Abstract
Other chapters in this volume have focused on gene transfer approaches for inhibition of immune responses against autoantigens. This chapter will discuss treatment of inflammatory responses directed against alloantigens, as a result of allogeneic transplantation of solid parenchymal organs or dispersed cells. From an immunological standpoint, autoimmunity and alloimmunity share many of the same basic mechanisms of lymphocyte activation and recruitment, reliance on clonal and nonclonal receptors to mediate cellular communication, participation of cytokines and adhesion receptors in diverse aspects of lymphocyte function, and effector mechanisms involving CD4+ and CD8+ T cells, B cells and macrophages [1]. The major difference between the two types of responses is that major histocompatibility complex (MHC) antigens are responsible for the vast majority of alloresponses, while autoimmunity for the most part involves responses to non-MHC antigens. Functional consequences of these differences are that MHC antigens can be presented directly to class I or class II allorestricted CD8+ or CD4+ T cells, respectively, or processed MHC-derived peptides can be represented indirectly to self-restricted, alloreactive T cells. In contrast, autoantigens are usually presented in the context of self MHC, often class II, to autoreactive CD4+ T cells. While these differences are clearly protean in nature and have enormous significance for the understanding and control of autoreactive and alloreactive immune responses, they have not resulted in clinically applicable experimental approaches or practical molecular interventions that substantially differentiate or affect one type of immune response in preference to the other. As a result, many of the approaches and considerations that are germane for control of alloimmunity are equally applicable to autoimmunity.
Keywords
- Major Histocompatibility Complex
- Gene Therapy
- Gene Transfer
- Major Histocompatibility Complex Class
- Adenoviral Vector
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.
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References
Bromberg JS (1997) Transplant immunology. In: Surgery scientific principles and practice. 2nd ed. Lippincott-Raven, Philadelphia, PA, 527–555
Muruve DA, Manfro RC, Strom TB, Libermann TA (1997) Ex vivo adenovirus-mediated gene delivery leads to long-term expression in pancreatic islet transplants. Transplantation64: 542–546
Qin L, Ding Y, Pahud DR, Robson ND, Shaked A, Bromberg JS (1997) Adenovirus-mediated gene transfer of viral interleukin-10 inhibits the immune response to both alloantigen and adenoviral antigen. Hum Gene Ther 8: 1365–1374
Gruber SA (1996) Local immunosuppression of organ transplants. RG Landes Company and Chapman & Hall, Austin, TX
Bromberg JS, Qin L (1996) Gene transfer to the transplanted organ. In: Local immunosuppression of organ transplants. RG Landes Company and Chapman & Hall, Austin, TX, 169–180
Shaked A, Csete ME, Shiraishi M, Miller AR, Moen RM, Busuttil RW, Economou JS (1994). Retroviral-mediated gene transfer into rat experimental liver transplant. Transplantation 57: 32–34
Csete ME, Benhamou PY, Drazan KE, Wu L, Mclntee DF, Afra R, Mullen Y, Busuttil RW, Shaked A (1995) Efficient gene transfer to pancreatic islets mediated by adenoviral vectors. Transplantation 59: 263–268
Drazan KE, Wu L, Olthoff KM, Jurim O, Busuttil RW, Shaked A (1995) Transduction of hepatic allografts achieves local levels of viral IL-10 which suppress alloreactivity in vitro. J Surg Res 59: 219–223
Nabel EG, Plautz G, Nabel GJ (1990) Site-specific gene expression in vivo by direct gene transfer into the arterial wall. Science 249: 1285–1288
Mehra MR, Stapleton DD, Cook JL, Zhang T, Ventura HO, Huang C, Maldonado B, Smart FW, Ré RN, Murgo JP, Barbee RW (1996) Adenovirus-mediated in vivo gene transfer in a rabbit model of allograft vasculopathy. J Heart Lung Transplant 15: 51–57
Hullett DA (1996) Gene therapy in transplantation. J Heart Lung Transplant 15: 857–862
Knechtle SJ (1996) Gene therapy and transplantation-A brief review. Transplant Proc 28: 19–23
Wang J, Jiao S, Wolff JA, Knechtle SJ (1992) Gene transfer and expression in rat cardiac transplants. Transplantation 53: 703–705
Wang J, Ma Y, Knechtle SJ (1996) Adenovirus-mediated gene transfer into rat allografts. Transplantation 61: 1726–1729
Csete ME, Drazan KE, Van Bree M, Mclntee DF, McBride WH, Bett A, Graham FL, Busuttil RW, Berk AJ, Shakad A (1994) Adenovirus-mediated gene transfer in the transplant setting. Transplantation 57: 1502–1507
Donahue JK, Kikkawa K, Johns DC, Marban E, Lawrence JH (1997) Ultrarapid, highly efficient viral gene transfer to the heart. Proc Natl Acad Sci USA 94: 4664–4668
Barr E, Leiden JM (1991) Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254: 1507–1509
Shaked A, Csete ME, Drazan KE, Bullington D, Wu L, Busuttil RW, Berk AJ (1994) Ade-novirus-mediated gene transfer in the transplant setting. Transplantation 57: 1508–1511
Drazan KE, Wu L, Shen X, Bullington D, Jurim O, Busuttil RW, Shaked A (1995) Ade-novirus-mediated gene transfer in the transplant setting. Transplantation 59: 670–673
Lieber A, Vrancken Peeters MTFD, Meuse L, Fausto N, Perkins J, Kay MA (1995) Ade-novirus-mediated urokinase gene transfer induces liver regeneration and allows for efficient retrovirus transduction of hepatocytes in vivo. Proc Natl Acad Sci USA 92: 6210–6214
Lieber A, Vrancken Peeters MTFD, Gown A, Perkins J, Kay MA (1995) A modified urokinase plasminogen activator induces liver regeneration without bleeding. Hum Gene Ther 6: 1029–1037
Bosch A, McCray Jr., Chang SMW, Ulich TR, Simonet WS, Jolly DJ, Davidson BL (1996) Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes. J Clin Invest 98: 2683–2687
Raper SE (1995) Hepatocyte transplantation and gene therapy. Clin Transplantation9: 249–254
Halbert CL, Standaert TA, Aitken ML, Alexander IE, Russell DW, Miller D (1997) Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration. J Virology 71: 5932–5941
