Strategies of gene transfer to the kidney

Kidney International

Volumn 53, Issue 2, 1998, Pages 264-272

  Imai, Enyu ^a   Isaka, Yoshitaka ^a  

^a OSAKA UNIVERSITY MEDICAL SCHOOL   (Japan)

Author keywords

Adenovirus; Gene therapy; HVJ liposome method; Liposome; Retrovirus

Indexed keywords

ANTISENSE OLIGODEOXYNUCLEOTIDE; LIPOSOME; OLIGONUCLEOTIDE; VIRUS VECTOR;

ADENOVIRUS; EXPRESSION VECTOR; GENE TECHNOLOGY; GENE THERAPY; GENE TRANSFER; HUMAN; KIDNEY COLLECTING TUBULE; KIDNEY MEDULLA; KIDNEY PELVIS; KIDNEY PERFUSION; KIDNEY TRANSPLANTATION; NEPHROLOGY; PARAINFLUENZA VIRUS 1; PRIORITY JOURNAL; REVIEW;

EID: 0031910363     PISSN: 00852538     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1523-1755.1998.00768.x     Document Type: Article

References (92)

1. BLEASE R, CULVER K, MILLER A, CARTER C, FLEISHER T, CLERICI M, SHEARER G, CHANG L, CHANG Y, TOLSTOSHEV P, GREENBLATT J, RSENBERG S, KLEIN H, BERGER M, MULLEN C, RAMSEY W, MUUL L, MORGAN R, ANDERSON W: T lymphocyte directed gene therapy for ADA-SCID: Initial trial results after 4 years. Science 270:475-480, 1995
2. MARCEL T, GRAUSZ J: The TMC worldwide gene therapy enrollment report, end 1996. Hum Gene Ther 8:775-800, 1997
3. ORKIN S, MOTULSKY A: NIH, Report and Recommendations of the Panel to Assess the Investment in Research on Gene Theraphy. Bethesda, National Institutes of Health, 1995
4. LEIDEN J: Gene therapy-Promise, pitfalls, and prognosis. N Engl J Med 333:871-873, 1945
5. CRYSTAL G: Transfer of genes to humans: Early lessons and obstacles to success. Science 270:404-411, 1495
6. HARRIS J, LEMOINE N: Strategies for targeted gene therapy. TIG 12:400-405, 1996
7. FRIEDMANN T: Human gene therapy - An immature genie, but certainly out of the bottle. Nature Med 2:144-147, 1996
8. ROSS G, ERICKSON R, KNORR D, MOTULSKY A, PARKMAN R, SAMULSKI J, STRAUS S, SMITH B: Gene therapy in the United States: A five-year status report. Hum Gene Ther 7:1781-1790, 1996
9. ROTH J, CRISTIANO R: Gene therapy for cancer. JNCI 89:21-39, 1997
10. KITAMURA M: Transfer of exogenous genes into the kidney. Exp Nephrol 2:313-317, 1994
11. RILEY DLW: The potential gene therapy for treatment of kidney diseases. Semin Nephrol 15:57-69, 1995
12. FINE LG: Gene transfer into the kidney: Promise for unravelling disease mechanisms, limitations for human gene therapy. (Editorial Review) Kidney Int 49:612-619, 1996
13. LIPKOWITZ M, KLOTMAN M, BRUGGEMAN L, NICKLIN P, HANSS B, RAPPAPORT J, KLOTMAN P: Molecular therapy for renal diseases. Ann J Kidney Dis 28:475-492, 1996
14. ISAKA Y, IMAI E: Molecular biological intervention. Semin Nephrol 16:591-598, 1996
15. NALDINI L, BLOMER U, GAGE F, TRONO D, VERMA I: Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 93:11382-11388, 1996
16. NALDINI L, BLOMER U, GALLAY P, ORY D, MULLIGAN R, GAGE F, VERMA I, TRONO D: In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263-267, 1996
17. YANG Y, LI Q, ERTL H, WILSON J: Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 69:2004-2015, 1995
18. ENGELHARDT J, YE X, DORANZ B, WILSON J: Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc Natl Acad Sci USA 91:6196-6120, 1994
