Prospects for designing 'universal' stem cell lines

The Immunological Barriers to Regenerative Medicine

Volumn , Issue , 2013, Pages 147-173

  Cicciarelli, James C ^a,b   Lemp, Nathan A ^a   Kasahara, Noriyuki ^c  

^a National Institute of Transplantation Los Angeles   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84905717841     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1007/978-1-4614-5480-9_9     Document Type: Chapter

References (109)

1. Marsh SG, Albert ED, Bodmer WF et al (2010) Nomenclature for factors of the HLA system. Tissue Antigens 75:291-455
2. Petersdorf EW, Malkki M (2005) Human leukocyte antigen matching in unrelated donor hematopoietic cell transplantation. Semin Hematol 42:76-84
3. Petersdorf EW, Hansen JA, Martin PJ et al (2001) Major-histocompatibility-complex class I alleles and antigens in hematopoietic-cell transplantation. N Engl J Med 345:1794-1800
4. Tiercy JM, Villard J, Roosnek E (2002) Selection of unrelated bone marrow donors by serology, molecular typing and cellular assays. Transpl Immunol 10:215-221
5. Fleischhauer K, Kernan NA, O'Reilly RJ et al (1990) Bone marrow-allograft rejection by T lymphocytes recognizing a single amino acid difference in HLA-B44. N Engl J Med 323:1818-1822
6. Petersdorf EW, Anasetti C, Martin PJ et al (2004) Limits of HLA mismatching in unrelated hematopoietic cell transplantation. Blood 104:2976-2980
7. Sasazuki T, Juji T, Morishima Y et al (1998) Effect of matching of class I HLA alleles on clinical outcome after transplantation of hematopoietic stem cells from an unrelated donor. Japan marrow donor program. N Engl J Med 339:1177-1185
8. Cicciarelli J (2004) HLA typing immunogenetics and transplantation. Curr Opin Organ Transpl 9:1-8
9. Rubinstein P (2001) HLA matching for bone marrow transplantation-how much is enough? N Engl J Med 345:1842-1844
10. Vargas-Diez E, Fernandez-Herrera J, Marin A et al (2003) Analysis of risk factors for acute cutaneous graft-versus-host disease after allogeneic stem cell transplantation. Br J Dermatol 148:1129-1134
11. Meier-Kriesche HU, Ojo AO, Leichtman AB et al (2001) Interaction of mycophenolate mofetil and HLA matching on renal allograft survival. Transplantation 71:398-401
12. Meier-Kriesche HU, Scornik JC, Susskind B et al (2009) A lifetime versus a graft life approach redefines the importance of HLA matching in kidney transplant patients. Transplantation 88:23-29
13. Cicciarelli J, Aswad S, Mendez R (2005) Significant HLA matching effect in a large urban transplant center composed primarily of minorities. Transplant Proc 37:658-660
14. Li L, Baroja ML, Majumdar A et al (2004) Human embryonic stem cells possess immune- privileged properties. Stem Cells 22:448-456
15. Drukker M, Katz G, Urbach A et al (2002) Characterization of the expression of MHC proteins in human embryonic stem cells. Proc Natl Acad Sci USA 99:9864-9869
16. Drukker M, Katchman H, Katz G et al (2006) Human embryonic stem cells and their differentiated derivatives are less susceptible to immune rejection than adult cells. Stem Cells 24:221-229
17. Tian X, Woll PS, Morris JK et al (2006) Hematopoietic engraftment of human embryonic stem cell-derived cells is regulated by recipient innate immunity. Stem Cells 24:1370-1380
18. Fairchild PJ, Cartland S, Nolan KF et al (2004) Embryonic stem cells and the challenge of transplantation tolerance. Trends Immunol 25:465-470
19. Boyd AS, Higashi Y, Wood KJ (2005) Transplanting stem cells: potential targets for immune attack. Modulating the immune response against embryonic stem cell transplantation. Adv Drug Deliv Rev 57:1944-1969