Yang Y, Nunes FA, Berencsi K, Furth EE, Gönczöl E, Wilson JM (1994) Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 91: 4407–4411
Riddell SR, Elliott M, Lewinsohn DA, Gilbert MJ, Wilson L, Manley SA, Lupton SD, Overell RW, Reynolds TC, Corey L, Greenberg P (1996) T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nature Med 2: 216–223
Tripathy SK, Black HB, Goldwasser E, Leiden JM (1996) Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med 2: 545–550
Kay MA, Meuse L, Gown AM, Linsley P, Hollenbaugh D, Aruffo A, Ochs HD, Wilson CB (1997) Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver. Proc Natl Acad Sci USA 94: 4686–4691
Kay MA, Holterman A, Meuse L, Gown A, Ochs HD, Linsley PS, Wilson CB (1995) Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration. Nature Genetics 11: 191–197
DeMatteo RP, Markmann KF, Kozarsky KF, Barker CF, Raper SE (1996) Prolongation of adenoviral transgene expression in mouse liver by lymphocyte subset depletion. Gene Therapy3: 4–12
Lochmüller H, Petrof BJ, Allen C, Prescott AS, Massie B, Karpati G (1995) Immunosuppression by FK506 markedly prolongs expression of adenovirus-delivered transgene in skeletal muscles of adult dystrophic [mdx] mice. Biochem Biophys Res Comm 213: 569–574
Vemuru RP, Davidson A, Aragona E, Chowdhury JR, Burk RD, Gupta S (1992) Immune tolerance to a defined heterologous antigen after intrasplenic hepatocyte transplantation: implications for gene therapy. FASEB J 6: 2836–2842
Boasquevisque CHR, Mora BN, Schmid RA, Lee TC, Nagahiro I, Cooper JD, Patterson GA (1997) Ex vivo adenoviral-mediated gene transfer to lung isografts during cold preservation. Ann Thorac Surg 63: 1556–1561
Colson YL, Lange J, Fowler K, Ildstad ST (1996) Mechanism for cotolerance in non-lethally conditioned mixed chimeras: negative selection of the V beta T-cell receptor repertoire by both host and donor bone marrow-derived cells. Blood 88: 4601–4610
Delaney CP, Murase N, Chen-Woan M, Fung JJ, Starzl TE, Demetris AJ (1996) Allogeneic hematolymphoid microchimerism and prevention of autoimmune disease in the rat. A relationship between allo-and autoimmunity. J Clin Invest 97: 217–225
Garcia-Morales R, Carreno M, Mathew J, Zucker K, Cirocco R, Ciancio G, Burke G, Roth D, Temple D, Rosen A et al (1997) The effects of chimeric cells following donor bone marrow infusions as detected by PCR-flow assays in kidney transplant recipients. J Clin Invest 99: 1118–1129
Eglitis MA, Kantoff P, Gilboa E, Anderson WF (1985) Gene expression in mice after high efficiency retroviral-mediated gene transfer. Science 230: 1395–1398
Keller G, Paige C, Gilboa E, Wagner EF (1985). Expression of a foreign gene in myeloid and lymphoid cells derived from multipotent haematopoietic precursors. Nature 318: 149–154
Snodgrass R, Keller G (1987) Clonal fluctuation within the haematopoietic system of mice reconstituted with retrovirus-infected stem cells. EMBO J 6: 3955–3960
Bowtell DDL, Johnson GR, Kelso A, Cory S (1987) Expression of genes transferred to hemopoietic stem cells by recombinant retroviruses. Mol Biol Med 4: 229–250
Chen BP, Fraser C, Reading C, Murray L, Uchida N, Galy A, Sasaki D, Tricot G, Jagannath S, Barlogie B et al (1995) Cytokine-mobilized peripheral blood CD34+Thy-1+Lin-human hematopoietic stem cells as target cells for transplantation-based gene therapy. Leukemia 9: S17–S25
Dunbar CE, Cottier-Fox M, O’Shaughnessy JA, Doren S, Carter C, Bereson R, Brown S, Moen RC, Greenblatt J, Stewart FM et al (1995) Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85: 3048–3057
Madsen JC, Superina RA, Wood KJ, Morris PJ (1988) Immunological unresponsiveness induced by recipient cells transfected with donor MHC genes. Nature 332: 161–164
Sykes M, Sachs DH, Nienhuis AW, Pearson DA, Moulton AD, Bodine DM (1993) Specific prolongation of skin graft survival following retroviral transduction of bone marrow with an allogeneic major histocompatibility complex gene. Transplantation 55: 197–202
Emery DW, Smith CV, Shafer GE, Karson EM, Sachs DH, LeGuern C (1993) Expression of allogeneic class II cDNA in swine peripheral blood mononuclear cells following retroviral-mediated gene transfer into bone marrow. Transplant Proc 25: 140–141
Emery DW, Shafer GE, Karson EM, Sachs DH, LeGuern C (1993) Retrovirus-mediated transfer and expression of an allogeneic major histocompatibility complex class II DRB cDNA in swine bone marrow cultures. Blood 81: 2460–2465
Emery DW, Sablinski T, Arn JS, LeGuern C, Sachs DH (1994) Bone marrow culture and transduction of stem cells in a miniature swine model. Blood Cells 20: 498–503
LeGuern C, Shimada H, Emery DW, Germana S, Shafer GE, Sachs DH (1995) Retrovirus-mediated transfer of MHC class II cDNA into swine bone marrow cells. J Mol Med73: 269–278
Fraser CC, Sykes M, Stanton Lee R, Sachs DH, LeGuern C (1995) Specific unresponsiveness to a retrovirally-transferred class I antigen is controlled through the helper pathway. J Immunol 154: 1587–1595
Chahine AA, Yu M, McKernan MM, Stoeckert C, Lau HT (1995) Immunomodulation of pancreatic islet allografts in mice with CTLA4Ig secreting muscle cells. Transplantation 59: 1313–1318
Carr-Brendel VE, Geller RL, Thomas TJ, Boggs DR, Young SK, Crudele J, Martinson LA, Maryanov DA, Johnson RC, Brauker JH (1997) Transplantation of cells in an immunoisolation device for gene therapy. Methods Mol Biol 63: 373–387
Ilan Y, Prakash R, Davidson A, Jona V, Droguett G, Horwitz MS, Chowdhury NR, Chowdhury JR (1997) Oral tolerization to adenoviral antigens permits long-term gene expression using recombinant adenoviral vectors. J Clin Invest 99: 1098–1106
DeMatteo RP, Raper SE, Ahn M, Fisher KJ, Burke C, Radu A, Wider G, Claytor BR, Barker CF, Markmann JF (1995) Gene transfer to the thymus a means of abrogating the immune response to recombinant adenovirus. Annals Surgery 222: 229–242
