-3979
140. Kaptein L.C.M., Einerhand M.P.W., Braakman E., Valerio D., and von Beusechem V.W. Bone marrowgene therapy for adenosine deaminase deficiency. Immunodeficiency 4 (1993) 335-345
141. Moritz T., Keller D.C., and Williams D.A. Human cord blood cells as targets for gene transfer: Potential use in genetic therapies of severe combined immunodeficiency disease. J Exp Med 178 (1993) 529-536
142. Boat T.F., Welsh M.J., and Beaudet A.L. Cystic fibrosis. In: Scriver C.R., Beaudet A.L., Sly W.S., and Valle D. (Eds). The Metabolic Basis of Inherited Disease (1989), McGraw Hill, New York 2649-2680
143. Rommens J.M., Iannuzzi M.C., Kerem B., Drumm M.L., Melmer G., Dean M., Rozmahel R., Cole J.L., Kennedy D., and Hidaka N. Identification of cystic fibrosis gene: chromosome walking and jumping. Science 245 (1989) 1059-1065
144. Riordan J.R. The cystic fibrosis transmembrane conductance regulator. Ann Rev Physiol 55 (1993) 609-630
145. Welsh M.J., and Smith A.E. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell 73 (1993) 1251-1254
146. Colledge W.H., and Evans M.J. Cystic fibrosis gene therapy. Br Med Bull 51 (1995) 82-90
147. Riordan J.R., Rommens J.M., Kerem B., Alon N., Rozmahel R., Grzelczak Z., Zielenski J., Lok S., Plavsic N., Chou J.-L., Drumm M.L., Iannuzzi M.C., Collins F.S., and Tsui L.-C. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 245 (1989) 1066-1073
148. Gregory R.J., Cheng S.H., Rich D.P., Marshall J., Paul S., Hehir K., Ostedgaard L., Klinger K.W., Welsh M.J., and Smith A.E. Expression and characterization of the cystic fibrosis transmembrane conductance regulator. Nature 347 (1990) 382-386
149. Cheng S.H., Gregory R.J., Marshall J., Paul S., Souza D.W., White G.A., O'Riordan C.R., and Smith A.E. Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis. Cell 63 (1990) 827-834
150. Dean M., and Santis G. Heterogeneity in the severity of cystic fibrosis and the role of CFTR gene mutations. Hum Genet 93 (1994) 364-368
151. Tizzano E.F., and Buchwald M. CFTR expression and organ damage in cystic fibrosis. Ann Int Med 123 (1995) 305-308
152. Rich D.P., Anderson M.P., Gregory R.J., Cheng S.H., Paul S., Jefferson D.M., Smith A.E., McCann J.D., Klinger K.W., Smith A.E., and Welsh M.J. Expression of the cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature. 347 (1990) 358-363
153. Olsen J.C., Johnson L.G., Stutts M.J., Sarkadi B., Vankaskas J.R., Swanstrum R., and Boucher R.C. Correction of the apical membrane chloride permeability defect in polarized cystic fibrosis airway epithelia following retroviral-mediated gene transfer. Hum Gene Ther. 3 (1992) 253-266
154. Weibel E.R. Design of airways and blood vessels considered as branching trees. In: Crystal R.G., and West J.B. (Eds). The Lung Scientific Foundations. The Lung Scientific Foundation (1991), Raven Press, New York 711-720
155. Mastrangeli A., Danel C., Rosenfeld M.A., Stratford-Perricaudet L., Perricaudet M., Paviriani A., Lecocq J.P., and Crystal R.G. Diversity of airway epithelial cell targets for in vivo recombinant adenovirus-mediated gene transfer. J Clin Invest 91 (1993) 225-234
156. McLachlin G., Davidson D.J., Stevenson B.J., Dickinson P., Davidson-Smith H., Dorin J.R., and Porteous D.J. Evaluation in vitro and in vivo of cationic liposome-expression construct complexes for cystic fibrosis gene therapy. Gene Ther 2 (1995) 614-622
157. Curiel D.T., Agarwal S., Romer M.U., Wagner E., Cotten M., Birnstiel M.L., and Boucher R.C. Gene transfer to respiratory epithelial cells via the receptor-mediated endocytosis pathway. Am J Resp Cell Molec Biol 6 (1992) 247-252
