Gene therapy for human inherited disorders: techniques and status

Critical Reviews in Oncology/Hematology - Tập 13 - Trang 33-54 - 1992
Rob C. Hoeben1, Dinko Valerio2, Alex J. van der Eb1, Hans van Ormondt1
1Department of Medical Biochemistry, Laboratory of Molecular Carcinogenesis University of Leiden, Leiden, The Netherlands
2Department of Gene Therapy, Institute for Applied Radiobiology and Immunology, ITRI-TNO, Rijswijk, The Netherlands

Tài liệu tham khảo

Graham, 1973, A new technique for the assay of infectivity of human adenovirus 5 DNA, Virology, 52, 456, 10.1016/0042-6822(73)90341-3 Vega, 1991, Prospects for homologous recombination in human gene therapy, Hum Genet, 87, 245, 10.1007/BF00200899 Koller, 1989, Germ-line transmission of a planned alteration made in a hypoxanthine phosphoribosyl-transferase gene by homologous recombination in embryonic stem cells, 86, 8927 Capecchi, 1989, Altering the genome by homologous recombination, Science, 244, 1288, 10.1126/science.2660260 Thompson, 1989, Germ line transmission and expression of a corrected HPRT gene produced by gene targeting in embryonic stem cells, Cell, 56, 313, 10.1016/0092-8674(89)90905-7 Friedmann, 1989, Progress toward human gene therapy, Science, 244, 1275, 10.1126/science.2660259 Miller, 1990, Progress toward human gene therapy, Blood, 76, 271, 10.1182/blood.V76.2.271.271 Anderson, 1980, Gene therapy in human beings: when is it ethical to begin?, N Engl J Med, 303, 1293, 10.1056/NEJM198011273032210 Williamson, 1982, Gene therapy, Nature, 298, 416, 10.1038/298416a0 Motulsky, 1983, Impact of genetic manipulation on society and medicine, Science, 219, 135, 10.1126/science.6336852 Anderson, 1989, Human gene therapy: why draw a line?, J Med Philos, 14, 681, 10.1093/jmp/14.6.681 Anderson, 1984, Prospects for human gene therapy, Science, 226, 401, 10.1126/science.6093246 Belmont, 1986, Developments leading to human gene therapy, 411 Nichols, 1988 Ledley, 1987, Somatic gene therapy for human disease: background and prospects. Part I, J Pediatr, 110, 1, 10.1016/S0022-3476(87)80278-0 Ledley, 1987, Somatic gene therapy for human disease: background and prospects. Part II, J Pediatr, 110, 167, 10.1016/S0022-3476(87)80148-8 Walters, 1986, The ethics of human gene therapy, Nature, 320, 225, 10.1038/320225a0 Pursel, 1989, Genetic engineering of livestock, Science, 244, 1281, 10.1126/science.2499927 Capecchi, 1989, The new mouse genetics: altering the genome by gene targeting, Trends Genet, 5, 70, 10.1016/0168-9525(89)90029-2 Palmiter, 1986, Germ-line transformation of mice, Ann Rev Genet, 20, 465, 10.1146/annurev.ge.20.120186.002341 Leyte, 1991, Sulfation of Tyr1680 of human blood coagulation factor VIII is essential for the interaction of factor VIII with von Willebrand factor, J Biol Chem, 266, 740, 10.1016/S0021-9258(17)35234-1 Thompson, 1986, Structure, function, and molecular defects of factor IX, Blood, 67, 565, 10.1182/blood.V67.3.565.565 Fernlund, 1983, Beta-hydroxyaspartic acid in vitamin K-dependent proteins, J Biol Chem, 258, 12509, 10.1016/S0021-9258(17)44205-0 Rosenfeld, 1992, In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium, Cell, 68, 143, 10.1016/0092-8674(92)90213-V Rosenfeld, 1991, Adenovirus-mediated transfer of a recombinant α1-antitrypsin gene to the lung epithelium in vivo, Science, 252, 431, 10.1126/science.2017680 Hammer, 1984, Partial correction of murine hereditary growth hormone disorder by germ-line incorporation of a new gene, Nature, 311, 65, 10.1038/311065a0 Dzierzak, 1988, Lineage-specific expression of a human Beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells, Nature, 331, 35, 10.1038/331035a0 Capecchi, 1980, High efficiency transformation by direct microinjection of DNA into cultured cells, Cell, 22, 479, 10.1016/0092-8674(80)90358-X Johnston, 1990, Biolistic transformation: microbes to mice, Nature, 346, 776, 10.1038/346776a0 Potter, 1988, Electroporation in biology: methods, applications, and instrumentation, Anal Biochem, 174, 361, 10.1016/0003-2697(88)90035-8 Potter, 1984, Enhancer-dependent expression of human kappa-immunoglobulin genes introduced into mouse pre-B lymphocytes by electroporation, 81, 7161 Felgner, 1987, Lipofection: a highly efficient, lipid-mediated DNA transfection procedure, 84, 7413 Nicolau, 1987, Liposomes as