Gene therapy for chronic granulomatous disease

Expert Opinion on Biological Therapy

Volumn 4, Issue 9, 2004, Pages 1423-1434

  Barese, Cecilia N ^a   Goebel, W Scott ^a   Dinauer, Mary C ^a,b  

^a INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Chronic granulomatous disease; Gene therapy; Haematopoietic stem cell; Lentivirus; NADPH oxidase; Phagocytes; Retrovirus

Indexed keywords

ANTIBIOTIC AGENT; ANTIFUNGAL AGENT; CORTICOSTEROID; COTRIMOXAZOLE; FIBRONECTIN; GAMMA INTERFERON; INFLIXIMAB; ITRACONAZOLE; LENTIVIRUS VECTOR; PREDNISOLONE; RECOMBINANT REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE GP47 SUBUNIT; RECOMBINANT REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE GP91 SUBUNIT; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE; RETRONECTIN; RETROVIRUS VECTOR; SALAZOSULFAPYRIDINE; UNCLASSIFIED DRUG;

ANTIBIOTIC PROPHYLAXIS; BACTERIAL INFECTION; BONE MARROW; BONE MARROW PURGING; CELL RENEWAL; CELL REPOPULATION; CELL SURVIVAL; CLINICAL TRIAL; COLITIS; DRUG RESISTANCE; DRUG RESISTANCE GENE; ENZYME ACTIVITY; ENZYME RECONSTITUTION; EXPERIMENTAL TRANSPLANTATION; GENE; GENE EXPRESSION; GENE MUTATION; GENE THERAPY; GENE TRANSFER; GENETIC SELECTION; GRANULOMATOSIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNE DEFICIENCY; IN VITRO STUDY; KNOCKOUT MOUSE; MYCOSIS; NEUTROPHIL; NONHUMAN; NONMYELOABLATIVE CONDITIONING; NUCLEOTIDE SEQUENCE; PHAGOCYTE; RECURRENT INFECTION; REVIEW; X CHROMOSOME LINKED DISORDER; XENOGRAFT;

ANIMALS; BONE MARROW CELLS; BONE MARROW TRANSPLANTATION; CELL LINE, TRANSFORMED; CELLS, CULTURED; CLINICAL TRIALS, PHASE I; DISEASE SUSCEPTIBILITY; DOSAGE COMPENSATION, GENETIC; GENE THERAPY; GENETIC VECTORS; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFECTION; INFLAMMATION; MICE; MICE, KNOCKOUT; NADPH OXIDASE; PHAGOCYTES; PHAGOCYTOSIS; RECURRENCE;

BACTERIA (MICROORGANISMS); LENTIVIRUS; UNIDENTIFIED RETROVIRUS;

EID: 4544220302     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.9.1423     Document Type: Review

References (126)

1. HEYWORTH PG, CROSS AR, CURNUTTE JT: Chronic granulomatous disease. Curr. Opin. Immunol. (2003) 15(5):578-584.
2. WINKELSTEIN JA, MARINO MC, JOHNSTON RB JR et al.: Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine (Baltimore) (2000) 79(3):155-169. The largest and most complete epidemiology report on CGD.
3. ROOS D, DE BOER M, KURIBAYASHI F et al.: Mutations in the X-linked and autosomal recessive forms of chronic granulomatous disease. Blood (1996) 87(5):1663-1681.
4. SEGAL BH, LETO TL, GALLIN JI, MALECH HL, HOLLAND SM: Genetic, biochemical, and clinical features of chronic granulomatous disease. Medicine (Baltimore) (2000) 79(3):170-200. A complete review on genetics, biochemical mechanisms, clinical presentation and treatment of CGD.
5. GOLDBLATT D: Current treatment options for chronic granulomatous disease. Expert Opin. Pharmacother. (2002) 3(7):857-863.
6. HORWITZ ME, BARRETT AJ, BROWN MR et al.: Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N. Engl. J. Med. (2001) 344(12):881-888. An interesting study describing the efficacy of non-myeloablative allogeneic stem cell transplantation in 10 CGD patients.
