Review: Stem cells and gene therapy

Laboratory Hematology

Volumn 16, Issue 3, 2010, Pages 53-73

  Alenzi, Faris Q ^a   Lotfy, Mahmoud ^b   Tamimi, Waleed G ^c   Wyse, Richard K H ^d  

^a PRINCE SATTAM BIN ABDULAZIZ UNIVERSITY   (Saudi Arabia)

^b MENOUFIA UNIVERSITY   (Egypt)

^c KING SAUD BIN ABDULAZIZ UNIVERSITY FOR HEALTH SCIENCES   (Saudi Arabia)

^d Imperial College of Medicine   (United Kingdom)

Author keywords

Gene therapy; Stem cells; Transfection; Viral vectors

Indexed keywords

ADENOVIRUS VECTOR; ANTISENSE OLIGODEOXYNUCLEOTIDE; ASIALOGLYCOPROTEIN RECEPTOR; BASIC FIBROBLAST GROWTH FACTOR; BETA GALACTOSIDASE; DYSTROPHIN; GAG PROTEIN; GLUCURONOSYLTRANSFERASE; GRANULOCYTE COLONY STIMULATING FACTOR; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; GREEN FLUORESCENT PROTEIN; HUMAN GROWTH HORMONE; IMMUNOGLOBULIN; INTERLEUKIN 10; PARVOVIRUS VECTOR; POL PROTEIN; RECOMBINANT INTERLEUKIN 12; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE 2; SCATTER FACTOR; STEROID; TRANSFERRIN RECEPTOR; TRANSMEMBRANE CONDUCTANCE REGULATOR; VASCULOTROPIN; WISKOTT ALDRICH SYNDROME PROTEIN;

ADENO ASSOCIATED VIRUS; ADENOSINE DEAMINASE DEFICIENCY; ADENOVIRUS 2; ADENOVIRUS 5; ARTERIOSCLEROSIS; BETA THALASSEMIA; BONE MARROW TRANSPLANTATION; BRONCHIECTASIS; BURST FORMING UNIT E; CANCER GENE THERAPY; CELL DIFFERENTIATION; CELL PROLIFERATION; CHRONIC GRANULOMATOUS DISEASE; CHRONIC HEPATITIS; CHRONIC SINUSITIS; CLINICAL TRIAL; COLONY FORMATION; COMPETITIVE INHIBITION; CORNEA TRANSPLANTATION; CORNEAL GRAFT REJECTION; CRIGLER NAJJAR SYNDROME; CYTOMEGALOVIRUS; CYTOTOXIC T LYMPHOCYTE; DNA MODIFICATION; DRUG EFFICACY; DRUG SAFETY; EMBRYONIC STEM CELL; EX VIVO GENE TRANSFER; GENE CONTROL; GENE EXPRESSION; GENE IDENTIFICATION; GENE THERAPY; GENE VECTOR; GERM CELL; GRAFT REJECTION; GRAFT VERSUS HOST REACTION; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHY; HERPES SIMPLEX VIRUS; HUMAN; IMMUNE RESPONSE; IMMUNOGENICITY; IMMUNOSUPPRESSIVE TREATMENT; IN VITRO GENE TRANSFER; LIPOSOMAL GENE DELIVERY SYSTEM; LIVER CANCER; LIVER CIRRHOSIS; MESENCHYMAL STEM CELL; MOLONEY LEUKEMIA ONCOVIRUS; MYELOID PROGENITOR CELL; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PLURIPOTENT HEMATOPOIETIC STEM CELL THERAPY; PLURIPOTENT STEM CELL; POXVIRUS; PROTEIN PHOSPHORYLATION; RESTENOSIS; RETROVIRUS; REVIEW; SEVERE COMBINED IMMUNODEFICIENCY; SOMATIC CELL GENETICS; SOMATIC GENE THERAPY; STEM CELL; TISSUE ENGINEERING; VESICULAR STOMATITIS VIRUS; VIRAL GENE DELIVERY SYSTEM; VIRUS GENOME; VIRUS HEPATITIS; VIRUS REPLICATION; WISKOTT ALDRICH SYNDROME;

ADULT STEM CELLS; EMBRYONIC STEM CELLS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; STEM CELL TRANSPLANTATION;