19. GORZIGLIA M, KADAN M, YEI S, LIM J, LEE G, LUTHRA R, TRAPNELL B: Elimination of both E1 and E2a from adenovirus vectors further improves prospects for in vivo human gene therapy. J Virol 70:4173-4178, 1996
20. YEH P, DEDIEU J, ORSINI C, VIGNE E, DENEFLE P, PERRICAUDET M: Efficient dual transcomplementation of adenovirus E1 and E4 regions from a 293-derived cell line expressing a minimal E4 functional unit. J Virol 70:559-565, 1996
21. DEMATTEO R, CHU G, AHN M, CHANG E, BARKER C, MARKMANN J: Long-lasting adenovirus transgene expression in mice through neonatal intrathymic tolerance infduction without the use of immunosuppression. J Virol 71:5330-5335, 1997
22. PONNAZHAGAN S, ERIKSON D, KEARNS W, ZHOU S, NAHREINI P, WANG X, SRIVASTAVA A: Lack of site-specific integration of the recombinant adeno-asociated virus 2 genomes in human cells. Hum Gene Ther 8:275-284, 1997
23. BAUDARD M, FLOTTE T, ARAN J, THIERRY A, PASTAN I, PANG M, KEARNS W, GOTTESMAN M: Expression of the human multidrug resistance and glucocerebrosidase cDNA from adeno-associated vectors: Efficient promotor activity of AAV sequences and in vivo delivery via liposomes. Hum Gene Ther 7:1309-1322, 1996
24. KESSLER P, PODSAKOFF G, CHEN X, MCQUISTON S, CPLOSI P, MATELIS L, KURTZMAN G, BYRNE B: Gene delivery to skeletal muscle results in sutained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 93:14082-14087, 1996
25. FISHER K, JOOSS K, ALSTON J, YANG Y, HAECKER S, HIGH K, PATHAK R, RAPER S, WILSON J: Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med 3:306-312, 1997
26. YANAGIHAWRA I, KANEDA Y, INUI K, OKADA S: Liposome-Mediated Gene Transfer. Edited by DICKSON G, Molecular, Cell Biology of Human Gene Therapeutics, London, Chapman and Hall, 1995
27. FELGNER P, GADEK T, HOLM M, ROMAN R, CHAN H, WENZ M, NORTHROP J, RINGOLD G, DANIELSEN M: Lipofectin: A highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 84:7413-7417. 1987
28. LEDLEY F: Nonviral gene therapy: The promise of genes as pharmaceutical products. Hum Gene Ther 6:1129-1144, 1995
29. GOYAL K, HUANG L: Gene therapy using DC-chol liposomes. J liposome Res 5:49-60, 1995
30. WHEELER C, FELGNER P, TSAI Y, MARSHALL J, SUKHU L, DOH S, HARTIKKA J, NIETUPSKI J, MANTHORPE M, NICHOLS M, PLEWE M, LIANG X, NORMAN J, SMITH A, CHENG S: A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc Natl Acad Sci USA 93:11454-11459, 1996
31. LEE E, MARSHALL J, SIEGEL C, JIANG C, YEW N, NICHOLS M, NIETUPSKI J, ZIEGLER R, LANE M, WANG K, WAN N, SCHEULE R, HARRIS D, SMITH A: Detailed analysis of structure and formations of cationic lipids for efficient gene transfer to the lung. Hum Gene Ther 7:1701-1707, 1996
32. OKADA Y: Sendai virus-induced cell fusion. Meth Enzymol 221:18-41, 1993
33. KANEDA Y, MORISHITA R, TOMITA N: Increased expression of DNA cointroduced with nuclear protein in adult rat liver. J Mol Med 73:289-297, 1995
34. WANG C, HUANG L: pH-sensitive immunoliposomes mediate target-cell-specific delivery and controlledexpression of a foreign gene in mouse. Proc Natl Acad Sci USA 84:7851-7855, 1987
35. SPRAGG D, ALFORD D, GREFERATH R, LARSEN C, LEE K, GURTNER G, CYBULSKY M, TOSI P, NICOLAU C, GIMBRONE M JR: Immunotargeting of liposomes to activated vascular endothelial cells: A strategy for site-selective delivery in the cardiovascular system. Proc Natl Acad Sci USA 94:8795-8800, 1997