carriers for in vivo gene transfer and expression, Methods Enzymol, 149, 157, 10.1016/0076-6879(87)49054-X Felgner, 1989, Cationic liposome-mediated transfection, Nature, 337, 387, 10.1038/337387a0 Lopata, 1984, High level transient expression of a chloramphenicol acetyl transferase gene by DEAE-dextran mediated DNA transfection coupled with a dimethyl sulfoxide or glycerol shock treatment, Nucleic Acids Res, 12, 5707, 10.1093/nar/12.14.5707 Weiss, 1984 Brown, 1989, Retroviral integration: structure of the initial covalent product and its precursor, and a role for the viral IN protein, 86, 2525 Shih, 1988, Highly preferred targets for retrovirus integration, Cell, 53, 531, 10.1016/0092-8674(88)90569-7 Scherdin, 1990, Transcriptionally active genome regions are preferred integration sites for retrovirus integration, J Virol, 64, 907, 10.1128/JVI.64.2.907-912.1990 Mooslehner, 1990, Retroviral integration sites in transgenic Mov mice frequently map in the vicinity of transcribed DNA regions, J Virol, 64, 3056, 10.1128/JVI.64.6.3056-3058.1990 Rohdewohld, 1987, Retrovirus integration and chromatin structure: Moloney murine leukemia virus integration sites map near DNase I-hypersensitive sites, J Virol, 63, 336, 10.1128/JVI.61.2.336-343.1987 Vijaya, 1986, Acceptor sites for retroviral integration map near DNase I-hypersensitive sites in chromatin, J Virol, 60, 683, 10.1128/JVI.60.2.683-692.1986 Brown, 1988, Retroviral vectors, Vol.III, 189 Miller, 1990, Retrovirus packaging cells, Hum Gene Therapy, 1, 5, 10.1089/hum.1990.1.1-5 Miller, 1986, Factors involved in production of helper virus-free retrovirus vectors, Somat Cell Mol Genet, 12, 175, 10.1007/BF01560664 Valerio, 1992, Retrovirus vectors for gene therapy procedures, 211 Cone, 1984, High-efficiency gene transfer into mammalian cells: generation of helper-free recombinant retrovirus with broad mammalian host range, 81, 6349 Mann, 1983, Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus, Cell, 33, 153, 10.1016/0092-8674(83)90344-6 Danos, 1988, Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges, 85, 6460 Scarpa, 1991, Characterization of recombinant helper viruses from Moloney-based vectors in ecotropic and amphotropic packaging cell lines, Virology, 180, 849, 10.1016/0042-6822(91)90105-K Bosslman, 1987, Replication-defective chimeric helper provirus and factors affecting generation of competent virus: Expression of Moloney murine leukemia virus structural genes via the metallothionein promoter, Mol Cell Biol, 7, 1797, 10.1128/MCB.7.5.1797 Markowitz, 1988, Construction and use of a safe and efficient amphotropic packaging cell line, Virology, 167, 400, 10.1016/0042-6822(88)90101-8 Markowitz, 1988, A safe packaging line for gene transfer: separating viral genes of two different plasmids, J Virol, 62, 1120, 10.1128/JVI.62.4.1120-1124.1988 Morgenstern, 1990, Advanced mammalian gene transfer: high titre retroviral vectors with multiple drug selection markers and a complementary helper-free packaging cell line, Nucleic Acids Res, 18, 3587, 10.1093/nar/18.12.3587 Scadden, 1990, Human cells infected with retroviral vectors acquire an endogenous murine provirus, J Virol, 64, 424, 10.1128/JVI.64.1.424-427.1990 Hatzoglou, 1990, Efficient packaging of a specific VL30 retro-element by psi-2 cells which produce MoMLV recombinant retroviruses, Hum Gene Ther, 1, 385, 10.1089/hum.1990.1.4-385 Morgan, 1990, Applications of the polymerase chain reaction in retroviral-mediated gene transfer and the analysis of gene-marked human TIL cells, Hum Gene Ther, 1, 135, 10.1089/hum.1990.1.2-135 Cornetta, 1991, Safety issues related to retroviral-mediated gene transfer in humans, Hum Gene Ther, 2, 5, 10.1089/hum.1991.2.1-5 Barklis, 1986, Chromosomal position or virus mutation permits retrovirus expression in embryonomal carcinoma cells, Cell, 47, 391, 10.1016/0092-8674(86)90596-9 Feuer, 1989, Two blocks in Moloney murine leukemia virus expression in undifferentiated F9 embryonal carcinoma cells as determined by transient expression assays, J Virol, 63, 2317, 10.1128/JVI.63.5.2317-2324.1989 Linney, 1984, Non-function of a Moloney murine leukemia virus regulatory sequence in F9 embryonal carcinoma cells, Nature, 308, 470, 10.1038/308470a0 Loh, 1987, Proviral sequences that restrict retroviral expression in mouse embryonal