EID: 78649259768     PISSN: 10802924     EISSN: None     Source Type: Journal    
DOI: 10.1532/LH96.10010     Document Type: Review

References (165)

1. Reya T, Morrison SJ, Clarke MF, Weissman IL. Stem cells, cancer, and cancer stem cells. Nature. 2001;414:105-111.
2. Bongso A, Richards M. History and perspective of stem cell research. Best Pract Res Clin Obstet Gynaecol. 2004;18:827-842.
3. Bongso A, Fong C-Y. Human embryonic stem cells: their nature, properties, and uses. In: Baharvand H, ed. Trends in Stem Cell Biology and Technology. New York, NY: Humana Press; 2009:1-18.
4. Yoshimizu T, Sugiyama N, De Felice M, et al. Germline-specific expression of the Oct-4/green fluorescent protein (GFP) transgene in mice. Dev Growth Differ. 1999;41:675-684.
5. Pesce M, Wang X, Wolgemuth DJ, Schöler H. Differential expression of the Oct-4 transcription factor during mouse germ cell differentiation. Mech Dev. 1998;71:89-98.
6. Bongso A, Tan S. Human blastocyst culture and derivation of embryonic stem cell lines. Stem Cell Rev. 2005;1:87-98.
7. Fong CY, Sathananthan AH, Wong PC, Bongso A. Nine-day-old human embryo cultured in vitro: a clue to the origins of embryonic stem cells. Reprod Biomed Online. 2004;9:321-325.
8. Bjornson CR, Rietze RL, Reynolds BA, Magli MC, Vescovi AL. Turning brain into blood: a hematopoietic fate adopted by adult neural stem cells in vivo. Science. 1999;283:534-537.
9. Jackson KA, Mi T, Goodell MA. Hematopoietic potential of stem cells isolated from murine skeletal muscle. Proc Natl Acad Sci U S A. 1999;96:14482-14486.
10. Clarke DL, Johansson CB, Wilbertz J, et al. Generalized potential of adult neural stem cells. Science. 2000;288:1660-1663.
11. Krause DS, Theise ND, Collector MI, et al. Multi-organ, multilineage engraftment by a single bone marrow-derived stem cell. Cell. 2001;105:369-377.
12. McGuckin CP, Forraz N, Baradez MO, et al. Production of stem cells with embryonic characteristics from human umbilical cord blood. Cell Prolif. 2005;38:245-255.
13. Fong CY, Richards M, Manasi N, Biswas A, Bongso A. Comparative growth behavior and characterization of stem cells from human Wharton's jelly. Reprod Biomed Online. 2007;15:708-718.
14. Sarugaser R, Lickorish D, Baksh D, Hosseini MM, Davies JE. Human umbilical cord perivascular (HUCPV) cells: a source of mesenchymal progenitors. Stem Cells. 2005;23:220-229.
15. Alenzi FQ, Alenazi BQ, Ahmad SY, Salem ML, Al-Jabri AA, Wyse RK. The haemopoietic stem cell: between apoptosis and self renewal. Yale J Biol Med. 2009;82:7-18.
16. Ploemacher RE. Stem cells: characterization and measurement. Baillieres Clin Haematol. 1997;10:429-444.
17. Gordon MY. Human haemopoietic stem cell assays. Blood Rev. 1993;7:190-197.
18. Al-Hajj M, Becker MW, Wicha M, Weissman I, Clarke MF. Therapeutic implications of cancer stem cells. Curr Opin Genet Dev. 2004;14:43-47.
19. Crowe DL, Parsa B, Sinha UK. Relationships between stem cells and cancer stem cells. Histol Histopathol. 2004;19:505-509.
20. Sikora MA, Olszewski WL. Stem cells - biology and therapeutic application [in Polish]. Postepy Hig Med Dosw (Online). 2004;58:202-208.
21. Mayhall EA, Paffett-Lugassy N, Zon LI. The clinical potential of stem cells. Curr Opin Cell Biol. 2004;16:713-720.
22. Al-Hajj M, Clarke MF. Self-renewal and solid tumor stem cells. Oncogene. 2004;23:7274-7282.
23. Dontu G, Al-Hajj M, Abdallah WM, Clarke MF, Wicha MS. Stem cells in normal breast development and breast cancer. Cell Prolif. 2003;36:59-72. (Pubitemid 37521844)
24. Galderisi U, Cipollaro M, Giordano A. Stem cells and brain cancer. Cell Death Differ. 2006;13:5-11.
25. Gordon MY, Blackett NM. Routes to repopulation - a unification of the stochastic model and separation of stem-cell subpopulations. Leukemia. 1994;8:1068-1072.
