Hematopoietic stem cell gene therapy of murine protoporphyria by methylguanine-DNA-methyltransferase-mediated in vivo drug selection

Gene Therapy

Volumn 11, Issue 22, 2004, Pages 1638-1647

  Richard, E ^a   Robert, E ^a   Cario Andre M ^a   Ged, C ^a   Geronimi F ^a   Gerson, S L ^b   de Verneuil, H ^a   Moreau Gaudry, F ^a  

^a INSERM   (France)

^b CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

BCNU; Erythroid specific expression; Hematopoietic stem cells; In vivo drug selection; MGMT; Protoporphyria

Indexed keywords

6 O BENZYLGUANINE; CARMUSTINE; DNA; FERROCHELATASE; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; METHYLATED DNA PROTEIN CYSTEINE METHYLTRANSFERASE; PROTOPORPHYRIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL POPULATION; CELL SELECTION; CISTRON; CONTROLLED STUDY; DISEASE MODEL; DISEASE SEVERITY; DNA MODIFICATION; ERYTHROCYTE COUNT; ERYTHROPOIETIC PROTOPORPHYRIA; GENE EXPRESSION; GENE MUTATION; GENE THERAPY; GENE TRANSFER; GRAFT REJECTION; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IN VIVO STUDY; INHERITANCE; MOUSE; NONHUMAN; PHOTODERMATOSIS; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN EXPRESSION; TREATMENT OUTCOME;

ANIMALS; ANTINEOPLASTIC AGENTS; CARMUSTINE; FEMALE; FERROCHELATASE; GENE THERAPY; GENETIC ENGINEERING; GENETIC VECTORS; GUANINE; LENTIVIRUS; MALE; MICE; MICE, INBRED BALB C; MODELS, ANIMAL; O(6)-METHYLGUANINE-DNA METHYLTRANSFERASE; PROTOPORPHYRIA, ERYTHROPOIETIC; STEM CELL TRANSPLANTATION; STEM CELLS; TRANSDUCTION, GENETIC;

ANIMALIA; MURINAE;

EID: 8844226671     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302335     Document Type: Article

References (45)