158. Rich D.P., Couture L.A., Cardoza L.M., Guiggio V.M., Amentano D., Espino P.C., Hehir K., Welsh M.J., Smith A.E., and Gregory R.J. Development and analysis of recombinant adenoviruses for gene therapy of cystic fibrosis. Hum Gene Ther 4 (1993) 461-476
159. Flotte T.R., Solow R., Owens R.A., Afione S., Zeitlin P.L., and Carter B.J. Gene expression from adenoassociated virus vectors in airway epithelial cells. Am J Respir Cell Molec Biol 7 (1992) 349-356
160. Zabner J., Couture L.A., Gregory R.J., Graham S.M., Smith A.E., and Welsh M.J. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75 (1993) 207-216
161. Brigham K.L., Meyrick B., Christman B., Magnuson M., King G., and Berry Jr. L.C. In vivo transfection of murine lung with a functioning prokaryotic gene using a liposome vehicle. Am J Med Sci 298 (1989) 278-281
162. Stribling R., Brunette E., Liggitt D., Gaensler K., and Debs R. Aerosol gene delivery in vivo. Proc Natl Acad Sci 89 (1992) 11277-11281
163. Alton E.W., Middleton P.G., Caplen N.J., Smith S.N., Steel D.M., Munkonge F.M., Jeffrey P.K., Geddes D.M., Hart S.L., and Williamson R. Non-invasive liposome mediated gene delivery can correct the ion transport defect in the cystic fibrosis mutant mice. Nature Genet 5 (1993) 135-142
164. Hyde S.C., Gill D.R., Higgins C.F., Trezise A.E., MacVinish U., 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
165. Caplen N.J., Alton E.W., Middleton P.G., Dorin J.R., Stevenson B.J., Gao X., Durham S.R., Jeffrey P.K., Hodson M.E., and Coutelle C. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med 1 (1995) 39-46
166. Middleton P.G., Caplen N.J., Gao X., Huang L., Dorin J.R., Porteous D.J., Williamson R., Geddes D.M., and Alton E.W.F.W. The electrophysiological effects of in vivo liposome mediated CFTR delivery to the nasal epithelium. Pediatr Pulmonol Suppl 10 (1994) 229-230
167. Farhood H., Gao X., Son K., Yang Y.Y., Lazo J.S., Huang L., Barsoum J., Bottega R., and Epand R.M. Cationic liposomes for direct gene transfer in therapy of cancer and other diseases. Ann NY Acad Sci 716 (1994) 23-35
168. 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
169. Frenkel E.P. Gaucher disease; a heterogeneous clinical complex for which effective enzyme replacement has come of age. Am J Med Sci 305 (1993) 331-344
170. Brady R.O., Barton N.W., and Grabowski G.A. The role of neurogenetics in Gaucher disease. Arch Neurol 50 (1993) 1212-1224
171. Barton N.W., Furbish F.S., Murray G.J., Garfield M., and Brady R.O. Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad Sci 87 (1990) 1913-1916
172. Beutler E., Kay A., Saven A., Garver P., Thurston D., Dawson A., and Rosenbloom B. Enzyme replacement therapy for Gaucher disease. Blood 78 (1991) 1183-1189
173. Xu L., Stahl S.K., Dave H.P., Schiffmann R., Correll P.H., Kessler S., and Karlsson S. Correction of the enzyme deficiency in hematopoetic cells of Gaucher patients using a clinically acceptable retroviral supernatant transduction protocol. Exp Hematol 22 (1994) 223-230
174. Kerr W.G., and Mule J.J. Gene therapy: current status and future prospects. J Leuk Biol 56 (1994) 211-214
175. Emery A.E.H. Duchenne Muscular Dystrophy. 2nd edn (1988), University Press, New York 1-317
176. Griggs C.R., Moxley R.T., Mendell J.R., Fenichel G.M., Brooke M.H., and Pestronk A. Prednisone in Duchenne dystrophy. A randomized controlled trial defining the time course and dose response. Arch Neurol 48 (1991) 383-388
177. Koenig M., Hoffman E.P., Bertelson C.J., Monaco A.P., Feener C., and Kunkel L.M. Complete cloning of the DMD cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 50 (1987) 509-517