26. Novak JP, Stewart CC. Stochastic versus deterministic in haemopoiesis: what is what? Br J Haematol. 1991;78:149-154.
27. Deguchi T, Komada Y, Sugiyama K, et al. Expression of homing- associated cell adhesion molecule (H-CAM/CD44) on human CD34+ hematopoietic progenitor cells. Exp Hematol. 1999;27:542-552.
28. Gordon MY, Marley SB, Lewis JL, et al. Treatment with interferon- alpha preferentially reduces the capacity for amplification of granulocyte-macrophage progenitors (CFU-GM) from patients with chronic myeloid leukemia but spares normal CFU-GM. J Clin Invest. 1998;102:710-715.
29. Metcalf D. Control of granulocytes and macrophages: molecular, cellular, and clinical aspects. Science. 1991;254:529-533.
30. Eaves AC, Eaves CJ. Erythropoiesis in culture. Clin Haematol. 1984;13:371-391.
31. Hoffman R. Regulation of megakaryocytopoiesis. Blood. 1989;74:1196-1212.
32. Gordon MY, Amos TA. Stochastic effects in hemopoiesis. Stem Cells. 1994;12:175-179.
33. Till JE, McCulloch EA. A direct measurement of the radiation sensitivity of normal mouse bone marrow cells. Radiat Res. 1961;14:213-222.
34. Humphries RK, Eaves AC, Eaves CJ. Self-renewal of hemopoietic stem cells during mixed colony formation in vitro. Proc Natl Acad Sci U S A. 1981;78:3629-3633.
35. Nakahata T, Ogawa M. Identification in culture of a class of hemopoietic colony-forming units with extensive capability to self-renew and generate multipotential hemopoietic colonies. Proc Natl Acad Sci U S A. 1982;79:3843-3847.
36. Abkowitz JL, Catlin SN, Guttorp P. Evidence that hematopoiesis may be a stochastic process in vivo. Nat Med. 1996;2:190-197.
37. Nicola NA, Johnson GR. The production of committed hemopoietic colony-forming cells from multipotential precursor cells in vitro. Blood. 1982;60:1019-1029.
38. Morrison SJ, Weissman IL. The long-term repopulating subset of hematopoietic stem cells is deterministic and isolatable by phenotype. Immunity. 1994;1:661-673.
39. Metcalf D. Lineage commitment and maturation in hematopoietic cells: the case for extrinsic regulation. Blood. 1998;92:345-347.
40. Enver T, Heyworth CM, Dexter TM. Do stem cells play dice? Blood. 1998;92:348-351.
41. Siminovitch L, McCulloch A, Till JE. The identification of colony-forming cells among spleen colonies. J Cell Physiol. 1963;62:327-336.
42. Metcalf D. Regulator-induced suppression of myelomonocytic leukemic cells: clonal analysis of early cellular events. Int J Cancer. 1982;30:203-210.
43. Lewis JL, Marley SB, Blackett NM, Szydlo R, Goldman JM, Gordon MY. Interleukin 3 (IL-3), but not stem cell factor (SCF) increases self-renewal by human erythroid burst-forming units (BFU-E) in vitro. Cytokine. 1998;10:49-54.
44. Lewis JL, Marley SB, Davidson RJ, et al. Protein tyrosine kinase receptor signalling decreases progenitor cell self replication. Br J Haematol. 1998;102(suppl 1):23.
45. Lewis JL, Blackett NM, Gordon MY. The kinetics of colony formation by CFU-GM in vitro. Br J Haematol. 1994;88:440-442.
46. Han ZC, Caen JP. Cytokines acting on committed haematopoietic progenitors. Baillieres Clin Haematol. 1994:7:65-89.
47. Quesenberry PJ. The blueness of stem cells. Exp Hematol. 1991;19:725-728.
48. Lowry PA. Botanical musings. Exp Hematol. 1992;20:540.
49. Eglitis MA, Mezey E. Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice. Proc Natl Acad Sci U S A. 1997;94:4080-4085.
50. Ferrari G, Cusella-De Angelis G, Coletta M, et al. Muscle regeneration by bone marrow-derived myogenic progenitors. Science. 1998;279:1528-1530.
51. Till JE, McCulloch EA, Siminovitch L. A stochastic model of stem cell proliferation, based on the growth of spleen colony-forming cells. Proc Natl Acad Sci U S A. 1964;51:29-36.
