Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells

Molecular Therapy

Volumn 13, Issue 2, 2006, Pages 391-400

  Schambach, Axel ^a   Bohne, Jens ^a   Chandra, Saurabh ^b   Will, Elke ^b   Margison, Geoffrey P ^c   Williams, David A ^b   Baum, Christopher ^a,b  

^a HANNOVER MEDICAL SCHOOL   (Germany)

^b CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^c IMPERIAL CANCER RESEARCH FUND   (United Kingdom)

Author keywords

Cancer; Hematopoiesis; Human immunodeficiency virus; Murine leukemia virus; Retrovirus

Indexed keywords

6 O BENZYLGUANINE; ALKYLATING AGENT; CARMUSTINE; CD34 ANTIGEN; LENTIVIRUS VECTOR; METHYLATED DNA PROTEIN CYSTEINE METHYLTRANSFERASE; MUTANT PROTEIN; RETROVIRUS VECTOR;

ANIMAL CELL; ARTICLE; CELL SURVIVAL; CONTROLLED STUDY; ENHANCER REGION; GENE CASSETTE; GENE INSERTION; GENETIC TRANSDUCTION; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; LENTIVIRINAE; LONG TERMINAL REPEAT; MOUSE; NONHUMAN; PROMOTER REGION; PROTEIN EXPRESSION; RETROVIRUS; SELF INACTIVATING VECTOR; TRANSCRIPTION INITIATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS INACTIVATION;

ANIMALS; CELLS, CULTURED; GENE EXPRESSION REGULATION, VIRAL; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS; LENTIVIRUS; LEUKEMIA VIRUS, MURINE; MICE; MICE, INBRED C57BL; MUTAGENESIS, INSERTIONAL; O(6)-METHYLGUANINE-DNA METHYLTRANSFERASE; RNA PROCESSING, POST-TRANSCRIPTIONAL; TRANSDUCTION, GENETIC;

ANIMALIA; HUMAN IMMUNODEFICIENCY VIRUS; LENTIVIRUS; MURINE LEUKEMIA VIRUS;

EID: 30344464717     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.08.012     Document Type: Article

References (47)