20. Priddle H, Jones DR, Burridge PW et al (2006) Hematopoiesis from human embryonic stem cells: overcoming the immune barrier in stem cell therapies. Stem Cells 24:815-824
21. Ohnuki M, Takahashi K, Yamanaka S (2009) Generation and characterization of human induced pluripotent stem cells. Curr Protoc Stem Cell Biol 9:4A.2.1-4A.2.25
22. Okita K, Ichisaka T, Yamanaka S (2007) Generation of germline-competent induced pluripotent stem cells. Nature 448:313-317
23. Yu J, Vodyanik MA, Smuga-Otto K et al (2007) Induced pluripotent stem cell lines derived from human somatic cells. Science 318:1917-1920
24. Lyssiotis CA, Foreman RK, Staerk J et al (2009) Reprogramming of murine fibroblasts to induced pluripotent stem cells with chemical complementation of Klf4. Proc Natl Acad Sci USA 106:8912-8917
25. Drukker M, Benvenisty N (2004) The immunogenicity of human embryonic stem-derived cells. Trends Biotechnol 22:136-141
26. Bradley JA, Bolton EM, Pedersen RA (2002) Stem cell medicine encounters the immune system. Nat Rev Immunol 2:859-871
27. Pollak R, Blanchard JM (2000) Organ donor or graft pretreatment to prolong allograft survival: lessons learned in the murine model. Transplantation 69:2432-2439
28. Freland S, Chambers BJ, Andersson M et al (1998) Rejection of allogeneic and syngeneic but not MHC class I-deficient tumor grafts by MHC class I-deficient mice. J Immunol 160:572-579
29. Mhashilkar AM, Doebis C, Seifert M et al (2002) Intrabody-mediated phenotypic knockout of major histocompatibility complex class I expression in human and monkey cell lines and in primary human keratinocytes. Gene Ther 9:307-319
30. Beyer F, Doebis C, Busch A et al (2004) Decline of surface MHC I by adenoviral gene transfer of anti-MHC I intrabodies in human endothelial cells-new perspectives for the generation of universal donor cells for tissue transplantation. J Gene Med 6:616-623
31. Hannon GJ, Rossi JJ (2004) Unlocking the potential of the human genome with RNA interference. Nature 431:371-378
32. Hammond SM, Caudy AA, Hannon GJ (2001) Post-transcriptional gene silencing by double-stranded RNA. Nat Rev Genet 2:110-119
33. McManus MT, Petersen CP, Haines BB et al (2002) Gene silencing using micro-RNA designed hairpins. RNA 8:842-850
34. Castanotto D, Rossi JJ (2009) The promises and pitfalls of RNA-interference-based therapeutics. Nature 457:426-433
35. Hawkins PG, Santoso S, Adams C et al (2009) Promoter targeted small RNAs induce longterm transcriptional gene silencing in human cells. Nucleic Acids Res 37:2984-2995
36. Morris KV (2006) Therapeutic potential of siRNA-mediated transcriptional gene silencing. Biotechniques Suppl:7-13
37. Morris KV, Chan SW, Jacobsen SE et al (2004) Small interfering RNA-induced transcriptional gene silencing in human cells. Science 305:1289-1292
38. Gonzalez S, Castanotto D, Li H et al (2005) Amplification of RNAi-targeting HLA mRNAs. Mol Ther 11:811-818
39. Dull T, Zufferey R, Kelly M et al (1998) A third-generation lentivirus vector with a conditional packaging system. J Virol 72:8463-8471
40. Zufferey R, Dull T, Mandel RJ et al (1998) Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 72:9873-9880
41. Naldini L, Blomer U, Gallay P et al (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263-267
42. Blomer U, Naldini L, Kafri T et al (1997) Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J Virol 71:6641-6649
43. Kafri T, Blomer U, Peterson DA et al (1997) Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 17:314-317
44. Sakoda T, Kasahara N, Hamamori Y et al (1999) A high-titer lentiviral production system mediates efficient transduction of differentiated cells including beating cardiac myocytes. J Mol Cell Cardiol 31:2037-2047