7. HO CM, VOWELS MR, LOCKWOOD L, ZIEGLER JB: Successful bone marrow transplantation in a child with X-linked chronic granulomatous disease. Bone Marrow Transplant. (1996) 18(1):213-215.
8. CALVINO MC, MALDONADO MS, OTHEO E et al.: Bone marrow transplantation in chronic granulomatous disease. Eur. J. Pediatr. (1996) 155(10):877-879.
9. HOBBS JR, MONTEIL M, MCCLUSKEY DR, JURGES E, EL TUMI M: Chronic granulomatous disease 100% corrected by displacement bone marrow transplantation from a volunteer unrelated donor. Eur. J. Pediatr. (1992) 151(11):806-810.
10. KAMANI N, AUGUST CS, CAMPBELL DE, HASSAN NF, DOUGLAS SD: Marrow transplantation in chronic granulomatous disease: an update, with 6-year follow-up. J. Pediatr. (1988) 113(4):697-700.
11. WILLIAMS DA, LEMISCHKA IR, NATHAN DG, MULLIGAN RC: Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse. Nature (1984) 310(5977):476-480.
12. HU J, DUNBAR CE: Update on hematopoietic stem cell gene transfer using non-human primate models. Curr. Opin. Mol. Ther. (2002) 4(5):482-490.
13. GOEBEL W, KUME A, DINAUER M: Gene therapy for chronic granulomatous disease and leukocyte adhesion deficiency. Int. J. Ped. Hematol. Oncol. (2001) 7:359-372.
14. phox-deficient model of chronic granulomatous disease. Blood (1997) 89(7):2268-2275.
15. phox into bone marrow cells rescues defect in host defense against Aspergillus fumigatus in murine X-linked chronic granulomatous disease. Blood (1997) 89(1):41-48. First report of correction of an animal model of genetic blood disease using gene therapy.
16. DINAUER MC, GIFFORD MA, PECH N, LI LL, EMSHWILLER P: Variable correction of host defense following gene transfer and bone marrow transplantation in murine X-linked chronic granulomatous disease. Blood (2001) 97(12):3738-3745. A relevant study using the X-CGD knockout mouse model to demonstrate the efficacy of gene therapy and bone marrow transplantation to correct clinical manifestations of CGD.
17. LIESE J, KLOOS S, JENDROSSEK V et al.: Long-term follow-up and outcome of 39 patients with chronic granulomatous disease. J. Pediatr. (2000) 137(5):687-693.
18. CALE CM, JONES AM, GOLDBLATT D: Follow up of patients with chronic granulomatous disease diagnosed since 1990. Clin. Exp. Immunol. (2000) 120(2):351-355.
19. SPEERT DP, BOND M, WOODMAN RC, CURNUTTE JT: Infection with Pseudomonas cepacia in chronic granulomatous disease: role of nonoxidative killing by neutrophils in host defense. J. Infect. Dis. (1994) 170(6):1524-1531.
20. FOSTER CB, LEHRNBECHER T, MOL F et al.: Host defense molecule polymorphisms influence the risk for immune-mediated complications in chronic granulomatous disease. J. Clin. Invest. (1998) 102(12):2146-2155.
21. BROWN JR, GOLDBLATT D, BUDDLE J, MORTON L, THRASHER AJ: Diminished production of anti-inflammatory mediators during neutrophil apoptosis and macrophage phagocytosis in chronic granulomatous disease (CGD). J. Leukoc. Biol. (2003) 73(5):591-599.
22. SEGAL AW: The NADPH oxidase and chronic granulomatous disease. Mol. Med. Today. (1996) 2(3):129-135.
23. MORGENSTERN DE, GIFFORD MA, LI LL, DOERSCHUK CM, DINAUER MC: Absence of respiratory burst in X-linked chronic granulomatous disease mice leads to abnormalities in both host defense and inflammatory response to Aspergillus fumigatus. J. Exp. Med. (1997) 185(2):207-218.
24. PETERSEN JE, HIRAN TS, GOEBEL WS et al.: Enhanced cutaneous inflammatory reactions to Aspergillus fumigatus in a murine model of chronic granulomatous disease. J. Invest. Dermatol. (2002) 118(3):424-429.