1. Z. Li et al. Murine leukemia induced by retroviral gene marking Science 296 2002 497
2. S. Hacein-Bey-Abina et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302 2003 415-419
3. U. Modlich et al. Leukemias following retroviral transfer of multidrug resistance 1 are driven by combinatorial insertional mutagenesis Blood 105 2005 4235-4246
4. O.S. Kustikova et al. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking Science 308 2005 1171-1174
5. C. Baum et al. Side effects of retroviral gene transfer into hematopoietic stem cells Blood 101 2003 2099-2114
6. S.F. Yu et al. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells Proc. Natl. Acad. Sci. USA 83 1986 3194-3198
7. A. Wahlers et al. Influence of multiplicity of infection and protein stability on retroviral vector-mediated gene expression in hematopoietic cells Gene Ther. 8 2001 477-486
8. N.B. Woods et al. Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: Risk of insertional mutagenesis Blood 101 2003 1284-1289
9. S.L. Gerson MGMT: Its role in cancer aetiology and cancer therapeutics Nat. Rev. Cancer 4 2004 296-307
10. M.D. Milsom and L.J. Fairbairn Protection and selection for gene therapy in the hematopoietic system J. Gene Med. 6 2004 133-146
11. T. Moritz D.A. Williams Methods for gene transfer: Genetic manipulation of hematopoietic stem cells in: K.G. Blume S.J. Forman F.R. Appelbaum (Eds) Thomas' Hematopoietic Cell Transplantation 3rd ed. 2003 Blackwell Sci Malden, MA 107-117
12. S. Ragg 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. 60 2000 5187-5195
13. S.P. Zielske J.S. Reese K.T. Lingas J.R. Donze and S.L. Gerson In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning J. Clin. Invest. 112 2003 1561-1570
14. T. Neff et al. Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model J. Clin. Invest. 112 2003 1581-1588
15. T. Neff et al. Polyclonal chemoprotection against temozolomide in a large animal model of drug resistance gene therapy Blood 105 2005 997-1002
16. E. Richard et al. Hematopoietic stem cell gene therapy of murine protoporphyria by methylguanine-DNA-methyltransferase-mediated in vivo drug selection Gene Ther. 11 2004 1638-1647
17. D.A. Persons et al. Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells Blood 102 2003 506-513
18. J.E. Bowman J.S. Reese K.T. Lingas and S.L. Gerson Myeloablation is not required to select and maintain expression of the drug-resistance gene, mutant MGMT, in primary and secondary recipients Mol. Ther. 8 2003 42-50
19. 6-benzylguanine Cancer Res. 54 1994 6221-6227
20. 6-methylguanine-DNA methyltransferase mutants: implications for drug resistance gene therapy Hum. Gene Ther. 10 1999 2769-2778
21. M. Jansen et al. Protection of hematopoietic cells from O(6)-alkylation damage by O(6)-methylguanine DNA methyltransferase gene transfer: studies with different O(6)-alkylating agents and retroviral backbones Eur. J. Haematol. 67 2001 2-13
22. S.P. Zielske and S.L. Gerson Lentiviral transduction of P140K MGMT into human CD34(+) hematopoietic progenitors at low multiplicity of infection confers significant resistance to BG/BCNU and allows selection in vitro Mol. Ther. 5 2002 381-387
23. 6-benzylguanine and 1,3-bis(2-chloroethyl)-nitrosourea or temozolomide Hum. Gene Ther. 15 2004 758-769
24. T. Dull et al. A third-generation lentivirus vector with a conditional packaging system J. Virol. 72 1998 8463-8471
25. C. Baum S. Hegewisch-Becker H.G. Eckert C. Stocking and W. Ostertag Novel retroviral vectors for efficient expression of the multidrug-resistance (mdr-1) gene in early hemopoietic cells J. Virol. 69 1995 7541-7547
26. B. Lim D.A. Williams and S.H. Orkin Retroviral-mediated gene transfer of human adenosine deaminase: Expression of functional enzyme in murine hematopoietic stem cells in vivo Mol. Cell. Biol. 7 1987 3459-3465
27. P. Salmon et al. High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors Blood 96 2000 3392-3398
28. N.B. Woods et al. Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells Blood 96 2000 3725-3733
29. N.B. Woods A. Ooka and S. Karlsson Development of gene therapy for hematopoietic stem cells using lentiviral vectors Leukemia 16 2002 563-569
30. + cells derived from human and nonhuman primates Blood 100 2002 823-832
31. R. Zufferey J.E. Donello D. Trono and Z.J. Hope Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors J. Virol. 73 1999 2886-2892
32. M. Egelhofer et al. Inhibition of HIV-1 entry in cells expressing Gp41-derived peptides J. Virol. 78 2004 568-575
33. A. Schambach et al. Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors Mol. Ther. 2 2000 435-445
34. A. Follenzi L.E. Ailles S. Bakovic M. Geuna and L. Naldini Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences Nat. Genet. 25 2000 217-222
35. J. Kraunus et al. Self-inactivating retroviral vectors with improved RNA processing Gene Ther. 11 2004 1568-1578
36. H. Mikkola et al. Lentivirus gene transfer in murine hematopoietic progenitor cells is compromised by a delay in proviral integration and results in transduction mosaicism and heterogeneous gene expression in progeny cells J. Virol. 74 2000 11911-11918
37. N.B. Woods et al. Lentiviral-mediated gene transfer into haematopoietic stem cells J. Intern. Med. 249 2001 339-343
38. Z. Li et al. Predictable and efficient retroviral gene transfer into murine bone marrow repopulating cells using a defined vector dose Exp. Hematol. 31 2003 1206-1214
39. H. Hanawa et al. Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood Mol. Ther. 5 2002 242-251
40. S.P. Zielske and S.L. Gerson Limited lentiviral transgene expression with increasing copy number in an MGMT selection model: Lack of copy number selection by drug treatment Mol. Ther. 9 2004 923-931
41. L. Liu et al. Differential degradation rates of inactivated alkyltransferase in blood mononuclear cells and tumors of patients after treatment with O(6)-benzylguanine Clin. Cancer Res. 7 2001 2318-2324
42. + cells using amphotropic-, GALV-, or RD114-pseudotyped HIV-1-based vectors from stable producer cells Mol. Ther. 11 2005 452-459
43. E. Richard et al. A bicistronic SIN-lentiviral vector containing G156A MGMT allows selection and metabolic correction of hematopoietic protoporphyric cell lines J. Gene Med. 5 2003 737-747
44. K.E. Pollok et al. In vivo selection of human hematopoietic cells in a xenograft model using combined pharmacologic and genetic manipulations Hum. Gene Ther. 14 2003 1703-1714
45. X. Wu Y. Li B. Crise and S.M. Burgess Transcription start regions in the human genome are favored targets for MLV integration Science 300 2003 1749-1751
46. F.D. Bushman Targeting survival: Integration site selection by retroviruses and LTR-retrotransposons Cell 115 2003 135-138
47. 6-Alkylguanine-DNA alkyltransferase assay Methods Mol. Biol. 152 2000 49-61