45. Shichinohe T, Bochner BH, Mizutani K et al (2001) Development of lentiviral vectors for antiangiogenic gene delivery. Cancer Gene Ther 8:879-889
46. Borok Z, Harboe-Schmidt JE, Brody SL et al (2001) Vesicular stomatitis virus G-pseudotyped lentivirus vectors mediate efficient apical transduction of polarized quiescent primary alveolar epithelial cells. J Virol 75:11747-11754
47. Li W, Nadelman C, Gratch NS et al (2002) An important role for protein kinase C-delta in human keratinocyte migration on dermal collagen. Exp Cell Res 273:219-228
48. Chen M, Kasahara N, Keene DR et al (2002) Restoration of type VII collagen expression and function in dystrophic epidermolysis bullosa. Nat Genet 32:670-675
49. Koya RC, Kasahara N, Favaro PM et al (2003) Potent maturation of monocyte-derived dendritic cells after CD40L lentiviral gene delivery. J Immunother 26:451-460
50. Haga K, Lemp NA, Logg CR et al (2006) Permanent, lowered HLA class I expression using lentivirus vectors with shRNA constructs: averting cytotoxicity by alloreactive T lymphocytes. Transplant Proc 38:3184-3188
51. Hacke K, Falahati R, Flebbe-Rehwaldt L et al (2009) Suppression of HLA expression by lentivirus-mediated gene transfer of siRNA cassettes and in vivo chemoselection to enhance hematopoietic stem cell transplantation. Immunol Res 44:112-126
52. Benichou G, Yamada Y, Yun SH et al (2011) Immune recognition and rejection of allogeneic skin grafts. Immunotherapy 3:757-770
53. Figueiredo C, Seltsam A, Blasczyk R (2006) Class-, gene-, and group-specific HLA silencing by lentiviral shRNA delivery. J Mol Med 84:425-437
54. Figueiredo C, Horn PA, Blasczyk R et al (2007) Regulating MHC expression for cellular therapeutics. Transfusion 47:18-27
55. Figueiredo C, Goudeva L, Horn PA et al (2010) Generation of HLA-deficient platelets from hematopoietic progenitor cells. Transfusion 50:1690-1701
56. Ntokou IS, Iniotaki AG, Kontou EN et al (2011) Long-term follow up for anti-HLA donor specific antibodies postrenal transplantation: high immunogenicity of HLA class II graft molecules. Transpl Int 24:1084-1093
57. Smith JD, Banner NR, Hamour IM et al (2011) De novo donor HLA-specific antibodies after heart transplantation are an independent predictor of poor patient survival. Am J Transplant 11:312-319
58. Jaimes Y, Seltsam A, Eiz-Vesper B et al (2011) Regulation of HLA class II expression prevents allogeneic T-cell responses. Tissue Antigens 77:36-44
59. Elssner A, Jaumann F, Wolf WP et al (2002) Bronchial epithelial cell B7-1 and B7-2 mRNA expression after lung transplantation: a role in allograft rejection? Eur Respir J 20:165-169
60. Odobasic D, Kitching AR, Semple TJ et al (2005) Glomerular expression of CD80 and CD86 is required for leukocyte accumulation and injury in crescentic glomerulonephritis. J Am Soc Nephrol 16:2012-2022
61. Salomon B, Lenschow DJ, Rhee L et al (2000) B7/CD28 costimulation is essential for the homeostasis of the CD4+ CD25+ immunoregulatory T cells that control autoimmune diabetes. Immunity 12:431-440
62. Hunt JS, Langat DL (2009) HLA-G: a human pregnancy-related immunomodulator. Curr Opin Pharmacol 9:462-469
63. Urosevic M, Dummer R (2008) Human leukocyte antigen-G and cancer immunoediting. Cancer Res 68:627-630
64. Sheshgiri R, Rouas-Freiss N, Rao V et al (2008) Myocardial HLA-G reliably indicates a low risk of acute cellular rejection in heart transplant recipients. J Heart Lung Transplant 27:522-527