178. Ahn A.H., and Kunkel L.M. The structural and functional diversity of dystrophin. Nature Genet 3 (1993) 283-291
179. Oldfors A., Eriksson B.O., Kyllerman M., Martinsson R., and Wahlstrom J. Dilated cardiomyopathy and the dystrophin gene. Br Heart J 72 (1994) 344-348
180. Quantin B., Perricaudet L.D., Tajbakakhsh S., and Mandel J.L. Adenovirus as an expression vector in muscle cells in vivo. Proc Natl Acad Sci 87 (1990) 439-443
181. Ragot T., Vincent N., Chafey V., Vigne E., Gildenkrantz H., Couton D., Cartaud J., Briand P., Kaplan J.-C., Perricaudet M., and Kahn A. Efficient adenovirus-mediated transfer of human minidystrophin gene to skeletal muscle of mdx mice. Nature 361 (1993) 647-650
182. Karpati G., and Acsadi G. The principles of gene therapy in Duchenne Muscular Dystrophy. Clin Invest Med 17 (1994) 499-509
183. Scriver C.R., Kaufman S., and Woo S.L.C. The hyperphenylalaninemias. In: Scriver C.R., Beaudet A.L., Sly W.S., and Valle D. (Eds). The Metabolic Basis of Inherited Disease. 6th edn 1 (1989), McGraw Hill, New York 495-534
184. Smith I., Lobascher M.E., Stevenson J.R., Woolf O.H., Schmidt H., Grubel-Kaiser S., and Bickel H. Effect of stopping low-phenylalanine diet on intellectual progress of children with phenylketonuria. Br Med J 2 (1978) 723-726
185. Ledley F.D., Grenett H.E., DiLella A.G., Kwok S.C.M., and Woo S.L.C. Gene transfer and expression of human phenylalanine hydroxylase. Science 228 (1985) 77-79
186. Peng H., Armentano D., Mackenzie-Graham L., Shen R.-F., Darlington G., Ledley F.D., and Woo S.L. Retroviral-mediated gene transfer and expression of human phenylalanine hydroxylase in primary mouse hepatocytes. Proc Natl Acad Sci 85 (1988) 8146-8150
187. Liu T.-J., Kay M.A., Darlington G.J., and Woo S.L.C. Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice. Somat Cell Molec Genet 18 (1992) 89-96
188. Fang B., Eisensmith R.C., Li X.H.C., Finegold M.J., Shedlovsky A., Dove W., and Woo S.L.C. Gene therapy for phenylketonuria: phenotypic correction in a genetically deficient mouse model by adenovirus-mediated hepatic gene transfer. Gene Ther 1 (1994) 247-254
189. Brown M.S., and Goldstein J.L. A receptor-mediated pathway for cholesterol homeostasis. Science 232 (1986) 34-37
190. Goldstein J.L., and Brown M.S. Familial hypercholesterolemia. Lipoprotein and lipid metabolism disorders. In: Scriver C.R., Beaudet A.L., Sly W.S., and Valle D. (Eds). Metabolic Basis of Inherited Disease. 6th edn (1989), McGraw Hill, New York 1215-1250
191. Bilheimer D.W., Goldstein J.L., Grundy S.M., and Starzl T.E. Liver transplantation to provide low density lipoprotein receptors and lower plasma cholesterol in a child with homozygous familial hypercholesterolemia. N Engl J Med. 311 (1984) 1658-1664
192. Thompson G.R., Barbir M., Okabayshi K., Trayner I., and Larkin S. Plasmapharesis in familial hypercholesterolemia. Arteriosclerosis 9 Suppl (1989) I152-I157
193. Gordon B.R., Kelsey S.F., Bilheimer D.W., Brown D.C., Dau P.C., Gotto Jr. A.M., Illingworth D.R., Jones P.H., Leitman S.F., and Prihoda J.S. Treatment of refractory familial hypercholesterolemia by low-density lipoprotein apheresis using an automated dextran sulfate cellulose absorption system. Am J Cardiol. 70 (1992) 1010-1016
194. Weatherall D. Heroic gene therapy. Nature Genet 6 (1994) 325-326
195. 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 liver in a patient with familial hypercholesterolemia. Nature Genet 6 (1994) 335-341