25. YANG S, PANOSKALTSIS-MORTARI A, SHUKLA M, BLAZAR BR, HADDAD IY: Exuberant inflammation in nicotinamide adenine dinucleotide phosphate-oxidase-deficient mice after allogeneic marrow transplantation. J. Immunol (2002) 168(11):5840-5847.
26. MACEDO F, MCHUGH K, GOLDBLATT D: Pericardial effusions in two boys with chronic granulomatous disease. Pediatr. Radiol. (1999) 29(11):820-822.
27. BARESE CN, PODESTA M, LITVAK E, VILLA M, RIVAS EM: Recurrent eosinophilic cystitis in a child with chronic granulomatous disease. J. Pediatr. Hematol. Oncol. (2004) 26(3):209-212.
28. BAUER SB, KOGAN SJ: Vesical manifestations of chronic granulomatous disease in children. Its relation to eosinophilic cystitis. Urology (1991) 37(5):463-466.
29. GOLDBLATT D, BUTCHER J, THRASHER AJ, RUSSELL-EGGITT I: Chorioretinal lesions in patients and carriers of chronic granulomatous disease. J. Pediatr. (1999) 134(6):780-783.
30. PASIC S, MINIC A, MINIC P et al.: Long-term follow-up and prognosis of chronic granulomatous disease in Yugoslavia: is there a role for early bone marrow transplantation? J. Clin. Immunol. (2003) 23(1):55-61.
31. rd, HARBECK RJ, JOHNSTON RB JR: Recurrent severe infections in a girl with apparently variable expression of mosaicism for chronic granulomatous disease. J. Pediatr. (1985) 106(1):50-55.
32. CHOWDHURY MM, ANSTEY A, MATTHEWS CN: The dermatosis of chronic granulomatous disease. Clin. Exp. Dermatol. (2000) 25(3):190-194.
33. LOVAS JG, ISSEKUTZ A, WALSH N, MILLER RA: Lupus erythematosus-like oral mucosal and skin lesions in a carrier of chronic granulomatous disease. Chronic granulomatous disease carrier genodermatosis. Oral Surg. Oral. Med. Oral. Pathol. Oral. Radiol. Endod. (1995) 80(1):78-82.
34. GALLIN JI, ALLING DW, MALECH HL et al.: Itraconazole to prevent fungal infections in chronic granulomatous disease. N. Engl. J. Med. (2003) 348(24):2416-2422.
35. MOUY R, VEBER F, BLANCHE S et al.: Long-term itraconazole prophylaxis against Aspergillus infections in thirty-two patients with chronic granulomatous disease. J. Pediatr. (1994) 125(6 Pt 1):998-1003.
36. WEENING RS, LEITZ GJ, SEGER RA: Recombinant human interferon-gamma in patients with chronic granulomatous disease-European follow up study. Eur. J. Pediatr. (1995) 154(4):295-298.
37. EZEKOWITZ RA, DINAUER MC, JAFFE HS, ORKIN SH, NEWBURGER PE: Partial correction of the phagocyte defect in patients with X-linked chronic granulomatous disease by subcutaneous interferon gamma. N. Engl. J. Med. (1988) 319(3):146-151.
38. ISHIBASHI F, MIZUKAMI T, KANEGASAKI S et al.: Improved superoxide-generating ability by interferon gamma due to splicing pattern change of transcripts in neutrophils from patients with a splice site mutation in CYBB gene. Blood (2001) 98(2):436-441.
39. NO AUTHORS LISTED: A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease. The International Chronic Granulomatous Disease Cooperative Study Group. N. Engl. J. Med. (1991) 324(8):509-516.
40. AHLIN A, LARFARS G, BLINDER G, PALMBLAD J, GYLLENHAMMAR H: Gamma interferon treatment of patients with chronic granulomatous disease is associated with augmented production of nitric oxide by polymorphonuclear neutrophils. Clin. Diagn. Lab. Immunol. (1999) 6(3):420-424.