carcinoma cells, Mol Cell Biol, 7, 3775, 10.1128/MCB.7.10.3775 Loh, 1988, Negative regulation of retrovirus expression in embryonal carcinoma cells is mediated by an intragenic domain, J Virol, 62, 4086, 10.1128/JVI.62.11.4086-4095.1988 Niwa, 1985, Suppression of the hypomethylated Moloney leukemia virus genome in undifferentiated teratocarcinoma cells and inefficiency of transformation by a bacterial gene under controle of the long terminal repeat, Mol Cell Biol, 5, 2325, 10.1128/MCB.5.9.2325 Niwa, 1983, Independent mechanisms involved in suppression of the Moloney leukemia virus genome during differentiation of murine teratocarcinoma cells, Cell, 32, 1105, 10.1016/0092-8674(83)90294-5 Tsukiyama, 1991, Analysis of the binding proteins and activity of the long terminal repeat of Moloney murine leukemia virus during differentiation of mouse embryonal carcinoma cells, J Virol, 65, 2979, 10.1128/JVI.65.6.2979-2986.1991 Tsukiyama, 1989, Mechanism of suppression of the long terminal repeat of Moloney leukemia virus in mouse embryonal carcinoma cells, Mol Cell Biol, 9, 4670, 10.1128/MCB.9.11.4670 Weiher, 1987, Two distinct sequence elements mediate retroviral gene expression in embryonal carcinoma cells, J Virol, 61, 2742, 10.1128/JVI.61.9.2742-2746.1987 Gautsch, 1983, Delayed de novo methylation in teratocarcinoma suggests additional tissue-specific mechanisms for controlling gene expression, Nature, 301, 32, 10.1038/301032a0 Jaehner, 1982, De novo methylation and expression of retroviral genomes during mouse embryogenesis, Nature, 298, 623, 10.1038/298623a0 Jaenisch, 1984, Methylation, expression and chromosomal position of genes in mammals, Biochim Biophys Acta, 782, 1, 10.1016/0167-4781(84)90099-X Stuhlmann, 1981, Infectivity and methylation of retroviral genomes is correlated with expression in the animal, Cell, 26, 221, 10.1016/0092-8674(81)90305-6 Harbers, 1981, DNA methylation and gene expression: Endogenous retroviral genome becomes infectious after molecular cloning, 78, 7609 Simon, 1983, Retrovirus genomes methylated by mammalian but not by bacterial methylase are non-infectious, Nature, 304, 275, 10.1038/304275a0 Stewart, 1982, De novo methylation, expression and infectivity of retroviral genomes introduced into embryonal carcinoma cells, 79, 4098 Varmus, 1985, Replication of retroviruses, 75 Taketo, 1985, Isolation of embryonal carcinoma cell lines that express integrated recombinant genes flanked by the Moloney murine leukemia virus long terminal repeat, 82, 2422 Taketo, 1987, A cellular enhancer of retrovirus gene expression in embryonal carcinoma cells, 84, 3748 Peckham, 1989, Retrovirus activation in embryonal carcinoma cells by cellular promoters, Genes Devel, 3, 2062, 10.1101/gad.3.12b.2062 Valerio, 1986, One adenosine deaminase allele in a patient with severe combined immunodeficiency contains a point mutation abolishing enzyme activity, EMBO J, 5, 113, 10.1002/j.1460-2075.1986.tb04184.x McIvor, 1987, Human purine nucleoside phosphorylase and adenosine deaminase: gene transfer into cultured cells and murine hematopoietic stem cells by using recombinant amphotropic retroviruses, Mol Cell Biol, 7, 838, 10.1128/MCB.7.2.838 Guild, 1988, Development of retrovirus vectors useful for expressing genes in cultured murine embryonal cells and hematopoietic cells in vivo, J Virol, 62, 3795, 10.1128/JVI.62.10.3795-3801.1988 Valerio, 1989, Retrovirus-mediated gene transfer into embryonal carcinoma and hemopoietic stem cells: expression from a hybrid long terminal repeat, Gene, 84, 419, 10.1016/0378-1119(89)90516-7 Hock, 1988, Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietc cell lines, Blood, 74, 876, 10.1182/blood.V74.2.876.876 Kaleko, 1990, Expression of human adenosine deaminase in mice after transplantation of genetically-modified bone marrow, Blood, 75, 1733, 10.1182/blood.V75.8.1733.1733 Belmont, 1988, Expression of human adenosine deaminase in murine hematopoietic cells, Mol Cell Biol, 8, 5166, 10.1128/MCB.8.12.5116 Moore, 1990, Human adenosine deaminase expression in mice, Blood, 75, 2085, 10.1182/blood.V75.10.2085.2085 Wilson, 1990, Expression of human adenosine deaminase in mice reconstituted with retrovirus-transduced hematopoietic stem cells, 87, 439 Emerman, 1986, Comparison of promoter suppression in avian and murine retrovirus