196. Chowdhury J.R., Grossman M., Gupta S., Chowdhury N.R., Baker J.R., and Wilson J.M. Long term improvement of hypercholesterolemia after ex vivo therapy in LDLR-deficient rabbits. Science 254 (1991) 1802-1805
197. Grossman M., Rader D.J., Muller D.W.M., Kolansky D.M., Kozarsky K., Clark III 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 Med 1 (1995) 1148-1154
198. Raper S.E., Grossman M., Rader D.J., Thoene J.G., Clark III B.J., Kolansky D.M., Muller D.W., and Wilson J.M. Safety and feasibility of liver-directed ex vivo gene therapy for homozygous familial hypercholesterolemia. Ann Surg 223 (1996) 116-126
199. Wilson M.A. Medical news and perspectives. J Am Med Assoc 269 (1993) 837-838
200. Ishibashi S., Brown M.S., Goldstein J.L., Gerard R.D., Hammer R.E., and Herz J. Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery. J Clin Invest 92 (1993) 883-893
201. Kozarsky K.F., McKinley D.R., Austin L.L., Raper S.E., Stratford-Perricaudet L.D., and Wilson J.M. In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses. J Biol Chem 269 (1994) 13695-13702
202. Fagerhol M.K., and Laurell C.-B. The Pi system-inherited variants of serum ai antitrypsin. In: Steinberg A.G., and Bearn A.G. (Eds). Progress in Medical Genetics 3 (1970), Grune and Stratton, New York 96-111
203. Crystal R.G. The α 1-antitrypsin gene and its deficiency states. Trends Genet 5 (1989) 411-417
204. Crystal R.G. α 1-antitrypsin deficiency, emphysema and liver disease: genetic basis and strategies for therapy. J Clin Invest 85 (1990) 1343-1352
205. Brantly M., Nukiwa T., and Crystal R.G. Molecular basis of a 1-antitrypsin deficiency. Am J Med 84 (1988) 13-31
206. 1-antitrypsin deficiency detection by direct analysis of the mutation in the gene. Nature 304 (1983) 230-234
207. Hubbard R.C., Sellers D., Czerski L., Stephens L., and Crystal R.G. Biochemical efficacy and safety of monthly augmentation therapy for a 1-antitrypsin deficiency. J Am Med Assoc 260 (1988) 1259-1264
208. Kay M.A., Ponder K.P., and Woo S.L.C. Human gene therapy: present and future. Breast Cancer Res Treat 21 (1992) 83-93
209. 1-antitrypsin causes liver damage in transgenic mice. J Clin Invest 83 (1989) 1183-1190
210. Jaffe H.A., Danel C., Longenecker G., Metzger M., Setoguchi Y., Rosenfeld M.A., Gant T.W., Thorgeirsson S.S., Stratford-Perricaudet L.S., Perricaudet M., Pavirani A., Lecocq J.-P., and Crystal R.G. Adenoviral-mediated in vivo gene transfer and expression in normal rat liver. Nature Genet 1 (1992) 368-371
211. Kay M.A., Baley P., Rothenberg S., Leland F., Fleming L., Ponder K.P., Liu T.-J., Finegold M., Darlington G., Pokorny W., and Woo S.L.C. Expression of human α-1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci 89 (1992) 89-93
212. Alino S.F., Bobadilla M., Garcia-Sanz M., Lejarreta M., Unda F., and Hilario E. In vivo delivery of human alpha-1-antitrypsin gene to mouse hepatocytes by liposomes. Biochem Biophys Res Commun 192 (1993) 174-181
213. Knoell D.L., and Wewers M.D. Clinical implications of gene therapy for alpha-1-antitrypsin deficiency. Chest 107 (1995) 535-545
214. Joenje H., Mathew C., and Gluckman E. Fanconi anemia research: current status and prospects. Eur J Cancer 31A (1995) 268-272
215. Cervenka J., Arthur D., and Yasis C. Mitomycin C test for diagnostic differentiation of idiopathic aplastic anemia and Fanconi anemia. Pediatrics 67 (1981) 119-127
216. Hoeijmakers J.H.J., and Bootsma D. DNA Repair-2 pieces of the puzzle. Nature Genet 1 (1992) 313-314
217. Joenje H., and Gille J.J.P. Oxygen metabolism and chromosomal breakage in Fanconi anemia. In: Schroeder R., Obe G., and Auerbach A.D. (Eds). Clinical, Cytogenetic and Experimental Aspects of Fanconi Anemia (1989), Springer Verlag, Berlin 174-182