41. SCHIFF D, RAE J, MARTIN TR, DAVIS BH, CURNUTTE J: Increased phagocyte FcγRI expression and improved Fcγ-receptor mediated phagocytosis after in vivo recombinant human interferon-gamma treatment of normal human subjects. Blood (1997) 90:3187-3194.
42. SCHAPPI MG, SMITH W, GOLDBLATT D, LINDLEY KJ, MILLA PJ: Colitis in chronic granulomatous disease. Arch. Dis. Child. (2001) 84(2):147-151.
43. STEIN RB, HANAUER SB: Medical therapy for inflammatory bowel disease. Gastroenterol. Clin. North Am. (1999) 28(2):297-321.
44. SEGER RA, GUNGOR T, BELOHRADSKY BH et al.: Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000. Blood (2002) 100(13):4344-4350. A large study that describes the European experience on stem cell transplantation in severely ill CGD patients using myeloablation and non-depleted allografts.
45. ROSEN-WOLFF A, SOLDAN W, HEYNE K et al.: Increased susceptibility of a carrier of X-linked chronic granulomatous disease (CGD) to Aspergillus fumigatus infection associated with age-related skewing of lyonization. Ann. Hematol. (2001) 80(2):113-115.
46. CROSS AR, HEYWORTH PG, RAE J, CURNUTTE JT: A variant X-linked chronic granulomatous disease patient (X91+) with partially functional cytochrome b. J. Biol. Chem. (1995) 270(14):8194-8200.
47. SCHAPIRO BL, NEWBURGER PE, KLEMPNER MS, DINAUER MC: Chronic granulomatous disease presenting in a 69-year-old man. N. Engl. J. Med. (1991) 325(25):1786-1790.
48. 558-negative form of the disease. J. Leukoc. Biol. (1992) 51(2):164-171.
49. BORDIGNON C, RONCAROLO MG: Therapeutic applications for hematopoietic stem cell gene transfer. Nat. Immunol. (2002) 3(4):318-321.
50. BRENNER S, MALECH HL: Current developments in the design of oncoretrovirus and lentivirus vector systems for hematopoietic cell gene therapy. Biochim. Biophys. Acta (2003) 1640(1):1-24.
51. DONAHUE RE, DUNBAR CE: Update on the use of nonhuman primate models for preclinical testing of gene therapy approaches targeting hematopoietic cells. Hum. Gene Ther. (2001) 12(6):607-617.
52. MILLER DG, ADAM MA, MILLER AD: Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol. Cell. Biol. (1990) 10(8):4239-4242.
53. KIEM HP, ANDREWS RG, MORRIS J et al.: Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor. Blood (1998) 92(6):1878-1886.
54. DUNBAR CE, TAKATOKU M, DONAHUE RE: The impact of ex vivo cytokine stimulation on engraftment of primitive hematopoietic cells in a non-human primate model. Ann. NY Acad. Sci. (2001) 938:236-244; discussion 244-235.
55. MORITZ T, WILLIAMS DA: Gene transfer into the hematopoietic system. Curr. Opin. Hematol. (1994) 1(6):423-428.
56. MACNEILL EC, HANENBERG H, POLLOK KE et al.: Simultaneous infection with retroviruses pseudotyped with different envelope proteins bypasses viral receptor interference associated with colocalization of gp70 and target cells on fibronectin CH-296. J. Virol. (1999) 73(5):3960-3967.
57. WU T, KIM HJ, SELLERS SE et al.: Prolonged high-level detection of retrovirally marked hematopoietic cells in nonhuman primates after transduction of CD34+ progenitors using clinically feasible methods. Mol. Ther. (2000) 1(3):285-293.
58. SELLERS SE, TISDALE JF, AGRICOLA BA, DONAHUE RE, DUNBAR CE: The presence of the carboxy-terminal fragment of fibronectin allows maintenance of non-human primate long-term hematopoietic repopulating cells during extended ex vivo culture and transduction. Exp. Hematol. (2004) 32(2):163-170.
59. HUHN RD, TISDALE JF, AGRICOLA B et al.: Retroviral marking and transplantation of rhesus hematopoietic cells by nonmyeloablative conditioning. Hum. Gene Ther. (1999) 10(11):1783-1790.