52. Suda J, Suda T, Ogawa M. Analysis of differentiation of mouse hemopoietic stem cells in cultures by sequential replating of paired progenitors. Blood. 1984;64:393-399.
53. Janković GM, Colović MO, Petrović MO, Rolović ZN, Janosević S. The stochastic blueness of leukemic stem cells in experimental antineoplastic therapy. Exp Hematol. 1993;21:1399-1400.
54. Rolink A, Kudo A, Karasuyama H, Kikuchi Y, Melchers F. Longterm proliferating early pre B cell lines and clones with the potential to develop to surface Ig-positive, mitogen reactive B cells in vitro and in vivo. EMBO J. 1991;10:327-336.
55. Crystal RG. Transfer of genes to humans: early lessons and obstacles to success. Science. 1995;270:404-410.
56. Battler A, Leor J, eds. Stem Cell and Gene-Based Therapy: Frontiers in Regenerative Medicine. London: Springer; 2006.
57. Roybal JL, Santore MT, Flake AW. Stem cell and genetic therapies for the fetus. Semin Fetal Neonatal Med. 2010;15:46-51.
58. Machida CA, ed. Viral Vectors for Gene Therapy: Methods and Protocols. New York, NY: Humana Press; 2003.
59. Curiel DT. Adenovirus facilitation of molecular conjugate- mediated gene transfer. Prog Med Virol. 1993;40:1-18.
60. Perales JC, Ferkol T, Molas M, Hanson RW. An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. Eur J Biochem. 1994;226:255-266.
61. Trowbridge IS, Newman RA, Domingo DL, Sauvage C. Transferrin receptors: structure and function. Biochem Pharmacol. 1984;33:925-932.
62. Aisen P, Listowsky I. Iron transport and storage proteins. Annu Rev Biochem. 1980;49:357-393.
63. Dautry-Varsat A, Ciechanover A, Lodish HF. pH and the recycling of transferrin during receptor-mediated endocytosis. Proc Natl Acad Sci U S A. 1983;80:2258-2262.
64. Schneider C, Owen MJ, Banville D, Williams JG. Primary structure of human transferrin receptor deduced from the mRNA sequence. Nature. 1984;311:675-678.
65. Zerial M, Melancon P, Schneider C, Garoff H. The transmembrane segment of the human transferrin receptor functions as a signal peptide. EMBO J. 1986;5:1543-1550.
66. Wilson JM, Grossman M, Wu CH, Chowdhury NR, Wu GY, Chowdhury JR. Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits. J Biol Chem. 1992;267:963-967.
67. Hart SL, Harbottle RP, Cooper R, Miller A, Williamson R, Coutelle C. Gene delivery and expression mediated by an integrinbinding peptide. Gene Ther. 1995;2:552-554.
68. Drazan KE, Wu L, Olthoff KM, Jurim O, Busuttil RW, Shaked A. Transduction of hepatic allografts achieves local levels of viral IL-10 which suppress alloreactivity in vitro. J Surg Res. 1995;59:219-223.
69. Qin L, Pahud DR, Ding Y, et al. Efficient transfer of genes into murine cardiac grafts by Starburst polyamidoamine dendrimers. Hum Gene Ther. 1998;9:553-560.
70. DeBruyne LA, Li K, Chan SY, Qin L, Bishop DK, Bromberg JS. Lipid-mediated gene transfer of viral IL-10 prolongs vascularized cardiac allograft survival by inhibiting donor-specific cellular and humoral immune responses. Gene Ther. 1998;5:1079-1087.
71. Le Doux JM, ed. Gene Therapy Protocols Volume 2: Design and Characterization of Gene Transfer Vectors. 3rd ed. New York, NY: Humana Press; 2008.
72. Harlow E, Whyte P, Franza BR Jr, Schley C. Association of adenovirus early-region 1A proteins with cellular polypeptides. Mol Cell Biol. 1986;6:1579-1589.
73. Engelhardt JF, Ye X, Doranz B, Wilson JM. Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc Natl Acad Sci U S A. 1994;91:6196-6200.
74. Mitani K, Graham FL, Caskey CT, Kochanek S. Rescue, propagation, and partial purification of a helper virus-dependent adenovirus vector. Proc Natl Acad Sci U S A. 1995;92:3854-3858.