218. Roselli F., Sanceau J., Gluckman E., Wietzerbin J., and Moustacchi E. Abnormal lymphokine production-a novel feature of the genetic disease Fanconi anemia. II. Spontaneous overproduction of tumor necrosis factor alpha. A possible tool in detecting heterozygous individuals. Blood. 83 (1994) 1216-1225
219. Joenje H., Lo Ten Foe J.R., Arwert F., and ten Kate L.P. Complementation testing and genetic classification of Fanconi's anemia. Lancet 343 (1994) 862
220. Strathdee C.A., Gavish H., Shannon W.R., and Buchwald M. Cloning of cDNAs for Fanconi's anemia by functional complementation. Nature 356 (1992) 763-767
221. Auerbach A.D. Fanconi anemia. Dermatol Clin 13 (1995) 41-49
222. Walsh C.E., Grompe M., Vanin E., Buchwald M., Young N.S., Nienhuis A.W., and Lui J.M. A functionally active retrovirus vector for gene therapy in Fanconi anemia group C. Blood 84 (1994) 453-459
223. Walsh C.E., Nienhuis A.W., Samulski R.J., Brown M.G., Miller J.L., Young N.S., and Liu J.M. Phenotypic correction of Fanconi anemia in human hematopoetic cells with a recombinant adeno-associated virus vector. J Clin Invest 94 (1994) 1440-1448
224. Liu J.M., Kim S., and Walsh C.E. Retroviral-mediated transduction of the Fanconi anemia C complementing (FACC) gene in two murine transplantation models. Blood Cell Molec Dis 21 (1995) 56-63
225. Marshall E. Gene therapy's growing pains. Science 269 (1995) 1050-1055
226. Rosenberg S.A., Aeversold P.M., Cornetta K., Kasid A., Morgan R.A., Moen R., Karson E.M., Lotze M.T., Yang H.C., Topalian S.L., Merino M.J., Culver K., Miller A.D., Blaese R.M., and Anderson W.F. Gene transfer into humans-immunotherapy of patients with advanced melanoma using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med. 323 (1990) 570-578
227. Kradin R.L., Kurnick J.T., Lazarus D.S., Preffer R.I., Dubinett S.M., Pinto C., Gifford J., Davidson E., Grove B., Callahan R.J., and Strauss H.W. Tumor infiltrating lymphocytes and interleukin-2 in the treatment of advanced cancer. Lancet 1 (1989) 577-580
228. Rosenberg S.A. The immunotherapy and gene therapy of cancer. J Clin Oncol 10 (1992) 180-199
229. Tepper R., and Mule J.J. Experimental and clinical studies of cytokine gene modified tumor cells. Hum Gene Ther 5 (1994) 153-164
230. Hwu P., Yannelli J., Kriegler M., Anderson W.F., Perez C., Chiang Y., Schwartz S., Cowherd R., Del-gado C., Mule J., and Rosenberg S.A. Functional and molecular characterization of tumor-infiltrating lymphocytes transduced with tumor necrosis factor-alpha cDNA for the gene therapy of cancers in humans. J Immunol. 150 (1993) 4104-4115
231. Nabel G. Immunotherapy of malignancy by in vivo gene transfer into tumors. Hum Gene Ther 3 (1992) 399-410
232. Rosenberg S.A., Anderson W.F., Asher A.L., Blaese M.R., Ettinghausen S.E., Hwu P., Kasid A., Mule J.J., Parkinson D.R., Schwartzentruber D.J., Topalian S.L., Weber J.S., Yannelli J.R., Yang J.C., and Linehan W.M. Immunization of cancer patients using autologous cancer cells modified by insertion of the gene for tumor necrosis factor. Hum Gene Ther 3 (1992) 57-73
233. Osanto S., Brouwenstyn N., Vaessen N., Figdor C.G., Melief C.J.M., and Schrier P.I. Immunization with interleukin-2 transfected melanoma cells. A phase I-II study in patients with metastatic melanoma. Hum Gene Ther 4 (1993) 323-330
234. 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 NY Acad Sci 716 (1994) 144-153
235. Silver H.K.B., Klasa R.J., and Bally M.B. Genetic allogenization of malignant melanoma. Transfus Sci 17 (1996) 169-174