60. ROSENZWEIG M, MACVITTIE TJ, HARPER D et al.: Efficient and durable gene marking of hematopoietic progenitor cells in nonhuman primates after nonablative conditioning. Blood (1999) 94(7):2271-2286.
61. AIUTI A, CATTANEO F, CASSANI B et al.: Safety and efficacy of stem cell gene therapy combined with nonmyeloablative conditioning for the treatment of ADA-SCID. Blood (2003) 102(11):154a.
62. AIUTI A, SLAVIN S, AKER M et al.: Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science (2002) 296(5577):2410-2413.
63. BECKER S, WASSER S, HAUSES M et al.: Correction of respiratory burst activity in X-linked chronic granulomatous cells to therapeutically relevant levels after gene transfer into bone marrow CD34+ cells. Hum. Gene Ther. (1998) 9(11):1561-1570.
64. phox. Blood (1996) 88(5):1834-1840.
65. KUME A, DINAUER MC: Retrovirus-mediated reconstitution of respiratory burst activity in X-linked chronic granulomatous disease cells. Blood (1994) 84(10):3311-3316.
66. phox. Proc. Natl. Acad. Sci. USA (1993) 90(21):9832-9836.
67. phox-deficient chronic granulomatous disease. Proc. Natl. Acad. Sci. USA (1993) 90(16):7446-7450.
68. 558 defective forms of chronic granulomatous disease. Blood (1994)84(1):53-58.
69. phox deficient chronic granulomatous disease using peripheral blood progenitor cells as a target for retrovirus mediated gene transfer. Blood (1997) 89(5):1754-1761.
70. phox expression. Blood (1996) 87(9):3722-3730.
71. GREZ M, BECKER S, SAULNIER S et al.: Gene therapy of chronic granulomatous disease. Bone Marrow Transplant. (2000) 25(Suppl. 2):S99-S104.
72. + cells.
73. LOGAN AC, LUTZKO C, KOHN DB: Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr. Opin. Biotechnol. (2002) 13(5):429-436.
74. HORN PA, KEYSER KA, PETERSON LJ et al.: Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol. Blood (2004) 103:3710-3716.
75. LEVASSEUR DN, RYAN TM, PAWLIK KM, TOWNES TM: Correction of a mouse model of sickle cell disease: lentiviral/antisickling β-globin gene transduction of immobilized, purified hematopoietic stem cells. Blood (2003) 102(13):4312-4319.
76. PERSONS DA, ALLAY ER, SAWAI N et al.: Successful treatment of mutine β-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood (2003) 102(2):506-513.
77. RIVELLA S, MAY C, CHADBURN A, RIVIERE I, SADELAIN M: A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer. Blood (2003) 101(8):2932-2939.
78. MAY C, RIVELLA S, CALLEGARI J et al.: Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin. Nature (2000) 406:82-86.
79. MIYOSHI H, SMITH KA, MOSIER DE, VERMA IM, TORBETT BE: Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science (1999) 283(5402):682-686.
80. GUENECHEA G, GAN OI, INAMITSU T et al.: Transduction of human CD34+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. Mol. Ther. (2000) 1(6):566-573.
81. phox corrects chronic granulomatous disease (CGD) phenotype in human X-CGD cells. J. Gene Med. (2000) 2(5):317-325.
82. phox lentivector corrects the oxidase defect in NOD/SCID mouse-repopulating peripheral blood-mobilized CD34+ cells from patients with X-linked chronic granulomatous disease. Blood (2002) 100(13):4381-4390.
83. POLLOCK JD, WILLIAMS DA, GIFFORD MA et al.: Mouse model of X-linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production. Nat. Genet. (1995) 9(2):202-209.
84. phox cDNA and functional expression in a human X-linked chronic granulomatous disease cell line. Blood (1996) 87(5):2005-2010.
85. DINAUER MC, LI LL, BJORGVINSDOTTIR H, DING C, PECH N: Long-term correction of phagocyte NADPH oxidase activity by retroviral-mediated gene transfer in murine X-linked chronic granulomatous disease. Blood (1999) 94(3):914-922.