Ilan Y, Attavar P, Takahashi M, Davidson A, Horwitz MS, Guida J, Chowdhury NR, Chowdhury JR (1996) Induction of central tolerance by intrathymic inoculation of adenoviral antigens into the host thymus permits long-term gene therapy in Gunn rats. J Clin Invest 98: 2640–2647
Nakano N, Rooke R, Benoist C, Mathis D (1997) Positive selection of T cells induced by viral delivery of neopeptides to the thymus. Science 275: 678–683
DeMatteo RP, Chu G, Ahn M, Chang E, Barker CF, Markmann JF (1997) Long-lasting adenovirus transgene expression in mice through neonatal intrathymic tolerance induction without the use of immunosuppression. J Virol 71: 5330–5335
Knechtle SJ, Wang J, Jiao S, Geissler EK, Sumimoto R, Wolff J (1994) Induction of specific tolerance by intrathymic injection of recipient muscle cells transfected with donor class I major histocompatibility complex. Transplantation 57: 990–996
Knechtle SJ, Wang J, Graeb C, Zhai Y, Hong X, FechnerJr. Geissler EK (1997) Direct MHC class I complementary DNA transfer to thymus induces donor-specific unresponsiveness, which involves multiple immunologic mechanisms. J Immunol 159: 152–158
Knechtle SJ, Zhai Y, Fechner J.(1996) Gene therapy in transplantation. Transplant Immunology 4: 257–264
Drazan KE, Olthoff KM, Wu L, Shen X, Gelman A, Shaked A (1996) Adenovirus-mediated gene transfer in the transplant setting. Transplantation 62: 1080–1084
Qin L, Ding Y, Bromberg JS (1996) Gene transfer of transforming growth factor-β1 prolongs murine cardiac allograft survival by inhibiting cell mediated immunity. Hum Gene Ther 7: 1981–1988
Qin L, Chavin KD, Ding Y, Favarro JP, Woodward JE, Lin J, Tahara H, Robbins P, Shaked A et al (1995) Multiple vectors effectively achieve gene transfer in a murine cardiac transplantation model: immunosuppression with TGF-β1 or vIL-10. Transplantation 59: 809–816
Levy AE, Alexander JW (1995) Administration of intragraft interleukin-4 prolongs cardiac allograft survival in rats treated with donor-specific transfusion/cyclosporine. Transplantation 60: 405–406
Mueller R, Krahl T, Sarvetnick N (1996) Pancreatic expression of interleukin-4 abrogates insulitis and autoimmune diabetes in nonobese diabetic (NOD) mice. J Exp Med 184: 1093–1099
Sarvetnick N (1996) Mechanisms of cytokine-mediated localized immunoprotection. J Exp Med 184: 1597–1600
Benhamou PY, Mullen Y, Shaked A, Bahmiller D, Csete ME (1996) Decreased alloreactivity to human islets secreting recombinant viral interleukin 10. Transplantation 62: 1306–1312
Qin L, Chavin KD, Tahara H, Ding Y, Favarro J, Woodward J, Lin J, Robbins PD, Lotze MT, Bromberg JS (1996) Retrovirus-mediated transfer of viral interleukin-10 gene prolongs murine cardiac allograft survival. J Immunol 156: 2316–2323
Kolls J, Peppel K, Silva M, Beutler B (1994) Prolonged and effective blockade of tumor necrosis factor activity through adenovirus-mediated gene transfer. Proc Natl Acad Sci USA 91: 215–219
Rogy MA, Auffenberg T, Espat NJ, Philip R, Remick D, Wollenberg GK, Copeland III EM, Moldawer L (1995) Human tumor necrosis factor receptor (p55) and interleukin 10 gene transfer in the mouse reduces mortality to lethal endotoxemia and also attenuates local inflammatory responses. J Exp Med 181: 2289–2293
Grewal IS, Grewal KD, Wong FS, Picarells DE, Janeway Jr, Flavell RA (1996) Local expression of transgene encoded TNFα in islets prevents autoimmune diabetes in nonobese diabetic (NOD) mice by preventing the development of auto-reactive islet-specific T cells. J Exp Med 184: 1963–1974
Tufariello J, Cho S, Horwitz MS (1994) The adenovirus E3 14.7-kilodalton protein which inhibits cytolysis by tumor necrosis factor increases the virulence of vaccinia virus in a murine pneumonia model. J Virol 68: 453–462
Tufariello J, Cho S, Horwitz MS (1994) Adenovirus E3 14.7-kilodalton protein, an antagonist of tumor necrosis factor cytolysis, increases the virulence of vaccinia virus in severe combined immunodeficient mice. Proc Natl Acad Sci USA 91: 10987–10991
Li Y, Kang J, Horwitz MS (1997) Interaction of an adenovirus 14.7-kilodalton protein inhibitor of tumor necrosis factor alpha cytolysis with a new member of the GTPase superfamily of signal transducers. J Virol 71: 1576–1582
Efrat S, Fejer G, Brownlee M, Horwitz MS (1995) Prolonged survival of pancreatic islet allografts mediated by adenovirus immunoregulatory transgenes. Proc Natl Acad Sci USA 92: 6947–6951
Ilan Y, Droguett G, Chowdhury NR, Li Y, Sengupta K, Thummala NR, Davidson A, Chowdhury JR, Horwitz MS (1997) Insertion of the adenoviral E3 region into a recombinant viral vector prevents antiviral humoral and cellular immune responses and permits long-term gene expression. Proc Natl Acad Sci USA 94: 2587–2592
Gainer AL, Korbutt GS, Rajotte RV, Warnock GL, Elliott JF (1994) Expression of CTLA4-Ig by biolistically transfected mouse islets promotes islet allograft survival. Transplantation 63: 1017–1044
Kato K, Shimozato O, Hoshi K, Wakimoto H, Hamada H, Yagita H, Okumura K (1996) Local production of the p40 subunit of interleukin 12 suppresses T-helper 1-mediated immune responses and prevents myoblast rejection. Proc Natl Acad Sci USA 93: 9085–9089
Chen S, Wilson JM, Vallance DK, Hartman JW, Davidson BL, Roessler BJ (1995) A recombinant adenoviral vector expressing a soluble form of VCAM-1 inhibits VCAM-l/VLA-4 adhesion in transduced synoviocytes. Gene Therapy 2: 469–480
Grunhaus A, Cho S, Horwitz MS (1994) Association of vaccinia virus-expressed adenovirus E3-19K glycoprotein with Class I MHC and its effects on virulence in a murine pneumonia model. Virology 200: 535–546
Bellgrau D (1995) A role for CD95 ligand in preventing graft rejection. Nature 377: 630–632
Griffith TS, Brunner T, Fletcher SM, Green DR, Ferguson TA (1995) Fas ligand-induced apoptosis as a mechanism of immune privilege. Science 270: 1189–1192
Lau HT, Yu M, Fontana A, Stoeckert CJ (1996) Prevention of islet allograft rejection with engineered myoblasts expressing FasL in mice. Science 273: 109–112