vectors, Nucleic Acids Res, 14, 9381, 10.1093/nar/14.23.9381 Emerman, 1984, Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism, Cell, 39, 449, 10.1016/0092-8674(84)90453-7 Hawley, 1987, Handicapped retroviral vectors efficiently transduce foreign genes into haematopoietic stem cells, 84, 2406 Yu, 1986, Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells, 83, 3194 Yee, 1987, Gene expression from transcriptionally disabled retroviral vectors, 84, 5197 Jolly, 1986, Variable stability of a selectable provirus after retroviral vector gene transfer into human cells, Mol Cell Biol, 6, 1141, 10.1128/MCB.6.4.1141 Xu, 1989, Factors affecting long-term stability of Moloney murine leukemia virus-based vectors, Virology, 171, 331, 10.1016/0042-6822(89)90600-4 Stone, 1986, Stability of retrovirally transduced markers in a rat cell line, Somat Cell Mol Genet, 12, 575, 10.1007/BF01671943 Hoeben, 1991, Inactivation of the Moloney murine leukemia virus long terminal repeat in murine fibroblast cell lines is associated with methylation and dependent on its chromosomal position, J Virol, 65, 904, 10.1128/JVI.65.2.904-912.1991 Palmer, 1991, Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes, 88, 1330 Shinnick, 1981, Nucleotide sequence of Moloney murine leukemia virus, Nature, 293, 543, 10.1038/293543a0 Adam, 1988, Identification of a signal in a murine retrovirus that is sufficient for packaging of nonretroviral RNA into virions, J Virol, 62, 3802, 10.1128/JVI.62.10.3802-3806.1988 Armentano, 1987, Effect of internal viral sequences on the utility of retroviral vectors, J Virol, 61, 1647, 10.1128/JVI.61.5.1647-1650.1987 Bender, 1989, Evidence that the packaging signal of Moloney murine leukemia virus extends into the gag region, J Virol, 61, 1426 Hwang, 1984, Expression of genes introduced into cells by retroviral infection is more efficient than that of genes introduced into cells by DNA transfection, J Virol, 50, 417, 10.1128/JVI.50.2.417-424.1984 Miller, 1985, Generation of helper-free amphotropic retroviruses that transduce a dominant-acting, methotrexate-resistant dihydrofolate reductase gene, Mol Cell Biol, 5, 431, 10.1128/MCB.5.3.431 Bodine, 1990, Development of a high-titer retrovirus producer cell line capable of gene transfer into rhesus monkey hematopoietic stem cells, 87, 3738 Bestwick, 1988, Overcoming interference to retroviral superinfection results in amplified expression and transmission of cloned genes, 85, 5404 Lynch, 1991, Production of high-titer helper virus-free retroviral vectors by cocultivation of packaging cells with different host ranges, J Virol, 65, 3887, 10.1128/JVI.65.7.3887-3890.1991 Muenchau, 1990, Analysis of retroviral packaging lines for generation of replication-competent virus, Virology, 176, 262, 10.1016/0042-6822(90)90251-L Cornetta, 1990, Amphotropic murine leukemia retrovirus is not an acute pathogen for primates, Hum Gene Ther, 1, 15, 10.1089/hum.1990.1.1-15 Donahue, 1992, In vivo evaluation of gene transfer to primate CD43-positive cells (abstract), J Cell Biochem, 16F, 46 Van Lohuizen, 1990, Tumorigenesis by slow-transforming retroviruses-an update, Biochim Biophys Acta, 1032, 213 Temin, 1990, Safety considerations in somatic gene therapy of human disease with retrovirus vectors, Hum Gene Ther, 1, 111, 10.1089/hum.1990.1.2-111 Miller, 1990, Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection, Mol Cell Biol, 10, 4239, 10.1128/MCB.10.8.4239 Kotin, 1990, Sitespecific intergration by adeno-associated virus, 87, 2211 Samulski, 1991, Targeted integration of adeno-associated virus (AAV) into human chromosome 19, EMBO J, 10, 3941, 10.1002/j.1460-2075.1991.tb04964.x Muzyczka, 1988, Use of adeno-associated virus as a mammalian transduction vector, 39 McLaughlin, 1988, Adenoassociated virus general transduction vectors: Analysis of proviral structures, J Virol, 62, 1963, 10.1128/JVI.62.6.1963-1973.1988 Laface, 1988, Gene transfer into hematopoietic progenitor cells mediated by an adeno-associated virus vector, Virology, 162, 483, 10.1016/0042-6822(88)90491-6 Geller, 1990, An efficient deletion mutant packaging system for defective herpes simplex virus vectors: Potential applications to human gene therapy and neuronal physiology, 87, 8950 Geller, 1988, A defective HSV-1 vector expresses Escherichia coli Beta-galactosidase