86. PAWLIUK R, EAVES C, HUMPHRIES K: Sustained high-level reconstitution of the hematopoietic system of preselected hematopoietic cells expressing a transduced cell-surface antigen. Hum. Gene Ther. (1997) 8:1595-1604.
87. PERSONS DA, ALLAY ER, RIBERDY JM et al.: Use of green fluorescent protein as a marker to identify and track genetically modified hematopoietic cells. Nat. Med. (1998) 4:1201-1205.
88. BONINI C, GREZ M, TRAVERSARI C et al.: Safety of retroviral gene marking with a truncated NGF receptor. Nat. Med. (2003) 9(4):367-369.
89. DEOLA S, SCARAMUZZA S, BIROLO RS et al.: Mobilized blood CD34+ cells transduced and selected with a clinically applicable protocol reconstitute lymphopoiesis in SCID-Hu mice. Hum. Gene Ther. (2004) 15(3):305-311.
90. phox and a ΔLNGFR cell surface marker. Hum. Gene Ther. (2003) 14(7):651-666. An important paper on long-term reconstitution of NADPH oxidase activity using preselected transduced cells for transplantation of X-CGD knockout mice.
91. LI Z, DULLMANN J, SCHIEDLMEIER B et al.: Murine leukemia induced by retroviral gene marking. Science (2002) 296(5567):497.
92. GOEBEL WS, DINAUER MC: Gene therapy for chronic granulomatous disease. Acta Haematol. (2003) 110(2-3):86-92.
93. GOEBEL WS, DINAUER MC: Retroviral-mediated gene transfer and nonmyeloablative conditioning: studies in a murine X-linked chronic granulomatous disease model. J. Pediatr. Hematol. Oncol. (2002) 24(9):787-790.
94. GOEBEL WS, TRAVERS JB, PECH N, DINAUER MC: Retroviral mediated gene transfer (RMGT) reduces cutaneous inflammation and granuloma formation in a murine model of X-linked chronic granulomatous disease (X-CGD). Blood (2001) 98(11):695a.
95. 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. USA (1997) 94(22):12133-12138. The first report of a Phase I gene therapy trial in CGD patients.
96. phox transduced CD34+ cells. Blood (1998) 92(Suppl. 1):690a.
97. SCHRODER AR, SHINN P, CHEN H et al.: HIV-1 integration in the human genome favors active genes and local hotspots. Cell (2002) 110(4):521-529.
98. WU X, LI Y, CRISE B, BURGESS SM: Transcription start regions in the human genome are favored targets for MLV integration. Science (2003) 300(5626):1749-1751.
99. CORNETTA K: Safety aspects of gene therapy. Br. J. Haematol. (1992) 80(4):421-426.
100. DONAHUE RE, KESSLER SW, BODINE D et al.: Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J. Exp. Med. (1992) 176(4):1125-1135.
101. HACEIN-BEY-ABINA S, VON KALLE C, SCHMIDT M et al.: LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 302(5644):415-419.
102. WILLIAMS DA, BAUM C: Medicine. Gene therapy-new challenges ahead. Science (2003) 302(5644):400-401.
103. VASSILOPOULOS G, JOSEPHSON NC, TROBRIDGE G: Development of foamy virus vectors. Methods Mol. Med. (2003) 76:545-564.
104. SCHMIDT M, GLIMM H, WISSLER M et al.: Efficient characterization of retrolenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Ann. NY Acad. Sci. (2003) 996:112-121.
105. LEURS C, JANSEN M, POLLOK KE et al.: Comparison of three retroviral vector systems for transduction of nonobese diabetic/severe combined immunodeficiency mice repopulating human CD34+ cord blood cells. Hum. Gene Ther. (2003) 14(6):509-519.
106. ALI M, TAYLOR GP, PITMAN RJ et al.: No evidence of antibody to human foamy virus in widespread human populations. AIDS Res. Hum. Retroviruses (1996) 12(15):1473-1483.
107. JOSEPHSON NC, TROBRIDGE G, RUSSELL DW: Transduction of long-term and mobilized peripheral blood-derived NOD/SCID repopulating cells by foamy virus vectors. Hum. Gene Ther. (2004) 15(1):87-92.