Kang S (1997) Fas ligand expression in islets of langerhans does not confer immune privilege and instead targets them for rapid destruction. Nature Med 3: 738–743
Chervonsky AV (1997) The role of Fas in autoimmune diabetes. Cell 89: 17–24
Allison J, Georgiou HM, Strasser A, Vaux DL (1997) Transgenic expression of CD95 ligand on islet beta cells induces a granulocytic infiltrate but does not confer immune privilege upon islet allograft. Proc Natl Acad Sci USA 94: 3943–3947
Seino K, Kayagaki N, Okumura K, Yagita H (1997) Antitumor effect of locally produced CD95 ligand. Nature Med 3: 165–170
Simons M, Edelman ER, DeKeyser J, Langer R, Rosenberg RD (1992) Antisense c-myb oligonucleotides inhibit intimal arterial smooth muscle cell accumulation in vivo. Nature 359: 67–70
Morishita R, Gibbons GH, Ellison KE, Nakajima M, Zhang L, Kaneda K, Ogihara T, Dzau V (1993) Single intraluminal delivery of antisense cdc2 kinase and proliferating-cell nuclear antigen oligonucleotides results in chronic inhibition of neointimal hyperplasia. Proc Natl Acad Sci USA 90: 8474–8478
Ohno T, Gordon D, San H, Pompili VJ, Imperiale MJ, Nabel GJ, Nabel EG (1994) Gene therapy for vascular smooth muscle cell proliferation after arterial injury. Science 265: 781–784
Chang MW, Barr E, Seltzer J, Jiang Y, Nabel GJ, Nabel EG, Parmacek MS, Leiden JM (1995) Cytostatic gene therapy for vascular proliferative disorders with a constitutively active form of the retinoblastoma gene product. Science 267: 518–522
Von Der Leyen HE, Gibbons GH, Morishita R, Lewis NP, Zhang L, Nakajima M, Kaneda Y, Cooke JP, Dzau V (1995) Gene therapy inhibiting neointimal vascular lesion: In vivo transfer of endothelial cell nitric oxide synthase gene. Proc Natl Acad Sci USA 92: 1137–1141
Mann MJ, Gibbons GH, Tsao PS, Von Der Leyen HE, Cooke JP, Buitrago R, Kernoff R, Dzau VJ (1997) Cell cycle inhibition preserves endothelial function in genetically engineered rabbit vein grafts. J Clin Invest 99: 1295–1301
Yang Y, Ertl HCJ, Wilson JM (1994) MHC class I restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1 deleted recombinant adenoviruses. Immunity 1: 433–442
Dai Y, Schwarz EM, Gu D, Zhang WW, Sarvetnick N, Verma IM (1995) Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 92: 1401–1405
Doherty PC, Allen W, Eichelberger M, Carding SR (1992) Roles of αβ and γδ T cell subsets in viral immunity. Annu Rev Immunol 10: 123–151
Yang Y, Li Q, Ertl HCJ, Wilson JM (1995) Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 69: 2004–2015
Yang Y, Su Q, Wilson JM (1996) Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs. J Virol 70: 7209–7212
Yang Y, Trinchieri G, Wilson JM (1995) Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung. Nature Med 1: 890–893
Yang Y, Xiang Z, Etrl HCJ, Wilson JM (1995) Upregulation of class I MHC antigens by interferon-γ is necessary for the T cell mediated elimination of recombinant adenovirus infected hepatocytes in vivo. Proc Natl Acad Sci USA 92: 7257–7261
Acsadi G, Lochmuller H, Jani A, Huard J, Massie B, Prescott S, Simoneau M, Petrof BJ, Karpati G (1996) Dystrophin expression in muscles of mdx mice after adenovirus-medi-ated in vivo gene transfer. Hum Gene Ther 7: 129–140
Zsengeller Z, Wert S, Hull W, Hu X, Yei S, Trapnell B, Whitsett J (1995) Persistence of replication-deficient adenovirus-mediated gene transfer in lungs of immune-deficient (nu/nu) mice. Hum Gene Ther 6: 457–467
Pilewski JM, Scott DJ, Wilson JM, Albelda SM (1995) ICAM-1 expression on bronchial epithelium after recombinant adenovirus infection. Am J Respir Cell Mol Biol 12: 142–148
Stark JM, Amin RS, Trapnell BC (1996) Infection of A549 cells with a recombinant adenovirus vector induces ICAM-1 expression and increased CD-18-dependent adhesion of activated neutrophils. Hum Gene Ther 7: 1669–1681
Amin R, Wilmott R, Schwarz Y, Trapnell B, Stark J (1995) Replication-deficient adenovirus induces expression of interleukin-8 by airway epithelial cells in vitro. Hum Gene Ther 6: 145–153
Worgall S, Wolff G, Falck-Pedersen E, Crystal RG (1997) Innate immune mechanisms dominate elimination of adenoviral vectors following in vivo administration. Hum Gene Ther8: 37–44
Yei S, Mittereder N, Wert S, Whitsett JA, Wilmott RW, Trapnell BC (1994) In vivo evaluation of the safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator DNA to lung. Hum Gene Ther 5: 733–746
Newman KD, Dunn PF, Owens JW, Schulick AH, Virmani R, Sukhova G, Libby P, Dichek DA (1995) Adenovirus-mediated gene transfer into normal rabbit arteries results in prolonged vascular cell activation, inflammation, and neointimal hyperplasia. J Clin Invest 96: 2955–2965
Vilquin JT, Guerette B, Kinoshita I, Roy B, Goulet M, Gravel C, Roy R, Tremblay JP (1995) FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer. Hum Gene Ther 6: 1391–1401
Gordon EM, Anderson WF (1994) Gene therapy using retroviral vectors. Curr Opin Biotech 5: 611–616
Gunzburg WH, Salmons B (1996) Development of retroviral vectors as safe, targeted gene delivery systems. J Mol Med 74: 171–182
Schofield JP, and Caskey CT (1995) Non-viral approaches to gene therapy. Br Med Bull 51: 56–71
Yamamoto S, Yamamoto T, Kataoka T, Kuramoto E, Yano O, Tokunaga T (1992) Unique palindromic sequences in synthetic oligonucleotides are required to induce IFN and augment INF-mediated natural killer activity. J Immunol 148: 4072–4076
Yamamoto T, Yamamoto S, Kataoka T, Komuro K, Kohase M, Tokunaga T (1994) Synthetic oligonucleotides with certain palindromes stimulate interferon production of human peripheral blood lymphocytes in vitro. Jpn J Cancer Res 85: 775–779
Tokunaga T, Yano O, Kuramoto E, Kimura Y, Yamamoto T, Kataoka T, Yamamoto S (1992) Synthetic oligonucleotides with particular base sequences from the cDNA encoding proteins of mycobacterium bovis BCG induce interferons and activate natural killer cells. Microbiol Immunol 36: 55–66
Ballas ZK, Rasmussen WL, Krieg AM (1996) Induction of NK activity in murine and human cells by CpG motifs in oligodeoxynucleotides. J Immunol 157: 1840–1845