in cultured peripheral neurons, Science, 241, 1667, 10.1126/science.2843986 Geller, 1991, A system, using neural cell lines, to characterize HSV-1 vectors containing genes which affect neuronal physiology, or neuronal promoters, J Neurosci Methods, 36, 91, 10.1016/0165-0270(91)90142-M Felgner, 1991, Gene therapeutics, Nature, 349, 351, 10.1038/349351a0 Benvenisty, 1986, Direct introduction of genes into rats and expression of the genes, 83, 9551 Nicolau, 1983, In vivo expression of rat insulin after intravenous administration of the liposome-entrapped gene for rat insulin I, 80, 1068 Nabel, 1990, Site-specific gene expression in vivo by direct gene transfer into the arterial wall, Science, 249, 1285, 10.1126/science.2119055 Wu, 1989, Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo, J Biol Chem, 264, 16985, 10.1016/S0021-9258(18)71447-6 Wu, 1988, Receptor-mediated gene delivery and expression in vivo, J Biol Chem, 263, 14621, 10.1016/S0021-9258(18)68081-0 Wolff, 1991, Conditions affecting direct gene transfer into rodent muscle in vivo, Biotechniques, 11, 474 Wolff, 1990, Direct gene transfer into mouse muscle in vivo, Science, 247, 1465, 10.1126/science.1690918 Acsadi, 1991, Direct gene transfer and expression into rat heart in vivo, New Biol, 3, 71 Acsadi, 1991, Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs, Nature, 352, 815, 10.1038/352815a0 Yang, 1990, In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment, 87, 9568 Parkman, 1986, The application of bone marrow transplantation to the treatment of genetic diseases, Science, 232, 1373, 10.1126/science.3520819 Golde, 1984 Van Beusechem, 1992, Prospects for human gene therapy Williams, 1984, Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse, Nature, 310, 476, 10.1038/310476a0 Dick, 1985, Introduction of a selectable gene in primitive stem cells capable of long-term reconstitution of the haematopoietic system of WWv mice, Cell, 42, 71, 10.1016/S0092-8674(85)80102-1 Keller, 1985, Expression of a foreign gene in myeloid and lymphoid cells derived from multipotent haematopoietic precursors, Nature, 318, 149, 10.1038/318149a0 Lemischka, 1986, Developmental potential and dynamic behavior of hematopoietic stem cells, Cell, 45, 917, 10.1016/0092-8674(86)90566-0 Valerio, 1986, The introduction of human ADA sequences into mouse haematopoietic stem cells, 335 Williams, 1988, Restriction of expression of an integrated recombinant retrovirus in primary but not in immortalized hematopoietic stem cells, Blood, 71, 1738, 10.1182/blood.V71.6.1738.1738 Williams, 1986, Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo, 83, 2566 Culver, 1991, Lymphocytes as cellular vehicles for gene therapy in mouse and man, 88, 3155 Culver, 1991, Lymphocyte gene therapy, Hum Gene Ther, 2, 107, 10.1089/hum.1991.2.2-107 Kasid, 1990, Human gene transfer: characterization of human tumor-infiltrating lymphocytes as vehicles for retroviral-mediated gene transfer in man, 87, 473 Culver, 1990, In vivo expression and survival of gene-modified T lymphocytes in rhesus monkeys, Hum Gene Ther, 1, 399, 10.1089/hum.1990.1.4-399 Fukayama, 1991, Respiratory tract gene transfer: transplantation of genetically modified T-lymphocytes directly to the respiratory epithelial surface, J Biol Chem, 266, 18339, 10.1016/S0021-9258(18)55275-3 Aebersold, 1990, Selection of gene-marked tumor infiltrating lymphocytes from post-treatment biopsies: a case study, Hum Gene Ther, 1, 373, 10.1089/hum.1990.1.4-373 Rosenberg, 1990, Gene transfer into humans-immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction, N Engl J Med, 323, 570, 10.1056/NEJM199008303230904 Gershon, 1990, First experiment approved, Nature, 346, 402, 10.1038/346402a0 Ledley, 1987, Retroviral gene transfer into primary hepatocytes: implications for genetic therapy of liver-specific functions, 84, 5335 Wilson, 1988, Retrovirus-mediated transduction of adult hepatocytes, 85, 3014 Wolff, 1987, Adult mammalian hepatocyte as target cell for retroviral gene transfer: a model for gene therapy, Somat Cell Mol Genet, 13, 423, 10.1007/BF01534943 Wolff, 1987, Expression of retrovirally expressed genes in primary cultures of adult rat hepatocytes, 84, 3344 Wilson, 1988, Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit, 85, 4421 Miyanohara, 1988, Efficient expression of