75. Fisher KJ, Choi H, Burda J, Chen SJ, Wilson JM. Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology. 1996;217:11-22.
76. Clemens PR, Kochanek S, Sunada Y, et al. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther. 1996;3:965-972.
77. McCown TJ, Xiao X, Li J, Breese GR, Samulski RJ. Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 1996;713:99-107.
78. Kaplitt MG, Xiao X, Samulski RJ, et al. Long-term gene transfer in porcine myocardium after coronary infusion of an adeno- associated virus vector. Ann Thorac Surg. 1996;62:1669-1676.
79. Samulski RJ, Zhu X, Xiao X, et al. Targeted integration of adenoassociated virus (AAV) into human chromosome 19. EMBO J. 1991;10:3941-3950.
80. Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol. 1996;70:8098-8108. (Pubitemid 26339120)
81. Roizman B, Sears AE. Herpes simplex viruses and their replication. In: Fields BN, Knipe DM, Howley PM, et al, eds. Fields' Virology. 3rd ed. Philadelphia, Pa: Lippincott-Raven; 1996:2231-2295.
82. Cornetta K, Morgan RA, Anderson WF. Safety issues related to retroviral-mediated gene transfer in humans. Hum Gene Ther. 1991;2:5-14.
83. Mocarski ES, Post LE, Roizman B. Molecular engineering of the herpes simplex virus genome: insertion of a second L-S junction into the genome causes additional genome inversions. Cell. 1980;22:243-255.
84. Schnell MJ, Buonocore L, Kretzschmar E, Johnson E, Rose JK. Foreign glycoproteins expressed from recombinant vesicular stomatitis viruses are incorporated efficiently into virus particles. Proc Natl Acad Sci U S A. 1996;93:11359-11365.
85. Moss B. Genetically engineered poxviruses for recombinant gene expression, vaccination, and safety. Proc Natl Acad Sci U S A. 1996;93:11341-11348.
86. Neriishi S, Yabe Y, Oda N, et al. Effects in newborn mice of tumor-producing agents carried in tissue culture. J Natl Cancer Inst. 1961;26:611-619.
87. Schofield JP, Caskey CT. Non-viral approaches to gene therapy. Br Med Bull. 1995;51:56-71.
88. Davis HL, Demeneix BA, Quantin B, Coulombe J, Whalen RG. Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal muscle. Hum Gene Ther. 1993;4:733-740.
89. McDonnell WM, Askari FK. DNA vaccines. N Engl J Med. 1996;334:42-45.
90. Yang Y, Nunes FA, Berencsi K, Furth EE, Gönczöl E, Wilson JM. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A. 1994;91:4407-4411.
91. Cheng L, Ziegelhoffer PR, Yang NS. In vivo promoter activity and transgene expression in mammalian somatic tissues evaluated by using particle bombardment. Proc Natl Acad Sci U S A. 1993;90:4455-4459.
92. Ulmer JB, Donnelly JJ, Parker SE, et al. Heterologous protection against influenza by injection of DNA encoding a viral protein. Science. 1993;259:1745-1749.
93. Liu D, Song YK. Cationic liposome-mediated transfection in vivo (review). Gene Ther Mol Biol. 1998;2:59-68.
94. Hawley-Nelson P, Ciccarone V, Gebeyehu G, Jessee J. LipofectAMINE reagent: a new, higher efficiency polycationic liposome transfection reagent. Focus. 1993;15:73-79.
95. Caplen NJ, Kinrade E, Sorgi F, et al. In vitro liposome-mediated DNA transfection of epithelial cell lines using the cationic liposome DC-Chol/DOPE. Gene Ther. 1995;2:603-613.
96. Nabel GJ, Nabel EG, Yang ZY, et al. Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans. Proc Natl Acad Sci U S A. 1993;90:11307-11311.
97. Nabel GJ, Chang AE, Nabel EG, et al. Immunotherapy for cancer by direct gene transfer into tumors. Hum Gene Ther. 1994;5:57-77.
98. Nabel EG, Yang Z, Liptay S, et al. Recombinant platelet-derived growth factor B gene expression in porcine arteries induce intimal hyperplasia in vivo. J Clin Invest. 1993;91:1822-1829.