236. Tonkinson J.L., and Stein C.A. Antisense oligodeoxynucleotides as clinical therapeutic agents. Cane Invest 14 (1996) 54-65
237. Maher L.J. Prospects for the therapeutic use of antigene oligonucleotides. Cane Invest 14 (1996) 66-82
238. Vogelstein B. Cancer. A deadly inheritance. Nature 348 (1990) 681-682
239. Selivanova G., and Wilman K.G. p53: a cell cycle regulator activated by DNA damage. Adv Cancer Res 66 (1995) 143-180
240. Keurbitz S.J., Plunkett B.S., Walsh W.V., and Kastan M.B. Wild-type p53 in a cell cycle checkpoint determinant following irradation. Proc Natl Acad Sci 89 (1992) 7491-7495
241. Lane D.P. p53, guardian of the genome. Nature 358 (1992) 15-16
242. Chang F., Syrjanen S., and Syrjanen K. Implications of p53 tumor-suppressor gene in clinical oncology. J Clin Oncol 13 (1995) 1009-1022
243. Casey G., Lo-Hsueh M., Lopez M.E., Vogelstein B., and Stanbridge E.J. Growth suppression of human breast cancer cells by the introduction of a wild-type p53 gene. Oncogene 6 (1991) 1791-1797
244. Ozturk M., Ponchel F., and Puisieux A. p53 as a potential target in cancer therapy. Bone Marrow Transpl 9 S (1992) 164-170
245. Fujiwara T., Grimm E.A., and Roth J.A. Gene therapeutics and gene therapy for cancer. Curr Opin Oncol 6 (1994) 96-105
246. Eastham J.A., Hall S.J., Sehgal I., Wang J., Timme T.L., Yang G., Connell-Crowley L., Elledge S.I., Zhang W.W., and Harper J.W. In vivo gene therapy with p53 or p21 adenovirus for prostate cancer. Cancer Res 55 (1995) 5151-5155
247. Workman P. To pop or not to pop: p53 as a critical modulator of tumour responsiveness to therapy in vivo?. Hum Exper Tox 14 (1995) 222-225
248. Wills K.N., Maneval C., Menzel P., Harris M.P., Sutjipto S., Vaillancourt M.-T., Huang W.-M., Johnson D.E., Anderson S.C., Wen S.F., Bookstein R., Shepard H.M., and Gregory R.J. Development and characterization of recombinant adenoviruses encoding human p53 for gene therapy of cancer. Hum Gene Ther 5 (1994) 1079-1088
249. Fujiwara T., Cai D.W., Georges R., Mukhapakhyat T., Grimm E.A., and Roth J.A. Therapeutic effect of a retroviral wild-type p53 expression vector in an orthotopic lung cancer model. J Natl Cancer Inst 86 (1994) 1458-1462
250. Lesoon-Wood L.A., Kim W.H., Kleinman H.K., Weintraub B.D., and Mixson 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
251. Cai D.W., Mukhopadhyay T., and Roth J.A. Suppression of lung cancer cell growth by ribozyme-mediated modification of p53 pre-mRNA. Cancer Gene Ther 2 (1995) 199-205
252. Freeman S.M., Abboud C.N., Whartenby K.A., Packman C.H., Koeplin D.S., Moolten F.L., and Abraham G.N. The "bystander effect" tumor regression when a fraction of the tumor mass is genetically modified. Cancer Res 53 (1993) 5274-5283
253. 8+ HIV-specific T cells for HIV seropositive patients undergoing allogeneic bone marrow transplant. Hum Gene Ther. 3 (1992) 319-328
254. Sorrentino B.P., Brandt S.J., Bodine D., Gottesman M., Pastan I., Cline A., and Nienhuis A.W. Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR-1. Science 257 (1992) 99-103
255. Deisseroth AB, RAC Report 9406-077, 1995; June.
256. Merson M.H. Slowing the spread of HIV: agenda for the 1990s. Science 260 (1993) 1266-1268
257. Wong-Staal F. Human immunodeficiency virus and their replication. In: Fields B.N., and Knipe D.M. (Eds). Fundamental Virology (1991), Raven Press Ltd., New York 709-723
258. Yu M., Poeschla E., and Wong-Staal F. Progress towards gene therapy for HIV infection. Gene Ther 1 (1995) 13-26
259. Baltimore D. Gene therapy: intracellular immunization. Nature 355 (1988) 395-396