retroviral vector-transduced human low density lipoprotein (LDL) receptor in LDL receptor-deficient rabbit fibroblasts in vitro, 85, 6538 Armentano, 1990, Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: potential for gene therapy of hemophilia B, 87, 6141 Demetriou, 1986, Survival, organization, and function of microcarrier-attached hepatocytes transplanted in rats, 83, 7475 Demetriou, 1986, Replacement of liver function in rats by transplantation of microcarrier attached hepatocytes, Science, 233, 1190, 10.1126/science.2426782 Demetriou, 1986, New method of hepatocyte transplantation and extracorporeal liver support, Ann Surg, 204, 259, 10.1097/00000658-198609000-00005 Vroemen, 1986, Hepatocyte transplantation for enzyme deficiency in congenic rats, Transplantation, 42, 130, 10.1097/00007890-198608000-00005 Vroemen, 1988, Transplantation of isolated hepatocytes into the pancreas, Eur surg Res, 20, 1, 10.1159/000128734 Anderson, 1989, Gene expression in implanted rat hepatocytes following retroviral-mediated gene transfer, Somat Cell Mol Genet, 15, 215, 10.1007/BF01534872 Fuller, 1988, Transplantation of isolated hepatocytes. A review of current ideas, J Hepatol, 7, 368, 10.1016/S0168-8278(88)80010-2 Thompson, 1989, Heparin-binding growth factor 1 induces the formation of organoid neovascular structures in vivo, 86, 7928 Thompson, 1988, Site-directed neovessel formation in vivo, Science, 241, 1349, 10.1126/science.2457952 Hatzoglou, 1990, Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase, J Biol Chem., 265, 17285, 10.1016/S0021-9258(17)44901-5 Kaleko, 1991, Persistent gene expression after retroviral gene transfer into liver cells in vivo, Hum Gene Ther, 2, 27, 10.1089/hum.1991.2.1-27 Ferry, 1991, Retroviral-mediated gene transfer into hepatocytes in vivo, 88, 8377 Kaneda, 1989, Increased expression of DNA cointroduced with protein in adult rat liver, Science, 243, 375, 10.1126/science.2911748 Wu, 1987, Receptor-mediated in vitro gene transformation by a soluble DNA carrier system, J Biol Chem, 262, 4429, 10.1016/S0021-9258(18)61209-8 Wu, 1988, Evidence for targeted gene delivery to HepG2 hepatoma cells in vitro, Biochemistry, 27, 887, 10.1021/bi00403a008 Zwiebel, 1989, High-level recombinant gene expression in rabbit endothelial cells transduced by retroviral vectors, Science, 244, 220, 10.1126/science.2911735 Axelrod, 1990, Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophilic dogs, 87, 5173 Nabel, 1989, Recombinant gene expression in vivo within endothelial cells of the arterial wall, Science, 244, 1342, 10.1126/science.2499928 Wilson, 1989, Implantation of vascular grafts lined with genetically modified endothelial cells, Science, 244, 1344, 10.1126/science.2734614 Dichek, 1989, Seeding of intravascular stents with genetically engineered endothelial cells, Circulation, 80, 1347, 10.1161/01.CIR.80.5.1347 Palmer, 1987, Efficient retrovirus-mediated transfer and expression of a human adenosine deaminase gene in diploid skin fibroblasts from an adenosine deaminase-deficient human, 84, 1055 Palmer, 1989, Production of human factor IX in animals by genetically modified skin fibroblasts: Potential therapy for hemophilia B, Blood, 73, 438, 10.1182/blood.V73.2.438.438 Hoeben, 1990, Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer, J Biol Chem, 265, 7318, 10.1016/S0021-9258(19)39116-1 Hoeben, 1989, Towards gene therapy in hemophilia A: expression of factor VIII with a retroviral vector system, Thromb Haemost, 62, 209 Sher, 1983, Acceptance of allogenic fibroblasts in skin equivalent transplants, Transplantation, 36, 552, 10.1097/00007890-198311000-00015 St.Louis, 1988, An alternative approach to somatic cell gene therapy, 85, 3150 Scharfmann, 1991, Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants, 88, 4626 Hoeben, 1991, Towards gene therapy in haemophilia A: Vectors for the expression of bloodclotting-factor VIII in vivo Selden, 1987, Regulation of insulin-gene expression.Implications for gene therapy, N Engl J Med, 317, 1067, 10.1056/NEJM198710223171706 Garver, 1987, Clonal gene therapy: transplanted mouse fibroblast clones express human alpha 1-antitrypsin gene in vivo, Science, 237, 762, 10.1126/science.3497452 Selden, 1987, Implantation of genetically engineered fibroblasts into mice: implications for gene therapy, Science, 