108. GOEBEL WS, YODER MC, PECH NK, DINAUER MC: Donor chimerism and stem cell function in a murine congenic transplantation model after low-dose radiation conditioning: effects of a retroviral-mediated gene transfer protocol and implications for gene therapy. Exp. Hematol. (2002) 30(11):1324-1332.
109. KURAMOTO K, FOLLMAN D, HEMATTI P et al.: The impact of low-dose busulfan on clonal dynamics in non-human primates. Blood (2004) (In Press).
110. SUGIMOTO Y, TSUKAHARA S, SATO S et al.: Drug-selected co-expression of P-glycoprotein and gp91 in vivo from an MDR1-bicistronic retrovirus vector Ha-MDR-IRES-gp91. J. Gene Med. (2003) 5(5):366-376.
111. PERSONS DA, ALLAY JA, BONIFACINO A et al.: Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors. Blood (2004) 103(3):796-803.
112. GERSON SL: MGMT: its role in cancer aetiology and cancer therapeutics. Nat. Rev. Cancer (2004) 4(4):296-307.
113. ALLAY JA, PERSONS DA, GALIPEAU J et al.: In vivo selection of retrovirally transduced hematopoietic stem cells. Nat. Med. (1998) 4(10):1136-1143.
114. POLLOK KE, HARTWELL JR, BRABER A et al.: In vivo selection of human hematopoietic cells in a xenograft model using combined pharmacologic and genetic manipulations. Hum. Gene Ther. (2003) 14(18):1703-1714.
115. BOWMAN JE, REESE JS, LINGAS KT, GERSON SL: Myeloablation is not required to select and maintain expression of the drug-resistance gene, mutant MGMT, in primary and secondary recipients. Mol. Ther. (2003) 8(1):42-50.
116. PERSONS DA, ALLAY JA, BONIFACINO A et al.: Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques transplanted with hematopoietic stem cells expressing dihydrofolate reductase vectors. Blood (2003) 103(3):796-803.
117. ZIELSKE SP, REESE JS, LINGAS KT, DONZE JR, GERSON SL: In vivo selection of MGMT (P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning. J. Clin. Invest. (2003) 112:1561-1570.
118. NEFF T, HORN PA, PETERSON LJ et al.: Methylguanine methyltransferase- mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J. Clin. Invest. (2003) 112(10):1581-1588.
119. 6-methylguanine-DNA methyltransferase-transduced marrow progenitors repopulate nonmyeloablated mice after drug selection. Blood(2000) 95(10):3078-3084.
120. ANTONCHUK J, SAUVAGEAU G, HUMPHRIES RK: HOXB4-induced expansion of adult hematopoietic stem cells ex vivo. Cell (2002) 109(1):39-45.
121. SAWAI N, PERSONS DA, ZHOU S, LU T, SORRENTINO BP: Reduction in hematopoietic stem cell numbers with in vivo drug selection can be partially abrogated by HOXB4 gene expression. Mol. Ther. (2003) 8(3):376-384.
122. RICHARD RE, WEINREICH M, CHANG KH et al.: Modulating erythrocyte chimerism in a mouse model of pyruvate kinase deficiency. Blood (2004) 103(12):4432-4439.
123. RICHARD RE, WOOD B, ZENG H et al.: Expansion of genetically modified primary human hemopoietic cells using chemical inducers of dimerization. Blood (2000) 95(2):430-436.
124. NEFF T, HORN PA, VALLI VE et al.: Pharmacologically regulated in vivo selection in a large animal. Blood (2002) 100(6):2026-2031.
125. NAGASHIMA T, UEDA Y, HANAZONO Y et al.: In vivo expansion of gene-modified hematopoietic cells by a novel selective amplifier gene utilizing the erythropoietin receptor as a molecular switch. J. Gene Med. (2004) 6(1):22-31.
126. KUME A, KOREMOTO M, XU R et al.: In vivo expansion of transduced murine hematopoietic cells with a selective amplifier gene. J. Gene Med. (2003) 5(3):175-181.