36. FENDER P, RUIGROK R, GOUT E, BUFFET S, CHROBOCZEK J: Adenovirus dodecahedron, a new vector for human gene transfer. Nature Biotech 15:52-56, 1997
37. KOSEKI C, HERZLINGER D, AL-AWQATI Q: Integration of embryonic cells carrying a reporter gene into functioning nephrons. Am J Physiol 261:C550-C554, 1991
38. AKAGI Y, ISAKA Y, ARAI M, KANEKO T, TAKENAKA M, MORIYAMA T, KANEDA Y, ANDO A, ORITA Y, KAMADA T, UEDA N, IMAI E: Inhibition of TGF-B1 expression by antisense oligonucleotides suppressed extracellular matrix accumulation in experimental glomerulonephritis. Kidney Int 50:148-155, 1996
39. IMAI E, ISAKA Y, AKAGI Y, KANEDA Y: Gene transfer into the glomerulus by the hemagglutinating virus of Japan-liposome method. Exp Nephrol 5:112-117, 1997
40. TOMITA N, HIGAKI J, MORISHITA R, KATO K, MIKAMI H, KANEDA Y, OGIHARA T: Direct in vivo gene introduction into rat kidney. Biochem Biophys Res Commun 186:129-134, 1992
41. ISAKA Y, FUJIWARA Y, UEDA N, KANEDA Y, KAMADA T, IMAI E: Glomerulosclerosis induced by in vivo transfection of transforming growth factor-beta or platelet-derived growth factor gene into the rat kidney. J Clin Invest 92:2597-2601, 1993
42. ARAI M, WADA A, ISAKA Y, AKAGI Y, SUGIURA T, MIYAZAKI M, MORIYAMA T, KANEDA Y, NARUSE K, NARUSE M, OKITA Y, ANDO A, KAMADA T, UEDA N, IMAI E: In vivo transfection of genes for renin and angiotensinogen into the glomerular cells induced phenotypic change of the mesangial cells and glomerular sclerosis. Biochem Biophys Res Comm 206:525-532, 1995
43. HEIKKILA P, PARPALA T, LUKKARINEN O, WEBER M, TRYGGVASON K: Adenovirus-mediated gene transfer into kidney glomeruli using an ex vivo and in vivo kidney perfusion system-first steps towards gene therapy of Alport syndrome. Gene Ther 3:21-27, 1996
44. TRYGGVASON K, HEIKKIKA P, PETTERSSON E, TIBELL A, THORNER P: Can Alport syndrome be treated by gene therapy? Kidney Int 1493-1499, 1997
45. KITAMURA M, TAYLOR S, UNWIN R, BURTON S, SHIMIZU F, FINE LG: Gene transfer into the rat renal 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, 1994
46. KITAMURA M: Gene delivery into the glomerulus via mesangial cell vectors. Exp Nephrol 5:118-125, 1997
47. RAPPAPORT J, HANSS B, KOPP J, COPELAND T, BRUGGEMAN L, COFFMAN T, KLOTMAN P: Transport of phosphorothioute oligonucleotides in kidney: Implications for molecular therapy. Kidney Int 47:1462-1469, 1995
48. OBERBAUER R, SCHREINER G, MEYER T: Renal uptake of an 18-mer phosphorothioate pligonucleotide. Kidney Int 48:1226-1232, 1995
49. +/Pi cotransporter by antisense oligonucleotides. Proc Natl Acad Sci USA 93:4903-4906, 1996
50. LIEN Y, LAN LW: Liposome-mediated gene transfer into kidney tubules. Exp Nephrol 5:132-136, 1997
51. BOSCH R, WOOLF A, FINE L: Gene transfer into the mammalian kidney: Direct retrovirus transduction of regenerating tubular epithelial cells. Exp Nephrol 1:49-54, 1993
52. MOULLIER P, FRIEDLANDER G, CALISE D, RONCO P, PERRICAUDET M, FERRY N: Adenoviral-mediated gene transfer to renal tubular cells in vivo. Kidney Int 45:1220-1225, 1994
53. ZHU G, NICOLSON A, COWLEY B, ROSEN S, SUKHATME V: In vivo adenovirus-mediated gene transfer into normal and cystic rat kidneys. Gene Ther 3:298-304, 1996