260. Friedman A.D., Triezenberg S.J., and McKnight S.L. Expression of truncated viral transactivator selectively impedes lytic infection by its cognate virus. Nature 355 (1988) 452-454
261. Herskowitz I. Function inactivation of genes by dominant negative mutations. Nature 329 (1987) 219-222
262. Malim M.H., and Cullen B.R. HIV-1 structural gene expression requires the binding of multiple Rev monomers to the viral RRE: implications for HIV-1 latency. Cell 65 (1991) 241-248
263. Dalgleish A.G., Beverley P.C., Clapham P.R., Crawford D.H., Greaves M.F., and Weiss R.A. The CD4 (T4) antigen is an essential component of the receptor for the AIDS retrovirus. Nature 312 (1984) 763-767
264. Deen K.C., McDougal J.S., Inacker R., Folena-Wasserman G., Anthos J., Rosenberg J., Maddon P.J., Axel R., and Sweet R.W. A soluble form of CD4 (T4) inhibits AIDS virus infection. Nature 331 (1988) 82-84
265. Morgan R.A., Looney D.J., Muenchau D.D., Wong-Staal F., Gallo R.C., and Anderson W.R. Retroviral vectors expressing soluble CD4: a potential gene therapy for AIDS. AIDS Res Hum Retrovir 6 (1990) 183-191
266. Daar E.S., and Ho D.D. Relative resistance of primary HIV-1 isolates to neutralization by soluble CD4. Am J Med 90 (1991) 22S-26S
267. OrlofF S.L., Kennedy M.S., Belperron A.A., Maddon P.J., and McDougal J.S. Two mechanisms of soluble CD4 (sCD4)-mediated inhibition of human immunodeficiency virus type 1 (HIV-1) infectivity and their relation to primary HIV-1 isolates with reduced sensitivity to sCD4. J Virol. 67 (1993) 1461-1471
268. Stein C.A., and Cheng Y.C. Antisense oligonucleotides as therapeutic agents-is the bullet really magical?. Science 261 (1993) 1004-1012
269. Chatterjee S., Johnson P.R., and Wong Jr. K.K. Dual-target inhibition of HIV-1 in vitro my means of an adenoassociated virus antisense vector. Science 258 (1992) 1485-1488
270. Gilboa E., and Smith C. Gene therapy for infectious diseases: the AIDS model. Trends Genet 10 (1994) 139-144
271. Sullenger B.A., Gallardo H.F., Ungers G.E., and Gilboa E. Overexpression of TAR sequences renders cells resistant to human immunodeficiency virus replication. Cell 63 (1990) 601-608
272. Lee T.C., Sullenger B.A., Gallardo H.F., Ungers G.E., and Gilboa E. Overexpression of RRE-derived sequences inhibits HIV-1 replication in CEM cells. New Biol 4 (1992) 66-74
273. Sarver N., Cantin E.M., Chang P.S., Zaia J.A., Ladne P.A., Stephens D.A., and Rossi J.J. Ribozymes as potential anti-HIV-1 therapeutic agents. Science 247 (1990) 1222-1225
274. Lorentzen E.U., Wieland U., Kuhn J.E., and Braun R.W. In vitro cleavage of HIV-1 vif RNA by synthetic ribozyme. Virus Genes 5 (1991) 17-23
275. Sioud M., and Drlica K. Prevention of human immunodeficiency virus type 1 integrase expression in Escherichia coli by a ribozyme. Proc Natl Acad Sci 88 (1991) 7303-7307
276. 4+ lymphocyte-derived cell lines conferred by using retroviral vectors expressing an HIV-1 RNA-specific ribozyme. J Virol. 65 (1991) 5531-5534
277. Yu M., Ojwang J., Yamada O., Hampel A., Rapapport J., Looney D., and Wong-Staal F. A hairpin ribozyme inhibits expression of diverse strains of human immunodeficiency virus type 1. Proc Natl Acad Sci 90 (1993) 6340-6344
278. Warner J.F., Anderson C.G., Laube L., Jolly D.J., Townsend K., Chada S., and Louis D. Induction of HIV-specific CTL and antibody responses in mice using retroviral vector-transduced cells. AIDS Res Hum Retrovir 7 (1991) 645-655
279. Jolly D, Retroviral vectors expressing HIV env as immunotherapeutics for HIV infection. UCLA/UCI AIDS Symposium: Gene Therapy Approaches to Treatment of HIV Infection. 1993; 12.