236, 714, 10.1126/science.3472348 Tani, 1989, Implantation of fibroblasts transfected with human granulocyte colony-stimulating factor cDNA into mice as a model of cytokine-supplement gene therapy, Blood, 74, 1274, 10.1182/blood.V74.4.1274.1274 Fenjves, 1989, Systemic distribution of apolipoprotein E secreted by grafts of epidermal keratinocytes: implications for epidermal function and gene therapy, 86, 8803 Morgan, 1987, Expression of exogenous growth hormone gene by transplantable human epidermal cells, Science, 237, 1476, 10.1126/science.3629250 Flowers, 1990, Long-term transplantation of canine keratinocytes made resistant to G418 through retrovirus-mediated gene transfer, 87, 2349 Teumer, 1992, Human growth hormone in the blood of athymic mice grafted with cultures of hormone-secreting human keratinocytes, FASEB J, 4, 3245, 10.1096/fasebj.4.14.2227214 Partridge, 1991, Muscle transfection made easy, Nature, 352, 757, 10.1038/352757a0 Salminen, 1991, Implantation of recombinant rat myocytes into adult skeletal muscle—a potential gene therapy, Hum Gene Ther, 2, 15, 10.1089/hum.1991.2.1-15 Barr, 1991, Systemic delivery of recombinant proteins by genetically modified myoblasts, Science, 254, 1507, 10.1126/science.1962212 Dhawan, 1991, Systemic delivery of human growth hormone by injection of genetically engineered myoblasts, Science, 254, 1509, 10.1126/science.1962213 Drumm, 1990, Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer, Cell, 62, 1227, 10.1016/0092-8674(90)90398-X Garver, 1987, Production of glycosylated physiologically ‘normal’ human α1-antitrypsin by mouse fibroblasts modified by insertion of a human α1-antitrypsin cDNA using a retroviral vector, 84, 1050 Sorge, 1987, Complete correction of the genetic defect of type I Gaucher disease fibroblasts by retroviral-mediated gene transfer, 84, 906 Kohn, 1991, Toward gene therapy for Gaucher disease, Hum Gene Ther, 2, 101, 10.1089/hum.1991.2.2-101 Fink, 1990, Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease, 87, 2334 Dichek, 1991, Retroviral vector-mediated in vivo expression of low-density-lipoprotein receptors in the Watanabe heritable hyperlipidemic rabbit, Somat Cell Mol Genet, 17, 287, 10.1007/BF01232823 Harrison, 1991, Activation of a diphtheria toxin-A gene by expression of human immunodeficiency virus-1 Tat and Rev proteins in transfected cells, Hum Gene Ther, 2, 53, 10.1089/hum.1991.2.1-53 Sarver, 1990, Ribozymes as potential anti-HIV-1 therapeutic agents, Science, 247, 1222, 10.1126/science.2107573 Wolff, 1989, Grafting fibroblasts genetically modified to produce L-dopa in a rat model of Parkinson disease, 86, 9011 Rosenberg, 1990, Adoptive immunotherapy for cancer, Sci Am, 262, 62, 10.1038/scientificamerican0590-62 Giblett, 1972, Adenosine deaminase deficiency in two patients with severe impaired cellular immunity, Lancet, ii, 1067, 10.1016/S0140-6736(72)92345-8 Kredich, 1983, Immunodeficiency diseases caused by adenosine deaminase and purine nucleoside phosphorylase deficiency, 1157 Kellems, 1985, Adenosine deaminase deficiency and severe combined immunodeficiencies, Trends Genet, 1, 278, 10.1016/0168-9525(85)90105-2 Fischer, 1986, Bone-marrow transplantation for immunodeficiencies and osteoporosis: European survey, 1968–1985, Lancet, II, 1080, 10.1016/S0140-6736(86)90477-0 Hershfield, 1987, Treatment of ADA deficiency with polyethylene glycol-modified ADA, N Engl J Med, 316, 589, 10.1056/NEJM198703053161005 Hershfield, 1989, ADA deficiency treatment, Science, 246, 1375, 10.1126/science.246.4936.1375-a Dooren, 1985, Severe combined immunodeficiency: Reconstitution of immune system following bone marrow transplantation, 3, 351 Hirschhorn, 1977, Defects in purine metabolism in immunodeficiency diseases, 67 Kantoff, 1986, Correction of adenosine deaminase deficiency in cultured human T and B cells by retrovirus-mediated gene transfer, 83, 6563 Braakman, 1992, Genetic correction of cultured T-cells from an adenosine deaminase deficient patient: Characteristics of non-transduced and transduced T-cells, Eur J Immunol, 22, 63, 10.1002/eji.1830220111 Apperley, 1991, Retroviral gene transfer of human adenosine deaminase in murine hematopoietic cells: effect of selectable marker sequences on long-term expression, Blood, 78, 310, 10.1182/blood.V78.2.310.310 Lim, 