54. ZEIGLER S, KERBY J, CURIEL D, DIETHELM A, THOMPSON J: Molecular conjugate-mediated gene transfer into isolated human kidneys. Transplantation 61:812-817, 1996
55. KELLY V, MOORE K: Application of a gene transfer strategy to identify molecules that incite autoimmune kidney injury. Exp Nephrol 5:144-151, 1997
56. NAITO T, YOKOYAMA H, MOORE K, DRNOFF G, MULLIGAN C, KELLY V: Macrophage growth factors introduced into the kidney initiate renal injury. Mol Med 2:297-312, 1996
57. STEIN C, CHENG Y: Antisense oligonucleotides as therapeutic agents Is the bullet really magical? Science 261:1004-1012, 1993
58. WANGER R: Gene inhibition using antisense oligodeoxynucleotides. Nature 372:333-335, 1994
59. ASKARI FM: Antisense-oligonucleotide therapy. N Engl J Med 334: 316-318, 1996
60. AKHTAR S, AGRAWAL S: In vivo studies with antisense oligonucleotides. TiPS 18:12-19, 1997
61. DUFF J, MONIA B, BERK B: Mitogen-activated protein (MAP) kinase is regulated by the MAP kinase phosphatase (MKP-1) in vascular smooth muscle cells. J Biol Chem 270:7161-7166, 1995
62. BURGESS T, FISHER E, ROSS S, BREADY J, QIAN Y, BAYEWITCH L, COHEN A, HERRERA C, HU S, KRAMER T, LOTT F, MARTIN F, PIERCE G, SIMONET L, FARRELL C: The antiproliferative activity of c-myb and c-myc antisense oligonucleotides in smooth muscle cells is caused by a nonsense mechanism. Prac Natl Acad Sci USA 92:4051-4055, 1995
63. PEREZ J, STEIN C, MAJUMDER S, OORSCHOT A, NARAYANAN R: Sequence-independent induction of Sp1 transcription factor activity by phospholotioate oligonucleotides. Proc Natl Acad Sci USA 91: 5957-5961, 1994
64. KASHIHAKA N, MAESHIMA Y, MAKINO H: Therapeutic intervention in glomerulonephritis by oligonucleotides. Exp Nephrol 5:126-131, 1997
65. NOIRI E, PERESLENI T, MILLER F, GOLIGORSKY MS: In vivo targeting of inducible NO synthase with oligodeoxynucleotides protects rat kidney against ischemia. J Clin Invest 97:2377-2383, 1996
66. HALLER H, DRAGUN D, MIETHKE A, PARK J, WEIS A, LIPPOLDT A, GROB V, LUFT F: Antisense oligonucleotides for ICAM-1 attenuate reperfusion injury and renal failure in the rat. Kidney Int 50:473-480, 1996
67. MORISHITA R, GIBBONS G, HORIUCHI M, ELLISON K, NAKAJIMA M, ZHANG L, KANEDA Y, OGIHARA T, DZAU V: A gene therapy strategy using a transcription factor decoy of the E2F binding site inhibits smooth muscle proliferation in vivo. Proc Natl Acad Sci USA 92:5855-5859, 1995
68. DZAU V, MANN M, MORISHITA R, KANEDA Y: Fusogenic viral liposome for gene therapy in cardiovascular diseases. Proc Natl Acad Sci USA 93:11421-11425, 1996
69. MILLER A: Putting muscle to work for gene therapy. Nature Med 3:278-279, 1997
70. TRIPATHY S, GOLDWASSER E, LU M, BARR E, LEIDEN J: Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus. Proc Natl Acad Sci USA 91:11557-11561, 1994
71. BLAU H, SPRINGER M: Muscle-mediated gene thrapy. N Engl J Med 333:1554-1556, 1995
72. ISAKA Y, AKAGI Y, KANEDA Y, YAMAUCHI A, ORITA Y, UEDA N, IMAI E: Gene therapy by TGF-β-receptor-IgG Fc chimera inhibited extracellular matrix accumulation in experimental glomerulonephritis. J Am Soc Nephrol 7:1735, 1996
73. SETOGUCHI Y, DANIEL C, CRYSTAL R: Stimulation of erythropoiesis by in vivo gene therapy: Physiologic consequences of transfer of the human erythropoietin gene to experimental animals using an adenovirus vector. Blood 84:2946-2953, 1994