Yi AK, Klinman DM, Martin TL, Matson S, Krieg AM (1996) Rapid immune activation by CpG motifs in bacterial DNA. J Immunol 157: 5394–5402
Yi AK, Chace JH, Cowdery JS, Krieg AM (1996) IFN-g promotes IL-6 and IgM secretion in response to CpG motifs in bacterial DNA oligodeoxynucleotides. J Immunol 156: 558–564
Yamamoto T, Yamamoto S, Kataoka T, Tokunaga T (1994) Lipofection of synthetic oligodeoxyribonucleotide having a palindromic sequence of AACGTT to murine splenocytes enhances interferon production and natural killer activity. Microbiol Immunol 38: 831–836
Kuramoto E, Yano O, Kimura Y, Baba M, Makino T, Yamamoto S, Yamamoto T, Kataoka T, Tokunaga T (1992) Oligonucleotide sequences required for natural killer cell activation. Jpn J Cancer Res 83: 1128–1131
Klinman DM, Yi AK, Beaucage SL, Conover J, Krieg AM (1996) CpG motifs present in bacterial DNA rapidly induce lymphocytes to secrete interleukin 6, interleukin 12, and interferon-γ. Proc Natl Acad Sci USA 93: 2879–2883
Stacey KJ, Sweet MJ, Hume DA (1996) Macrophages ingest and are activated by bacterial DNA. J Immunol 157: 2116–2122
Sato Y, Roman M, Tighe H, Lee D, Corr M, Nguyen MD, Silverman GJ, Lotz M, Carson DA, Raz E (1996) Immunostimulatory DNA sequences necessary for effective intradermal gene immunization. Science 273: 352–354
Gooding LR (1992) Virus proteins that counteract host immune defenses. Cell 71: 5–7
Kotwal G (1996) The great escape: immune evasion by pathogens. The Immunologist 4/5: 157–164
Bromberg JS, DeBruyne LA, Qin L (1997) Interactions between the immune system and gene therapy vectors. Bidirectional regulation of response and expression. Adv Immunol 69: 353–409
Hsu DH, De Waal Malefyt R, Fiorentino DF, Dang MN, Vieira P, De Vries J, Spits H, Mosmann TR, Moore KW (1990) Expression of interleukin-10 activity by Epstein-Barr virus protein BCRF1. Science 250: 830–832
de Waal Malefyt R, Haanen J, Spits H, Roncarlol MG, te Velde A, Figdor C, Johnson C, Kastelein R, Yssel H, de Vries JE (1991) Interleukin 10 (IL-10) and viral IL-10 strongly reduce antigen-specific human T cell proliferation by diminishing the antigen-presenting capacity of monocytes via down-regulation of class II major histocompatibility complex expression. J Exp Med 174: 915–924
Vieira P, de Waal Malefyt R, Dang MN, Johnson KE, Kastelein R, Fiorentino DF, de Vries JE, Roncarolo MG, Mosmann TR, and Moore KW (1991) Isolation and expression of human cytokine synthesis inhibitory factor cDNA clones: homology to Epstein-Barr virus open reading frame BCRFI. Proc Natl Acad Sci USA 88: 1172–1176
Suzuki T, Tahara H, Narula S, Moore KW, Robbins PD, Lotze MT (1995) Viral interleukin 10 (IL-10), the human herpes virus 4 cellular IL-10 homologue, induces local anergy to allogeneic and syngeneic tumors. J Exp Med 182: 477–486
Routes JM, Metz BA, Cook JL (1993) Endogenous expression of E1A in human cells enhances the effect of adenovirus E3 on class I major histocompatibility complex antigen expression. J Virol 67: 3176–3181
Burgert HG, Kvist S (1985) An adenovirus type 2 glycoprotein blocks cell surface expression of human histocompatibility class I antigens. Cell 41: 987–997
Beier DC, Cox JH, Vining DR, Cresswell P, Engelhard VH (1994) Association of human class I MHC alleles with the adenovirus E3/19K protein. J Immunol 152: 3862–3872
Anderson M, Paäbo S, Nilsson T, Peterson A (1985) Impaired intracellular transport of class I MHC antigens as a possible means for adenoviruses to evade immune surveillance. Cell 43: 215–222
Feuerbach D, Etteldorf S, Ebenau-Jehle C, Abastado JP, Madden D, Burgert HG (1994) Identification of amino acids within the MHC module important for the interaction with the adenovirus protein E3/19k. J Immunol 153: 1626–1636
Lee MG, Abina MA, Haddada H, Perricaudet M (1995) The constitutive expression of the immunomodulatory gp 19k protein in E1-, E3-adenoviral vectors strongly reduces the host cytotoxic T cell response against the vector. Gene Tber 2: 256–262
Trapnell BC, Gorziglia M (1994) Gene therapy using adenoviral vectors. Curr Opin Biotech 5: 617–625
Engelhardt JF, Ye X, Doranz B, Wilson JM (1994) Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc Natl Acad Sci USA 91: 6196–6200
Yang Y, Nunes FA, Berencsi K, Gonczol E, Englehardt JF, Wilson JM (1994) Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nature Genetics 7: 362–368
Dedieu JF, Vigne E, Torrent C, Jullien C, Mahfouz I, Caillaud JM, Aubailly N, Orsini C, Guillaume JM, Opolon P et al (1997) Long-term gene delivery into the livers of immunocompetent mice with E1/E4-defective adenoviruses. J Virol 71: 4626–4637
Armentano D, Zabner J, Sacks C, Sookdeo CC, Smith MP, St George JA, Wadsworth SC, Smith AE, Gregory RJ, St George JA (1997) Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J Virol 71: 2408–2416
Kochanek S, Clemens PR, Mitani K, Chen HH, Chan S, Caskey CT (1996) A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc Natl Acad Sci USA 93: 5731–5736
Clemens PR, Kochanek S, Sunada Y, Chan S, Chen HH, Campbell KP, Caskey CT (1996) In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther 3: 965–972
Challita PM, Kohn DB (1994) Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc Natl Acad Sci USA 91: 2567–2571
Fang B, Eisensmith RC, Wang H, Kay MA, Cross RE, Landen CN, Gordon G, Bellinger DA, Read MS, Hu PC et al (1995) Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression. Human Gene Ther 6: 1039–1044
Yang Y, Su Q, Grewal IS, Schilz R, Flavell RA, Wilson JM (1996) Transient subversion of CD40 ligand function diminishes immune responses to adenovirus vectors in mouse liver and lung tissues. J Virol 70: 6370–7377
McCoy RD, Davidson BL, Roessler BJ, Huffnagle GB, Simon RH (1995) Expression of human interleukin-1 receptor antagonist in mouse lungs using a recombinant adenovirus: effects on vector-induced inflammation. Gene Therapy 2: 437–442