99. Keogh MC, Chen D, Lupu F, et al. High efficiency reporter gene transfection of vascular tissue in vitro and in vivo using a cationic lipid-DNA complex. Gene Ther. 1997;4:162-171.
100. Plank C, Tang MX, Wolfe AR, Szoka FC Jr. Branched cationic peptides for gene delivery: role of type and number of cationic residues in formation and in vitro activity of DNA polyplexes. Hum Gene Ther. 1999;10:319-332.
101. Kalderon D, Roberts BL, Richardson WD, Smith AE. A short amino acid sequence able to specify nuclear location. Cell. 1984;39:499-509.
102. Boussif O, Lezoualc'h F, Zanta MA, et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci U S A. 1995;92:7297-7301.
103. Lengerke C, Daley GQ. Autologous blood cell therapies from pluripotent stem cells. Blood Rev. 2010;24;27-37.
104. Renström J, Kröger M, Peschel C, Oostendorp RA. How the niche regulates hematopoietic stem cells. Chem Biol Interact. 2010;184:7-15.
105. Pages JC, Bru T. Toolbox for retrovectorologists. J Gene Med. 2004;6:S67-S82.
106. Ferguson C, Larochelle A, Dunbar CE. Hematopoietic stem cell gene therapy: dead or alive? Trends Biotechnol. 2005;23;589-597.
107. Coffin JD, Florkiewicz RZ, Neumann J, et al. Abnormal bone growth and selective translational regulation in basic fibroblast growth factor (FGF-2) transgenic mice. Mol Biol Cell. 1995;6:1861-1873.
108. MacMillan V, Walton-Roche K, Davis J. Acidic fibroblast growth factor infusion reduces ischemic CA1 hippocampal damage in the gerbil. Can J Neurol Sci. 1993;20:37-40.
109. Neufeld G, Tessler S, Gitay-Goren H, Cohen T, Levi BZ. Vascular endothelial growth factor and its receptors. Prog Growth Factor Res. 1994;5:89-97.
110. Mühlhauser J, Pili R, Merrill MJ, et al. In vivo angiogenesis induced by recombinant adenovirus vectors coding either for secreted or nonsecreted forms of acidic fibroblast growth factor. Hum Gene Ther. 1995;6:1457-1465.
111. Morishita R, Gibbons GH, Ellison KE, et al. Evidence for direct local effect of angiotensin in vascular hypertrophy. In vivo gene transfer of angiotensin converting enzyme. J Clin Invest. 1994;94:978-984.
112. Fox JC, Shanley JR. Antisense inhibition of basic fibroblast growth factor induces apoptosis in vascular smooth muscle cells. J Biol Chem. 1996;271:12578-12584.
113. Bennett MR, Anglin S, McEwan JR, Jagoe R, Newby AC, Evan GI. Inhibition of vascular smooth muscle cell proliferation in vitro and in vivo by c-myc antisense oligodeoxynucleotides. J Clin Invest. 1994;93:820-828.
114. Simons M, Edelman ER, Rosenberg RD. Antisense proliferating cell nuclear antigen oligonucleotides inhibit intimal hyperplasia in a rat carotid artery injury model. J Clin Invest. 1994;93:2351-2356.
115. Simons M, Rosenberg RD. Antisense nonmuscle myosin heavy chain and c-myb oligonucleotides suppress smooth muscle cell proliferation in vitro. Circ Res. 1992;70:835-843.
116. von der Leyen HE, Gibbons GH, Morishita R, et al. Gene therapy inhibiting neointimal vascular lesion: in vivo transfer of endothelial cell nitric oxide synthase gene. Proc Natl Acad Sci U S A. 1995;92:1137-1141.
117. Bradbury MW. The structure and function of the blood-brain barrier. Fed Proc. 1984;43:186-190.
118. Bradbury MW, Cole DF. The role of the lymphatic system in drainage of cerebrospinal fluid and aqueous humour. J Physiol. 1980;299:353-365.
119. Streilein JW, Toews GB, Bergstresser PR. Corneal allografts fail to express Ia antigens. Nature. 1979;282:326-327.
120. Streilein JW, Bradley D. Analysis of immunosuppressive properties of iris and ciliary body cells and their secretory products. Invest Ophthalmol Vis Sci. 1991;32:2700-2710.
121. Wilson SE, Li Q, Weng J, et al. The Fas-Fas ligand system and other modulators of apoptosis in the cornea. Invest Ophthalmol Vis Sci. 1996;37:1582-1592.