74. WELLING T, DAVIDSON B, ZELENOCK J, STANLEY J, GORDON D, ROESSLER B, MESSINA L: Systemic delivery of the interleukin-1 receptor antagonist protein using a new strategy of direct adenoviral-mediated gene trabsfer to skeletal muscle capillary endothelium in the isolated rat hindlimb. Hum Gene Ther 7:1795-1802, 1996
75. KITAMURA M, BURTON S, ENGLISH J, KAWACHI H, FINE L: Transfer of a mutated gene encoding active transforming growth factor-B1 suppresses mitogenesis and IL-1 response in the glomerulus. Kidney Int 48:1747-1757, 1995
76. GOSSEN M, BUJARD H: Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA 89:5547-5551, 1992
77. KITAMURA M: Creation of a reversible on/off system for site-specific in vivo control of exogenous gene activity in the renal glomerulus. Proc Natl Acad Sci USA 93:7387-7391, 1996
78. BOHL D, NAFFAKH N, HEARD J: Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblasts. Nature Med 3:299-305, 1997
79. NO D, YAO T, EVANS R: Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc Natl Acad Sci USA 93:3346-3351, 1996
80. DELORT J, CAPECCHI M: TAXI/UAS: A molecular switch to control expression of genes in vivo. Hum Gene Ther 7:809-820, 1996
81. SAUER B, HENDERSON N: Cre-stimulated recommbination at LoxP-containing DNA sequences placed into the mammalian genome. Nucl Acids Res 17:147-161, 1989
82. GU H, MARTH J, ORBAN P, MOSSMANN H, RAJEWSKY K: Deletion of a DNA polymerase b gene segment in T cells using cell type-specific gene targeting. Science 265:103-106, 1994
83. ANTON M, GRAHAM F: Site-specific recombination mediated by an adenovirus vector expressing the Cre recombinase protein: A molecular switch for control of gene expression. J Virol 69:4600-4606, 1995
84. KANEGAE Y, LEE G, SATO Y, TANAKA M, NAKAI M, SASAKI T, SUGANO S, SAITO I: Efficient gene activation in mammalian cells by using recombinant adenovirus expressing site-specific Cre recombinase. Nucl Acids Res 23:3816-3821, 1995
85. MILLER N, WHELAN J: Progress in transcriptionally targeted and regulatable vectors for genetic therapy. Hum Gene Ther 8:803-815, 1997
86. BEALE E, CLOUTHIER D, HAMMER R: Cell-specific expression of cytosolic phosphoenolpyruvate carboxykinase in transgenic mice. FASEB J 6:3330-3337, 1992
87. IGARASHI P, WHYTE D, LI K, NAGAMI G: Cloning and kidney cell specific activity of the promoter of the murine renal Na-K-Cl cotransporter gene. J Biol Chem 271:9666-9674, 1996
88. FUSHIMI K, UCHIDA S, HARA Y, MARUMO F, SASAKI S: Cloning and expression of apical membrane water channel of rat kidney collecting tubule. Nature 361:549-552, 1993
89. KITAMURA M, SUTO T: Transfer of genetically engineered macrophages into the glomerulus. Kidney Int 51:1274-1279, 1997
90. YOKOO T, KITAMURA M: Gene transfer of interleukin-1 receptor antagonist into the renal glomerulus via a mesangial cell vector. Biochem Biophys Res Comm 226:883-888, 1996
91. VANDENBURGH H, TOTTO M, SHANSKY J, LEMAIRE J, CHANG A, PAYUMO F, GOODYAER A, RAVEN L: Tissue-engineered skeletal muscle organoids for reversible gene therapy. Hum Gene Ther 7:2195-2200, 1996
92. HUMES H: Application of gene and cell therapies in the tissue engineering of a bioartificial kidney. Int J Artif Organs 19:215-217, 1996