280. Morgan A.R., and Wells R.D. Specificity of the three-strand complex formation between double-strand DNA and single-strand RNA containing repeating nucleotide sequences. J Molec Biol 37 (1968) 63-80
281. Zamecnik P.C., and Stephenson M.L. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc Natl Acad Sci 75 (1978) 280-284
282. Walder R.Y., and Walder J.A. Role of RNase H in hybrid-arrested translation by antisense oligonucleotides. Proc Natl Acad Sci 85 (1988) 5011-5015
283. Scanlon K.J., Ohta Y., Ishida H., Kijima H., Ohkawa T., Kaminski A., Tsai J., Horng G., and Kashanisabet M. Oligonucleotide-mediated modulation of mammalian gene expression. FASEB J 9 (1995) 1288-1296
284. Janicek M.F., Sevin B.U., Nguyen H.N., and Averette H.E. Combination anti-gene therapy targeting c-myc and p53 in ovarian cancer cell lines. Gynecol Oncol 59 (1995) 87-92
285. Normanno N., Selvam M.P., Bianco C., Damiano V., Deangelis E., Grassi M., Magliulo G., Tortora G., Salomon D.S., and Ciardiello F. Amphiregulin anti-sense oligodeoxynucleotides inhibit growth and transformation of a human colon carcinoma cell line. Int J Cancer 62 (1995) 762-766
286. Hall W.A., Flores E.P., and Low W.C. Antisense oligonucleotides for central nervous system tumors. Neurosurgery 38 (1996) 376-383
287. Matsukura M., Koike K., and Zon G. Antisense phosphorothioates as antivirals against human immunodeficiency virus (HIV) and hepatitis B virus (HBV). Toxicol Lett 82-83 (1995) 435-438
288. Sczakiel G. Antisense strategies in the treatment of hematological malignancies. Stem Cells 13 Suppl 3 (1995) 88-92
289. Maher III L.J., Wold B., and Dervan P.B. Inhibition of DNA binding proteins by oligonucleotide-directed triple helix formation. Science 245 (1989) 725-730
290. Havre P.A., Gunther E.J., Gasparro F.P., and Glazer P.M. Targeted mutagenesis of DNA using triple helix-forming oligonucleotides linked to psoralen. Proc Natl Acad Sci 90 (1993) 7879-7883
291. Sandor Z., and Bredberg A. Repair of triple helix directed psoralen adducts in human cells. Nucl Acid Res 22 (1994) 2051-2056
292. Neckers L., Whitesell L., Rosolen A., and Geselowitz D.A. Antisense inhibition of oncogene expression. Crit Rev Oncogen 3 (1992) 175-231
293. Leonetti J.P., Degols G., Clarenc J.P., Mechti N., and Lebleu B. Cell delivery and mechanisms of action of antisense oligonucleotides. Prog Nucl Acid Res Molec Biol 44 (1993) 143-166
294. Sullenger B.A., and Cech T.R. Ribozyme-mediated repair of defective mRNA by targeted trans-splicing. Nature 371 (1994) 619-622
295. Woolf T.M., Chase J.M., and Stinchcomb D.T. Toward the therapeutic editing of mutated RNA sequences. Proc Natl Acad Sci 92 (1995) 8298-8302
296. Sullivan S.M. Development of ribozymes for gene therapy. J Invest Dermatol 103 (1994) 85S-89S
297. Thompson J.D., Macejak D., Couture L., and Stinchcomb D.T. Ribozymes in gene therapy. Nature Med 1 (1995) 277-278
298. Ciafrè S.A., Rinaldi M., Gasparini P., Seripa D., Bisceglia L., Zelante L., Farace M.G., and Fazio V.M. Stability and functional effectiveness of phosphorothioate modified duplex DNA and synthetic 'minigenes'. Nucl Acid Res 23 (1995) 4134-4142
299. Zhang R.W., Lu Z.H., Zhang X.S., Zhao H., Diasio R.B., Liu T.P., Jiang Z.W., and Agrawal S. In vivo stability and disposition of a self-stabilized oligodeoxynucleotide phosphorothioate in rats. Clin Chem 41 (1995) 836-843
300. Thiery A.R., and Dritschilo A. Intracellular availability of unmodified, phosphorothioated and liposomally encapsulated oligodeoxynucleotides for antisense activity. Nucl Acid Res 20 (1992) 5691-5698
301. Capaccioli S., DiPasquale G., Mini E., Mazzei T., and Quattrone A. Cationic lipids improve antisense oligonucleotide uptake and prevent degradation in cultured cells in human serum. Biochem Biophys Res Commun 197 (1993) 818-825
302. Clarenc J., Degols G., Leonetti J., Milhaud P., and Lebleu B. Delivery of antisense oligonucleotides by poly(L-lysine) conjugation and liposome encapsulation. Anti-Cancer Drug Design 8 (1993) 81-94
303. Krieg A.M., Tonkinson J., Matson S., Zhao Q., Saxon M., Zhang L.M., Bhanja U., Yakubov L., and Stein C.A. Modification of antisense phosphodiester oligodeoxynucleotides by a 5′ cholesteryl moiety increases cellular association and improves efficacy. Proc Natl Acad Sci 90 (1993) 1048-1052
304. Wang Q., and Marini J.C. Antisense oligodeoxynucleotides selectively suppress expression of the mutant α2(I) collagen allele in type IV osteogenesis imperfecta fibroblasts-a molecular approach to therapeutics of dominant negative disorders. J Clin Invest. 97 (1996) 448-454
305. Tomita N., Morishita R., Higaki J., Aoki M., Nakamura Y., Mikami H., Fukamizu A., Murakami K., Kaneda Y., and Ogihara T. Transient decrease in high blood pressure by in vivo transfer of antisense oligodeoxynucleotides against rat angiotensinogen. Hypertension 26 (1995) 131-136