Shiraishi M, Kusano T, Hara J, Hiroyasu S, Shao-Ping M, Makino Y, Muto Y (1996) Adenovirus-mediated gene transfer using ex vivo perfusion of the heart graft. Surg Today26: 624–628
Ardehali A, Fyfe A, Laks H, Drinkwater DC Jr, Qiao JH, Lusis AJ (1995) Direct gene transfer into donor hearts at the time of harvest. J Thoracic Cardiovas Surg 109: 716–719
Hortelano G, Al-Hendy A, Ofosu FA, Chang PL (1996) Delivery of human factor IX in mice by encapsulated recombinant myoblasts: a novel approach towards allogeneic gene therapy of hemophilia B. Blood 87: 5095–5103
Dwarki VJ, Belloni P, Nijjar T, Smith J, Couto L, Rabier M, Clift S, Berns A, Cohen LK (1995) Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA 92: 1023–1027
Rettinger SD, Kennedy SC, Wu X, Saylors RL, Hafenrichter DG, Flye MW, Ponder KP (1994) Liver-directed gene therapy: Quantitative evaluation of promoter elements by using in vivo retroviral transduction. Proc Natl Acad Sci USA 91: 1460–1464
Yao S, Farjo A, Roessler BJ, Davidson BL, Kurachi K (1996) Adenovirus-mediated transfer of human factor IX gene in immunodeficient and normal mice: evidence for prolonged stability and activity of the transgene in liver. Viral Immunol 9: 141–153
Guidotti LG, Ishikawa T, Hobbs MV, Matzke B, Schreiber R, Chisari FV (1996) Intracellular inactivation of the Hepatitis B virus by cytotoxic T lymphocytes. Immunity 4: 25–36
Tsui LV, Guidotti LG, Ishikawa T, Chisari FV (1995) Posttranscriptional clearance of hapatitis B virus RNA by cytotoxic T lymphocyte-activated hepatocytes. Proc Natl Acad Sci USA 92: 12398–12402
Harms JS, Splitter GA (1995) Interferon-g inhibits transgene expression driven by SV40 or CMV promoters but augments expression driven by the mammalian MHC I promoter. Hum Gene Ther 6: 1291–1297
Qin L, Ding Y, Pahud DR, Chang E, Imperiale MJ, Bromberg JS (1997) Promoter attenuation in gene therapy: IFNγ and TNFα inhibit transgene expression. Hum Gene Ther 8: 1851–1861
Lin H, Parmacek MS, Morle G, Boiling S, Leiden JM (1990) Expression of recombinant genes in myocardium in vivo after direct injection of DNA. Circulation 82: 2217–2221
Wolff JA, Malone RW, Williams P, Chang W, Ascadi G, Jani A, Feigner PL (1990) Direct gene transfer into mouse muscle in vivo. Science 247: 1456–1468
Guzman RJ, Lemarchand P, Crystal RG, Epstein SE, Finkel T (1993) Efficient gene transfer into myocardium by direct injection of adenovirus vectors. Circ Res 73: 1202–1207
Ardehali A, Fyfe A, Laks H, Drinkwater DC Jr, Qiao JH, Lusis AJ (1995) Direct gene transfer into donor hearts at the time of harvest. J Thoracic Cardiovas Surg 109: 716–719
Fyfe AI, Ardehali A, Laks H, Drinkwater DC, Lusis AJ (1995) Biologic modification of the immune response in mouse cardiac isografts using gene transfer. J Heart Lung Transplant 14: S165–S170
Gojo S, Kitamur S, Niwaya K, Yoshida Y, Sakaguchi H, Kawachi K (1996) Ex vivo gene transfer to transplanted heart greats using adenoviral vector. Transplant Proc 28: 1818–1819
Stephan DJ, Yang Z-Y, San H, Simari RD, Wheeler CJ, Feigner PL, Gordon D, Nabel GJ, Nabel EG (1996) A new cationic liposome DNA complex enhances the efficiency of arterial gene transfer in vivo. Hum Gene Ther 7: 1803–1812
Qin L, Chavin KD, Ding Y, Woodward JE, Favaro JP, Lin J, Bromberg JS (1994) Gene transfer for transplantation: Prolongation of allograft survival with transforming growth factor-β1. Annals Surg 220: 508–519
Moore KW, Vieira P, Fiorentino DF, Trounstine ML, Khan TA, Mosmann TR (1990) Homology of cytokine synthesis inhibitory factor (IL-10) to the Epstein-Barr virus gene BCRFI. Science 248: 1230–1234.
Chen WF, Zlotnik A (1991) IL-10: a novel cytotoxic T cell differentiation factor. J Immunol 147: 528–534
Rousset F, Garcia E, Defrance T, Peronne C, Vezzio N, Hsu DH, Kastelein R, Moore KW, Banchereay J (1992) Interleukin 10 is a potent growth and differentiation factor for activated human B lymphocytes. Proc Natl Acad Sci USA 89: 1890–1893
Thompson-Snipes L, Dhar V, Bond MW, Mosmann TR, Moore KW, Rennick DM (1991) Interleukin 10: a novel stimulatory factor for mast cells and their progenitors. J Exp Med 173: 507–510
de Waal Malefyt R, Abrams J, Bennett B, Figdor CG, de Vries JE (1991) Interleukin 10 (IL-10) inhibits cytokine synthesis by human monocytes: an autoregulatory role of IL-10 produced by monocytes. J Exp Med 174: 1209–1220
Mosmann TR (1994) Properties and functions of interleukin-10. Adv Immunol 56: 1–26
Moses HL, Yang EY, Pietenpol JA (1990) TGF-β stimulation and inhibition of cell proliferation: New mechanistic insights. Cell 63: 245–247
Nilsen-Hamilton M (1990) Transforming growth factor-β and its actions on cellular growth and differentiation. Curr Topics Develop Biol 24: 95–136
Mastrangeli A, Danel C, Rosenfeld MA, Stratford-Perricaudet L, Perricaudet M, Pavirani A, Lecocq JP, Crystal RG (1993) Diversity of airway epithelial cell targets for in vivo recombinant adenovirus-mediated gene transfer. J Clin Invest 91: 225–234
Lemarchand P, Jones M, Danel C, Yamada I, Mastrangeli A, Crystal RG (1994) In vivo adenovirus-mediated gene transfer to lungs. J Appl Physiol 76: 2840–2845
Chapelier A, Danel C, Mazmanian M, Bacha EA, Sellak H, Gilbert MA, Herve P, Lemarchand P (1996) Gene therapy in lung transplantation: Feasibility of ex vivo adenovirus-mediated gene transfer to the lung. Hum Gene Ther 7: 1837–1845
Mora BN, Boasquevisque CHR, Boglione M Ritter JH, Scheule RK, Yew NS, Nietupski J, Qin L, DeBruyne L, Bromberg JS et al (1997) Improvement in lung allograft function following transfection with transforming growth factor β1. The Society of Thoracic Surgeons, meeting abstract
Kitamura, M (1994) Transfer of exogenous genes into the kidney. Exp Nephrol 2: 313–317
Bosch RJ, Woolf AS, Fine LG (1993) Gene transfer into the mammalian kidney: direct retrovirus transduction of regenerating tubular epithelial cells. Exp Nephrol 1: 49–54
Moullier P, Friedlander G, Calise D, Ronco P, Perricaudet M, Ferry N (1994) Adenoviral-mediated gene transfer to renal tubular cells in vivo. Kidney International 45: 1220–1225