1989, Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells, 86, 8892 Moore, 1989, Expression vectors for human adenosine deaminase gene therapy, Genome, 31, 832, 10.1139/g89-146 Osborne, 1990, Long-term expression of human adenosine deaminase in mice after transplantation of bone marrow infected with amphotropic retroviral vectors, Hum Gene Ther, 1, 31, 10.1089/hum.1990.1.1-31 van Beusechem, 1990, Expression of human adenosine deaminase in mice transplanted with hemopoietic stem cells with amphotropic retroviruses, J Exp Med, 172, 729, 10.1084/jem.172.3.729 Brettler, 1989, Factor concentrates for treatment of hemophilia: which one to choose?, Blood, 73, 2067, 10.1182/blood.V73.8.2067.2067 Pierce, 1989, The use of purified clotting factor concentrates in hemophilia: influence of viral safety, cost, and supply on therapy, JAMA, 261, 3434, 10.1001/jama.261.23.3434 Smit, 1989, Physical condition, longevity and social performance of Dutch haemophiliacs. 1973–1985, Br Med J, 298, 235, 10.1136/bmj.298.6668.235 Goldsmith, 1986, Hemophilia: beaten on one front, is beset on others, JAMA, 256, 3200, 10.1001/jama.256.23.3200 Anson, 1987, Towards gene therapy for hemophilia B, Mol Biol Med, 4, 11 Yao, 1991, Expression of human factor IX in rat capillary endothelial cells: Toward somatic gene therapy for hemophilia B, 88, 8101 Evans, 1989, Molecular cloning of a cDNA encoding canine factor IX, Blood, 74, 207, 10.1182/blood.V74.1.207.207 Evans, 1989, Canine hemophilia B resulting from a point mutation with unusual consequences, 86, 10095 Verma, 1990, Gene therapy, Sci Amer, 263, 68, 10.1038/scientificamerican1190-68 Verma, 1990, Gene therapy, Sci Amer, 263, 81, 10.1038/scientificamerican1190-68 Israel, 1990, Retroviral-mediated transfer and amplification of a functional human factor VIII gene, Blood, 75, 1074, 10.1182/blood.V75.5.1074.1074 Rafield, 1990, Expression and secretion of human factor VIII by canine endothelial cells and mouse fibroblasts using retroviral-mediated gene transfer, Blood, 76, 434a Lynch, 1990, Towards somatic cell gene therapy using endothelial cells, J Cell Biochem, 14E, 222 Van Ormondt, 1991, Toward gene therapy in hemophilia A: Expression of factor VIII in human primary skin fibroblasts, 1, 47 Hoeben, 1992, Toward gene therapy for haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into haematopoietic progenitor cells, Thromb Haemost, 67, 341, 10.1055/s-0038-1648444 Kaufman, 1988, Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells, J Biol Chem, 263, 6352, 10.1016/S0021-9258(18)68793-9 Kaufman, 1990, Factors limiting biosynthesis and secretion of factor VIII in mammalian cells, 63 Kaufman, 1989, Genetic engineering of factor VIII, Nature, 342, 207, 10.1038/342207a0 Kaufman, 1991, Insight into the structure, function, and biosynthesis of factor VIII through recombinant factor VIII technology, Ann Hematol, 63, 155, 10.1007/BF01703248 Grosveld, 1987, Position-independent high-level expression of the human β-globin gene in transgenic mice, Cell, 51, 975, 10.1016/0092-8674(87)90584-8 Novak, 1990, High level beta-globin expression after retroviral transfer of locus activation-region containing human beta-globin gene derivatives into murine erythroleukemia cells, 87, 3386 Greaves, 1989, Human CD2 3′-flanking sequences confer high-level, T-cell-specific, position-independent gene expression in transgenic mice, Cell, 56, 979, 10.1016/0092-8674(89)90631-4 Bonifer, 1990, Tissue specific and position independent expression of the complete gene domain for chicken lysozyme in transgenic mice, EMBO J, 9, 2843, 10.1002/j.1460-2075.1990.tb07473.x Miller, 1989, Improved retroviral vectors for gene transfer and expression, Biotechniques, 7, 980 Sorge, 1984, Amphotropic retrovirus vector system for human cell gene transfer, Mol Cell Biol, 4, 1730, 10.1128/MCB.4.9.1730 Miller, 1986, Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production, Mol Cell Biol, 6, 2895, 10.1128/MCB.6.8.2895 Dougherty, 1989, New retrovirus helper cells with almost no nucleotide sequence homology to retrovirus vectors, J Virol, 63, 3209, 10.1128/JVI.63.7.3209-3212.1989 Miller, 1991, Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus, J Virol, 65, 2220, 10.1128/JVI.65.5.2220-2224.1991 Van Beusechem, 1992, Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone marrow cells