122. Streilein JW. Peripheral tolerance induction: lessons from immune privileged sites and tissues. Transplant Proc. 1996;28:2066-2070.
123. Streilein JW. Immunological non-responsiveness and acquisition of tolerance in relation to immune privilege in the eye. Eye (Lond). 1995;9:236-240.
124. Niederkorn JY. Immune privilege and immune regulation in the eye. Adv Immunol. 1990;48:191-226.
125. Niederkorn JY, Mellon J. Anterior chamber-associated immune deviation promotes corneal allograft survival. Invest Ophthalmol Vis Sci. 1996;37:2700-2707. (Pubitemid 27026555)
126. Vail A, Gore SM, Bradley BA, Easty DL, Rogers CA. Corneal transplantation in the United Kingdom and Republic of Ireland. Br J Ophthalmol. 1993;77:650-656.
127. Williams KA, Brereton HM, Coster DJ. Prospects for genetic modulation of corneal graft survival. Eye (Lond). 2009;23:1904-1909.
128. Williams KA, Coster DJ. The immunobiology of corneal transplantation. Transplantation. 2007;84:806-813.
129. Parker DG, Coster DJ, Brereton HM, et al. Lentivirus-mediated gene transfer of interleukin 10 to the ovine and human cornea. Clin Experiment Ophthalmol. 2010;38:405-413.
130. Kervick GN, Shepherd WF. The pattern of corneal graft rejection. Cornea. 1990;9:234-237.
131. Larkin DF, Alexander RA, Cree IA. Infiltrating inflammatory cell phenotypes and apoptosis in rejected human corneal allografts. Eye (Lond). 1997;11:68-74.
132. Duncan AW, Dorrell C, Gropmpe M. Stem cells and liver regeneration. Gastroenterology. 2009;137:466-481.
133. Berraondo P, Prieto J, Gonzalez-Aseguinolaza G. Advances in interleukin-12 gene therapy for acquired liver diseases. Curr Gene Ther. 2009;9:62-71.
134. Birraux J, Menzel O, Wildhaber B, Jond C, Nguyen TH, Chardot C. A step toward liver gene therapy: efficient correction of the genetic defect of hepatocytes isolated from a patient with Crigler-Najjar syndrome type 1 with lentiviral vectors. Transplantation. 2009;87:1006-1012.
135. Horiguchi, K, Hirano T, Ueki T, Hirakawa K, Fujimoto J. Treating liver cirrhosis in dogs with hepatocyte growth factor gene therapy via the hepatic artery. J Hepatobiliary Pancreat Surg. 2009;16:171-177.
136. Paez-Cortez J, Montano R, Iacomini J, Cardier J. Liver sinusoidal endothelial cells as possible vehicles for gene therapy: a comparison between plasmid-based and lentiviral gene transfer techniques. Endothelium. 2008;15:165-173.
137. Sharma R, Greenhough S, Medine CN, Hay DC. Three-dimensional culture of human embryonic stem cell derived hepatic endoderm and its role in bioartificial liver construction. J Biomed Biotechnol. 2010;2010:236147.
138. Yukawa H, Noguchi H, Oishi K, et al. Cell transplantation of adipose tissue-derived stem cells in combination with heparin attenuated acute liver failure in mice. Cell Transplant. 2009;18:611-618.
139. Khan AA, Parveen N, Mahaboob VS, et al. Safety and efficacy of autologous bone marrow stem cell transplantation through hepatic artery for the treatment of chronic liver failure: a preliminary study. Transplant Proc. 2008;40:1140-1144.
140. Kuo TK, Hung SP, Chuang CH, et al. Stem cell therapy for liver disease: parameters governing the success of using bone marrow mesenchymal stem cells. Gastroenterology. 2008;134:2111-2121.
141. Oertel M, Shafritz DA. Stem cells, cell transplantation and liver repopulation. Biochim Biophys Acta. 2008;1782:61-74.
142. Teimourian S, de Boer M, Roos D. Molecular basis of autosomal recessive chronic granulomatous disease in Iran. J Clin Immunol. 2010;30:587-592.
143. Freudenberg F, Wintergerst U, Roesen-Wolff A, et al. Therapeutic strategy in p47-phox deficient chronic granulomatous disease presenting as inflammatory bowel disease. J Allergy Clin Immunol. 2010;125:943-946.