Kitamura M, Taylor S, Unwin R, Burton S, Shimizu F, Fine LG (1994) Gene transfer into the rat glomerulus via a mesangial cell vector: Site-specific delivery, in situ amplification and sustained expression of an exogenous gene in vivo. J Clin Invest 94: 497–505
Woolf AS, Bosch RJ, Fine LG (1993) Gene transfer into the mammalian kidney: Microtransplantation of retrovirus-transduced metanephric tissue. Exp Nephrol 1: 41–48
Koseki C, Herzlinger D, Al-Aweqati Q (1991) Integration of embryonic nephrogenic cells carrying a reporter gene into functioning nephrons. Am J Physiol 261: C550–C554
Ziegler ST, Kerby JD, Curiel DT, Diethelm AG, Thompson JA (1996) Molecular conjugate-mediated gene transfer into isolated human kidneys. Transplantation 61: 812–817
Docherty K (1997) Gene therapy for diabetes mellitus. Clin Science 92: 321–330
Ferber S, Beltrande I, Rio H, Johnson JH, Noel RJ, Cassidy LE, Clark S, Becker TC, Hughes SD, Newgard CB (1994) GLUT-2 gene transfer into insulinoma cells confers both low and high affinity glucose-stimulated insulin release. J Biol Chem 269: 11523–11529
Deuschle U, Pepperkok R, Wang F, Giordano TJ, McAllister WT, Ansorge W, Bujard H (1989) Regulated expression of foreign genes in mammalian cells under the control of coliphage T3 RNA polymerase and lac repressor. Proc Natl Acad Sci USA 86: 5600–5604
Elfrat S, Fusco-Demane D, Lemberg H, Erman OA, Wang X (1995) Conditional transformation of a pancreatic β-cell line derived from transgenic mice expressing a tetracy-cline-regulated oncogene. Proc Natl Acad Sci USA 92: 3576–3580
Welsh M, Welsh N, Nilsson T, Arkhammar P, Pepinsky RB, Steiner DF, Berggren PO (1988) Stimulation of pancreatic islet beta-cell replication by oncogenes. Proc Natl Acad Sci USA 85: 116–120
Serup P, Jensen J, Andersen FG, Jaergensen MC, Blume N, Hoist JJ, Madsen OD (1996) Induction of insulin and islet amyloid polypeptide production in pancreatic islet glucagonoma cells by insulin promoter factor I. Proc Natl Acad Sci USA 93: 9015–9020
Csete ME, Afra R, Mullen Y, Drazan KE, Benhamou PY, Shaked A (1994) Adenoviral-mediated gene transfer to pancreatic islets does not alter islet function. Transplant Proc 26: 756–757
Mirenda V, Charreau B, Sigalla J, Cassard A, Huvelin JM, David A, Soulilou JP, Le Mauff B, Anegon I (1996) Xenoreactivity in the pig islet to human combination: Feasibility of adenovirus-mediated gene transfer into pig islets. Transplant Proc 28: 808–810
Korbutt GS, Smith DK, Rajotee RV, Elliott JF (1995) Expression of β-galactosidase in mouse pancreatic islets by adenoviral-mediated gene transfer. Transplant Proc 27: 3414
Bosch A, McCray PB Jr, Chang SM, Ulich TR, Simonet WS, Jolly DJ, Davidson BL (1996) Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes. J Clin Invest 98: 2683–2687
Gupta S, Wilson JM, Chowdhury JR (1992) Hepatocyte transplantation: Development of new systems for liver repopulation and gene therapy. Seminars in Liver Disease 12: 321–331
Wolff JA, Yee JK, Skelly HF, Moores JC, Respess JG, Friedmann T, Leffert H (1987) Expression of retrovirally transduced genes in primary cultures of adult rat hepatocytes. Proc Natl Acad Sci USA 84: 3344–3348
Peng H, Armentano D, MacKenzie-Graham L, Shen RF, Darlington G, Ledley FD, Woo SL (1988) Retroviral-mediated gene transfer and expression of human phenylalanine hydroxylase in primary mouse hepatocytes. Proc Natl Acad Sci USA 85: 8146–8150
Hatzoglou M, Lamers W, Bosch F, Wynshaw-Boris A, Clapp DW, Hanson RW (1990) Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase J Biol Chem 265: 17285–17293
Armentano D, Thompson AR, Darlington G, Woo SL (1990) Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: Potential for gene therapy of human hemophilia B. Proc Natl Acad Sci USA 87: 6141–6145
Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Parker Ponder K, Liu TJ, Finegold M, Darlington G, Pokorny W et al (1991) Expression of human α1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 89: 89–93
Wilson JM, Johnston DE, Jefferson DM, Mulligan RC (1988) Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit. Proc Natl Acad Sci USA 85: 4421–4425
Grossman M, Raper SE, Kozarsky K, Stein EA, Engelhardt JF, Muller D, Lupien PJ, Wilson JM (1994) Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolemia. Nature Genetics 6: 335–341
Fabrega AJ, Fasbender AJ, Struble S, Zabner J (1996) Cationic lipid-mediated transfer of the hIL-10 gene prolongs survival of allogeneic hepatocytes in Nagase analbuminemic rats. Transplantation 62: 1866–1871
Shirashi M, Kusano T, Hara J, Hiroyasu S, Shao-ping M, Makino Y, Muto Y (1997) Adenovirus-mediated gene transfer using in-situ perfusion of the liver graft. Transplant International 10: 202–206
Olthoff KM, Da Chen X, Gelman A, Turka L, Shaked A (1997) Adenovirus-mediated gene transfer of CTLA4Ig to liver allografts results in prolonged survival and local T-cell anergy. Transplant Proc 29: 1030–1031
Brenner MK, Heslop HE, Rill D, Li C, Nilson T, Roberts M, Smith C, Krance R, Rooney C (1994) Gene transfer and bone marrow transplantation. Cold Spring Harb Symp Quant Biol 59: 691–697
Rill DR, Moen RC, Buschle M, Bartholomew C, Foreman NK, Mirro J Jr, Krance RA, Ihle JN, Brenner MK (1992) An approach for the analysis of relapse and marrow reconstitution after autologous marrow transplantation using retrovirus-mediated gene transfer. Blood 79: 2694–2700
Cornetta K, Srour EF, Moore A, Davidson A, Broun ER, Hromas R, Moen RC, Morgan RA, Rubin L, Anderson WF et al (1996) Retroviral gene transfer in autologous bone marrow transplantation for adult acute leukemia. Hum Gene Ther 7: 1323–1329
Fletcher FA, Moore KA, Williams DE, Anderson D, Maliszewski C, Belmont JW (1991) Effects of leukemia inhibitory factor (LIF) on gene transfer efficiency into murine hematolymphoid progenitors. Adv Exp Med Biol 292: 131–138
Luskey BD, Rosenblatt M, Zsebo K, Williams DA (1992) Stem cell factor, interleukin-3, and interleukin-6 promote retroviral-mediated gene transfer into murine hematopoietic stem cells. Blood 80: 396–402