1. Cavazzana-Calvo M et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000; 288 669-672.
2. Hacein-Bey-Abina S et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002; 346: 1185-1193.
3. Schmidt M et al. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat Med 2003; 9: 463-468.
4. Uchida N et al. HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. Proc Natl Acad Sci USA 1998; 95 11939-11944.
5. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 1999; 283: 682-686.
6. - human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci USA 1999; 96: 2988-2993.
7. - cell transduction via lentivirus-based gene transfer vectors. Hum Gene Ther 1999; 10: 1479-1489.
8. Sirven A et al. Enhanced transgene expression in cord blood CD34(+)-derived hematopoietic cells, including developing T cells and NOD/SCID mouse repopulating cells, following transduction with modified trip lentiviral vectors. Mol Ther 2001; 3: 438-448.
9. Miyoshi H et al. Development of a self-inactivating lentivirus vector. J Virol 1998; 72: 8150-8157.
10. Zufferey R et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 1998; 72: 9873-9880.
11. Wu X et al. Development of a novel trans-lentiviral vector that affords predictable safety. Mol Ther 2000; 2: 47-55.
12. Logan AC, Lutzko C, Kohn DB. Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr Opin Biotechnol 2002; 13: 429-436.
13. Zaiss AK, Son S, Chang LJ. RNA 3′ readthrough of oncoretrovirus and lentivirus: implications for vector safety and efficacy. J Virol 2002; 76: 7209-7219.
14. Ramezani A, Hawley TS, Hawley RG. Performance- and safety-enhanced lentiviral vectors containing the human interferon-beta scaffold attachment region and the chicken beta-globin insulator. Blood 2003; 101: 4717-4724.
15. Aiuti A et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410-2413.
16. Wu T 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: 285-293.
17. Horn PA et al. Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons. Gene Therapy 2002; 9: 1464-1471.
18. Ragg S et al. Direct reversal of DNA damage by mutant methyltransferase protein protects mice against dose-intensified chemotherapy and leads to in vivo selection of hematopoietic stem cells. Cancer Res 2000; 60: 5187-5195.
19. 6benzylguanine and 1,3-bis(2-chloroethyl)-1-nitrosourea. Cancer Res 1997; 57: 5093-5099.
20. 6-methylguanine DNA methyltransferase-transduced marrow progenitors repopulate nonmyeloablated mice after drug selection. Blood 2000; 95: 3078-3084.
21. 6-benzylguanine. Biochemistry 1993; 32: 11998-12006.
22. 6-methylguanine-DNA methyltransferase mutants: implications for drug resistance gene therapy. Hum Gene Ther 1999; 10: 2769-2778.
23. Neff T et al. Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J Clin Invest 2003; 112: 1581-1588.
24. Zielske SP et al. In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning. J Clin Invest 2003; 112 1561-1570.
25. Pollok KE et al. In vivo selection of human hematopoietic cells in a xenograft model using combined pharmacologic and genetic manipulations. Hum Gene Ther 2003; 14: 1703-1714.
26. Persons DA et al. Successful treatment of murine {beta}-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood 2003; 102: 506-513.
27. Tutois et al. Erythropoietic protoporphyria in the house mouse. A recessive inherited ferrochelatase deficiency with anemia, photosensitivity, and liver disease. J Clin Invest 1991; 88: 1730-1736.
28. Sassa S. Hematological aspects of the porphyrias. Int J Hematol 2000; 1: 1-17.
29. Gouya L et al. The penetrance of dominant erythropoietic protoporphyria is modulated by expression of wildtype FECH. Nat Genet 2002; 30: 27-28.
30. Fontanellas A et al. Successful therapeutic effect in a mouse model of erythropoietic protoporphyria by partial genetic correction and fluorescence-based selection of hematopoietic cells. Gene Therapy 2001; 8: 618-626.
31. Pawliuk R et al. Long-term cure of the photosensitivity of murine erythropoietic protoporphyria by preselective gene therapy. Nat Med 1999; 7: 768-773.
32. Richard E et al. Gene therapy of a mouse model of protoporphyria with a self-inactivating erythroid-specific lentiviral vector without preselection. Mol Ther 2001; 4: 331-338.
33. Richard E et al. A bicistronic SIN-lentiviral vector containing G156A MGMT allows selection and metabolic correction of hematopoietic protoporphyric cell lines. J Gen Med 2003; 5: 737-747.
34. Halene S et al. Improved expression in hematopoietic and lymphoid cells in mice after transplantation of bone marrow transduced with a modified retroviral vector. Blood 1999; 94: 3349-3357.
35. Onodera M et al. Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency. Blood 1998; 91: 30-36.
36. Doerflinger N et al. Retroviral transfer and long-term expression of the adrenoleukodystrophy gene in human CD34+ cells. Hum Gene Ther 1998; 9: 1025-1036.
37. Dunbar CE, Kohn DB, Schiffmann R. Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation. Hum Gene Ther 1998; 9: 2629-2640.
38. Malech HL et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci USA 1997; 94: 12133-12138.
39. Davis BM, Humeau L, Dropulic B. In vivo selection for human and murine hematopoietic cells transduced with a therapeutic MGMT lentiviral vector that inhibits HIV replication. Mol Ther 2004; 9: 160-172.
40. Puig T et al. Myeloablation enhances engraftment of transduced murine hematopoietic cells, but does not influence long-term expression of the transgene. Gene Therapy 2002; 9: 1472-1479.
41. Chen ZP et al. Enhanced antitumor activity of sarCNU in comparison to BCNU in an extraneuronal monoamine transporter positive human glioma xenograft model. J Neurooncol 1999; 44: 7-14.
42. Zielske SP, Gerson SL. SarCNU mediates selection of P140K methylguanine-DNA-methyltransferase transduced human CD34(+) cells in vitro. Blood Cells Mol Dis 2003; 31: 48-50.
43. 6-benzylguanine, and an alkyltransferase-expressing retroviral vector. Mol Ther 2001; 1 78-87.
44. Moreau-Gaudry F et al. High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors. Blood 2001; 98: 2664-2672.
45. Camadro JM, Labbe PA. A simple ferrochelatase assay. Biochimie 1981; 63: 463-468.