65. Crispim JC, Duarte RA, Soares CP et al (2008) Human leukocyte antigen-G expression after kidney transplantation is associated with a reduced incidence of rejection. Transpl Immunol 18:361-367
66. Brugiere O, Thabut G, Pretolani M et al (2009) Immunohistochemical study of HLA-G expression in lung transplant recipients. Am J Transplant 9:1427-1438
67. Zarkhin V, Talisetti A, Li L et al (2010) Expression of soluble HLA-G identifies favorable outcomes in liver transplant recipients. Transplantation 90:1000-1005
68. Sasaki H, Xu XC, Smith DM et al (1999) HLA-G expression protects porcine endothelial cells against natural killer cell-mediated xenogeneic cytotoxicity. Transplantation 67:31-37
69. Zeng MH, Fang CY, Wang SS et al (2006) A study of soluble HLA-G1 protecting porcine endothelial cells against human natural killer cell-mediated cytotoxicity. Transplant Proc 38:3312-3314
70. del Rio ML, Buhler L, Gibbons C et al (2008) PD-1/PD-L1, PD-1/PD-L2, and other coinhibitory signaling pathways in transplantation. Transpl Int 21:1015-1028
71. Plege A, Borns K, Baars W et al (2009) Suppression of human T-cell activation and expansion of regulatory T cells by pig cells overexpressing PD-ligands. Transplantation 87:975-982
72. Najafian N, Sayegh MH (2000) CTLA4-Ig: a novel immunosuppressive agent. Expert Opin Investig Drugs 9:2147-2157
73. Yamashita K, Masunaga T, Yanagida N et al (2003) Long-term acceptance of rat cardiac allografts on the basis of adenovirus mediated CD40Ig plus CTLA4Ig gene therapies. Transplantation 76:1089-1096
74. Tomasoni S, Longaretti L, Azzollini N et al (2004) Favorable effect of cotransfection with TGF-beta and CTLA4Ig of the donor kidney on allograft survival. Am J Nephrol 24:275-283
75. Zhou X, Schmidtke P, Zepp F et al (2005) Boosting interleukin-10 production: therapeutic effects and mechanisms. Curr Drug Targets Immune Endocr Metabol Disord 5:465-475
76. Cypel M, Liu M, Rubacha M et al (2009) Functional repair of human donor lungs by IL-10 gene therapy. Sci Transl Med 1:4ra9
77. Oishi H, Okada Y, Kikuchi T et al (2010) Transbronchial human interleukin-10 gene transfer reduces acute inflammation associated with allograft rejection and intragraft interleukin-2 and tumor necrosis factor-alpha gene expression in a rat model of lung transplantation. J Heart Lung Transplant 29:360-367
78. Wonderlich ER, Leonard JA, Collins KL (2011) HIV immune evasion disruption of antigen presentation by the HIV Nef protein. Adv Virus Res 80:103-127
79. Johnson JM, Nicot C, Fullen J et al (2001) Free major histocompatibility complex class I heavy chain is preferentially targeted for degradation by human T-cell leukemia/ lymphotropic virus type 1 p12(I) protein. J Virol 75:6086-6094
80. Windheim M, Hilgendorf A, Burgert HG (2004) Immune evasion by adenovirus E3 proteins: exploitation of intracellular trafficking pathways. Curr Top Microbiol Immunol 273:29-85
81. Liu H, Fu J, Bouvier M (2007) Allele- and locus-specific recognition of class I MHC molecules by the immunomodulatory E3-19 K protein from adenovirus. J Immunol 178:4567-4575
82. Fu J, Li L, Bouvier M (2011) Adenovirus E3-19 K proteins of different serotypes and subgroups have similar, yet distinct, immunomodulatory functions toward major histocompatibility class I molecules. J Biol Chem 286:17631-17639
83. Toth K, Doronin K, Kuppuswamy M et al (2005) Adenovirus immunoregulatory E3 proteins prolong transplants of human cells in immunocompetent mice. Virus Res 108:149-159
84. Kojaoghlanian T, Joseph A, Follenzi A et al (2009) Lentivectors encoding immunosuppressive proteins genetically engineer pancreatic beta-cells to correct diabetes in allogeneic mice. Gene Ther 16:340-348