144. Roos D, Kuhns DB, Maddalena A, et al. Hematologically important mutations: the autosomal recessive forms of chronic granulomatous disease (second update). Blood Cells Mol Dis. 2010;44:291-299.
145. Gentsch M, Kaczmarczyk A, van Leeuwen K, et al. Alu-repeat-induced deletions within the NCF2 gene causing p67-phoxdeficient chronic granulomatous disease (CGD). Hum Mutat. 2010;31:151-158.
146. Malech HL, Maples PB, Whiting-Theobald N, et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci U S A. 1997;94:12133-12138.
147. Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Gene therapy for immunodeficiency diseases. Semin Hematol. 2004;41:272-278.
148. Di Santo JP, Rodewald HR. In vivo roles of receptor tyrosine kinases and cytokine receptors in early thymocyte development. Curr Opin Immunol. 1998;10:196-207.
149. Rodig SJ, Meraz MA, White JM, et al. Disruption of the Jak1 gene demonstrates obligatory and nonredundant roles of the Jaks in cytokine-induced biologic responses. Cell. 1998;93:373-383.
150. Suzuki H, Duncan GS, Takimoto H, Mak TW. Abnormal development of intestinal intraepithelial lymphocytes and peripheral natural killer cells in mice lacking the IL-2 receptor beta chain. J Exp Med. 1997;185:499-505.
151. Izuhara K, Heike T, Otsuka T, et al. Signal transduction pathway of interleukin-4 and interleukin-13 in human B cells derived from X-linked severe combined immunodeficiency patients. J Biol Chem. 1996;271:619-622. (Pubitemid 26035766)
152. Wengler GS, Lanfranchi A, Frusca T, et al. In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDXI). Lancet. 1996;348:1484-1487.
153. Niehues T, Schwarz K, Schneider M, et al. Severe combined immunodeficiency (SCID) associated neutropenia: a lesson from monozygotic twins. Arch Dis Child. 1996;74:340-342.
154. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Role of interleukin-2 (IL-2), IL-7, and IL-15 in natural killer cell differentiation from cord blood hematopoietic progenitor cells and from gamma c transduced severe combined immunodeficiency X1 bone marrow cells. Blood. 1996;88:3901-3909.
155. Nicolas N, Finnie NJ, Cavazzana-Calvo M, et al. Lack of detectable defect in DNA double-strand break repair and DNA-dependent protein kinase activity in radiosensitive human severe combined immunodeficiency fibroblasts. Eur J Immunol. 1996;26:1118-1122.
156. Derry JM, Ochs HD, Franche U. Isolation of a novel gene mutated in Wiscott-Aldrich syndrome. Cell. 1994;78:635-644.
157. Bunnell SC, Henry PA, Kolluri R, Kirchhausen T, Rickles RJ, Berg LJ. Identification of Itk/Tsk Src homology 3 domain ligands. J Biol Chem. 1996;271:25646-25656.
158. Aspenström P, Lindberg U, Hall A. Two GTPases, Cdc42 and Rac, bind directly to a protein implicated in the immunodeficiency disorder Wiskott-Aldrich syndrome. Curr Biol. 1996;6:70-75.
159. Zicha D, Allen WE, Brickell PM, et al. Chemotaxis of macrophages is abolished in the Wiskott-Aldrich syndrome. Br J Haematol. 1998;101:659-665.
160. Kohn DB. Update on gene therapy for immunodeficiencies. Clin Immunol. 2010;135:247-254.
161. Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med. 2009;360:447-458.
162. Hacein-Bey-Abina S, Garrigue A, Wang GP, et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest. 2008;118:3132-3142.
163. Ott MG, Schmidt M, Schwarzwaelder K, et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006;12:401-409.
164. Mavilio F, Pellegrini G, Ferrari S, et al. Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med. 2006;12:1397-1402.
165. Howe SJ, Mansour MR, Schwarzwaelder K, et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest. 2008;118:3143-3150.