Drize N, Chertov J, Sadovnikova E, Tiessen S, Zander A (1997) Long-term maintenance of hematopoiesis in irradiated mice by retrovirally transduced peripheral blood stem cells. Blood 89: 1811–1817
Yurasov S, Kollman TR, Kim A, Raker CA, Hachamovitch M, Wong-Staal F, Goldstein H (1997) Severe combined immunodeficiency mice engrafted with human T cells, B cells, and myeloid cells after transplantation with human fetal bone marrow or liver cells and implanted with human fetal thymus: A model for studying human gene therapy. Blood 89: 1800–1810
Kuhr T, Dougherty GJ, Klingermann H-G (1994) Transfer of the tumor necrosis factor a gene into hematopoietic progenitor cells as a model for site-specific cytokine delivery after marrow transplantation. Blood 84: 2966–2970
Rosenthal FM, Fruh R, Henschler R, Veelken H, Kulmburg P, Mackensen A, Gansbacher B, Mertelsmann R, Lindemannn A (1994) Cytokine therapy with gene-transfected cells: Single injection of irradiated granulocyte-macrophage colony-stimulating factor-transduced cells accelerates hematopoietic recovery after cytotoxic chemotherapy in mice. Blood 84: 2960–2965
Spencer HT, Sleep SE, Rehg JE, Blakley RL, Sorrentino BP (1996) A gene transfer strategy for making bone marrow cells resistant to trimetrexate. Blood 87: 2579–2587
Vinh DB, Mclvor RS (1993) Selective expression of methotrexate-resistant dihydrofolate reductase (DHFR) activity in mice transduced with DHFR retrovirus and administered methotrexate. J Pharmacol Exp Ther 267: 989–996
Bonini C, Ferrari G, Verzeletti S, Servida P, Zappone E, Ruggieri L, Ponzoni M, Rossini S, Mavilio F, Traversari C et al (1997) HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 276: 1719–1724
Fraser CC, Sykes M, Lee RS, Sachs DH, LeGuern C (1995) Specific unresponsiveness to a retrovirally-transferred class I antigen is controlled through the helper pathway. J Immunol 154: 1587–1595
Smith CV, Nakajima K, Mixon A, Guzetta PC, Rosengard BR, Fishbein JM, Sachs DH (1992) Successful induction of long-term specific tolerance to fully allogeneic renal allografts in miniature swine. Transplantation 53: 438–444
Banerjee PT, Ierino F, Kaynor GC, Giovino M, Sablinski T, Emery DW, Rosa MD, LeGuern C, Sachs DH, Monroy RL (1996) Retrovirus-mediated gene transfer and expression of swine MHC Class II genes in CD34+ monkey stem cells. Transplant Proc 28: 747–748
Salminen A, Elson HF, Mickley LA, Fojo AT, Gottesman MM (1991) Implantation of recombinant rat myocytes into adult skeletal muscle: a potential gene therapy. Hum Gene Ther 2: 15–26
Rando TA, Blau HM (1994) Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J Cell Biol 125: 1275–1287
Yao SN, Kurachi K (1993) Implanted myoblasts not only fuse with myofibers but also survive as muscle precursor cells. J Cell Sci 105: 957–963
Pagel CN, Morgan JE (1995) Myoblast transfer and gene therapy in muscular dystrophies. Microscopy Research and Technique 30: 469–479
Morgan, JE (1994) Cell and gene therapy in Duchenne muscular dystrophy. Hum Gene Ther 5: 165–173
Law PK, Goodwin TG, Fang Q, Hall TL, Quinley T, Vastagh G, Duggirala V, Larkin C, Florendo JA, Li L et al (1997) First human myoblast transfer therapy continues to show dystrophin after 6 years. Cell Transplant 6: 95–100
Dai Y, Roman M, Naviaux RK, Verma IM (1992) Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo. Proc Natl Acad Sci USA 89: 10892–10895
Yao SN, Smith KJ, Kurachi K (1994) Primary myoblast-mediated gene transfer: persistent expression of human factor IX in mice. Gene Ther 1: 99–107
Wang JM, Zheng H, Blaivas M, Kurachi K (1997) Persistent systemic production of human factor IX in mice by skeletal myoblast-mediated gene transfer: feasibility of repeat application to obtain therapeutic levels. Blood 90: 1075–1082
Baru M, Sha’anani J, Nur I (1995) Retroviral-mediated in vivo gene transfer into muscle cells and synthesis of human factor IX in mice. Intervirology 38: 356–360
Dhawan J, Pan LC, Pavlath GK, Travis MA, Lanctot AM, Blau HM (1991) Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254: 1509–1512
Dahler A, Wade RP, Muscat GE, Waters MJ (1994) Expression vectors encoding human growth hormone (hGH) controlled by human muscle-specific promoters: prospects for regulated production of hGH delivered by myoblast transfer or intravenous injection. Gene 145: 305–310
Al-Hendy A, Hortelano G, Tannenbaum GS, Chang PL (1995) Correction of the growth defect in dwarf mice with nonautologous microencapsulated myoblasts—an alternate approach to somatic gene therapy. Hum Gene Ther 6: 165–175
Hamamori Y, Samal B, Tian J, Kedes L (1994) Persistent erythropoiesis by myoblast transfer of erythropoietin cDNA. Hum Gene Ther 5: 1349–1356
Hamamori Y, Samal B, Tian J, Kedes L (1995) Myoblast transfer of human erythropoietin gene in a mouse model of renal failure. J Clin Invest 95: 1808–1813
Simonson GD, Groskreutz DJ, Gorman CM, MacDonald MJ (1996) Synthesis and processing of genetically modified human proinsulin by rat myoblast primary cultures. Hum Gene Ther 7: 71–78
Shull RM, Lu X, McEntee MF, Bright RM, Pepper KA, Kohn DB (1996) Myoblast gene therapy in canine mucopolysaccharidosis. I: Abrogation by an immune response to alpha-L-iduronidase. Hum Gene Ther 7: 1595–1603
Naffakh N, Bohl D, Salvetti A, Moullier P, Danos O, Heard JM (1994) Gene therapy for lysosomal disorders. Nouv Rev Fr Hematol 36(1): S11–S16
Jiao S, Gurevich V, Wolff JA (1993) Long-term correction of rat model of Parkinson’s disease by gene therapy. Nature 362: 450–453
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Bromberg, J.S., DeBruyne, L.A., Sung, R.S., Qin, L. (2000). Gene transfer to facilitate transplantation. In: Evans, C.H., Robbins, P.D. (eds) Gene Therapy in Inflammatory Diseases. Progress in Inflammation Research. Birkhäuser, Basel. https://doi.org/10.1007/978-3-0348-8478-5_10
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