85. Hewitt EW (2003) The MHC class I antigen presentation pathway: strategies for viral immune evasion. Immunology 110:163-169
86. Hansen TH, Bouvier M (2009) MHC class I antigen presentation: learning from viral evasion strategies. Nat Rev Immunol 9:503-513
87. Mocarski ES Jr (2004) Immune escape and exploitation strategies of cytomegaloviruses: impact on and imitation of the major histocompatibility system. Cell Microbiol 6:707-717
88. Basta S, Bennink JR (2003) A survival game of hide and seek: cytomegaloviruses and MHC class I antigen presentation pathways. Viral Immunol 16:231-242
89. Lehner PJ, Hoer S, Dodd R et al (2005) Downregulation of cell surface receptors by the K3 family of viral and cellular ubiquitin E3 ligases. Immunol Rev 207:112-125
90. Zuo J, Quinn LL, Tamblyn J et al (2011) The Epstein-Barr virus-encoded BILF1 protein modulates immune recognition of endogenously processed antigen by targeting major histocompatibility complex class I molecules trafficking on both the exocytic and endocytic pathways. J Virol 85:1604-1614
91. Fruh K, Bartee E, Gouveia K et al (2002) Immune evasion by a novel family of viral PHD/ LAP-finger proteins of gamma-2 herpesviruses and poxviruses. Virus Res 88:55-69
92. Bahar MW, Graham SC, Chen RA et al (2011) How vaccinia virus has evolved to subvert the host immune response. J Struct Biol 175:127-134
93. Spencer JV, Lockridge KM, Barry PA et al (2002) Potent immunosuppressive activities of cytomegalovirus-encoded interleukin-10. J Virol 76:1285-1292
94. Lafon M, Prehaud C, Megret F et al (2005) Modulation of HLA-G expression in human neural cells after neurotropic viral infections. J Virol 79:15226-15237
95. Wu J, Bonsra AN, Du G (2009) pSM155 and pSM30 vectors for miRNA and shRNA expression. Methods Mol Biol 487:205-219
96. Baltimore D, Boldin MP, O'Connell RM et al (2008) MicroRNAs: new regulators of immune cell development and function. Nat Immunol 9:839-845
97. Shivdasani RA (2006) MicroRNAs: regulators of gene expression and cell differentiation. Blood 108:3646-3653
98. Aagaard LA, Zhang J, von Eije KJ et al (2008) Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs. Gene Ther 15:1536-1549
99. Stern-Ginossar N, Elefant N, Zimmermann A et al (2007) Host immune system gene targeting by a viral miRNA. Science 317:376-381
100. Swaminathan S (2008) Noncoding RNAs produced by oncogenic human herpesviruses. J Cell Physiol 216:321-326
101. Boss IW, Renne R (2010) Viral miRNAs: tools for immune evasion. Curr Opin Microbiol 13:540-545
102. Santiago Y, Chan E, Liu PQ et al (2008) Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases. Proc Natl Acad Sci USA 105:5809-5814
103. Urnov FD, Miller JC, Lee YL et al (2005) Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435:646-651
104. Morbitzer R, Romer P, Boch J et al (2010) Regulation of selected genome loci using de novo-engineered transcription activator-like effector (TALE)-type transcription factors. Proc Natl Acad Sci USA 107:21617-21622
105. Christian M, Cermak T, Doyle EL et al (2010) Targeting DNA double-strand breaks with TAL effector nucleases. Genetics 186:757-761
106. Miller JC, Tan S, Qiao G et al (2011) A TALE nuclease architecture for efficient genome editing. Nat Biotechnol 29:143-148
107. Szczepek M, Brondani V, Buchel J et al (2007) Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nat Biotechnol 25:786-793
108. Doyon Y, Vo TD, Mendel MC et al (2011) Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods 8:74-79
109. Gabriel R, Lombardo A, Arens A et al (2011) An unbiased genome-wide analysis of zinc- finger nuclease specificity. Nat Biotechnol 29:816-823