Co-expression of MGMTP140K and α-L-iduronidase in primary hepatocytes from mucopolysaccharidosis type I mice enables efficient selection with metabolic correction

Journal of Gene Medicine

Volumn 10, Issue 3, 2008, Pages 249-259

  Wang, Daren ^a   Worsham, D Nicole ^a   Pan, Dao ^a,b  

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

^b UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

Author keywords

Ex vivo selection; Lentivirus vector; Lysosomal storage diseases; Metabolic cross correction; Methylguanine DNA methyltransferase; Primary hepatocytes

Indexed keywords

6 O BENZYLGUANINE; CARMUSTINE; ELONGATION FACTOR 1ALPHA; LENTIVIRUS VECTOR; LEVO IDURONIDASE; METHYLATED DNA PROTEIN CYSTEINE METHYLTRANSFERASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL SELECTION; CONCENTRATION RESPONSE; CONTROLLED STUDY; CORRELATION ANALYSIS; EX VIVO STUDY; FEASIBILITY STUDY; GENE EXPRESSION; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; HURLER SYNDROME; IN VITRO STUDY; INTERNAL RIBOSOME ENTRY SITE; LIVER CELL; LONG TERMINAL REPEAT; METABOLIC REGULATION; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROMOTER REGION; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; CELLS, CULTURED; DNA MODIFICATION METHYLASES; DNA REPAIR ENZYMES; FLOW CYTOMETRY; HEMATOPOIETIC STEM CELLS; HEPATOCYTES; IDURONIDASE; MICE; MUCOPOLYSACCHARIDOSIS I; TRANSGENES; TUMOR SUPPRESSOR PROTEINS;

ANIMALIA; LENTIVIRUS; MUS; SPLEEN FOCUS-FORMING VIRUS;

EID: 40549117882     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1141     Document Type: Article

References (41)

1. Sands MS, Davidson BL. Gene therapy for lysosomal storage diseases. Mol Ther 2006; 13: 839-849.
2. Hopwood JJ, Morris CP. The mucopolysaccharidoses. Diagnosis, molecular genetics and treatment. Mol Biol Med 1990; 7: 381-404.
3. Neufeld EF. Lysosomal storage diseases. Annu Rev Biochem 1991; 60: 257-280.
4. Souillet G, Guffon N, Maire I, et al. Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources. Bone Marrow Transplant 2003; 31: 1105-1117.
5. Staba SL, Escolar ML, Poe M, et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004; 350: 1960-1969.
6. Grewal S, Shapiro E, Braunlin E, et al. Continued neurocognitive development and prevention of cardiopulmonary complications after successful BMT for I-cell disease: a long-term follow-up report. Bone Marrow Transplant 2003; 32: 957-960.
7. Peters C, Balthazor M, Shapiro EG, et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 1996; 87: 4894-4902.
8. Biffi A, Capotondo A, Fasano S, et al. Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest 2006; 116: 3070-3082.
9. Hacein-Bey-Abina S, Le Deist F, Carlier F, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002; 346: 1185-1193.
10. Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410-2413.
11. 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.
12. Zielske SP, Gerson SL. 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 2002; 5: 381-387.
13. Sawai N, Zhou S, Vanin EF, Houghton P, Brent TP, Sorrentino BP. Protection and in vivo selection of hematopoietic stem cells using temozolomide, O6-benzylguanine, and an alkyltransferase-expressing retroviral vector. Mol Ther 2001; 3: 78-87.
14. Cai S, Hartwell JR, Cooper RJ, et al. In vivo effects of myeloablative alkylator therapy on survival and differentiation of MGMTP140K-transduced human G-CSF-mobilized peripheral blood cells. Mol Ther 2006; 13: 1016-1026.
15. 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: 1703-1714.
16. 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.
17. Ragg S, Xu-Welliver M, Bailey J, 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.
18. Persons DA, Allay ER, Sawai N, 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.
19. Neff T, Horn PA, Peterson Li. et al. Methylguanine methyltrans-ferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J Clin Invest 2003; 112: 1581-1588.
20. Neff T, Beard BC, Peterson LJ, Anandakumar P, Thompson J, Kiem HP. Polyclonal chemoprotection against temozolomide in a large-animal model of drug resistance gene therapy. Blood 2005; 105: 997-1002.
21. Pan D, Gunther R, Duan W, et al. Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow. Mol Ther 2002; 6: 19-29.
22. Follenzi A, Sabatino G, Lombardo A. Boccaccio C, Naldini L. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum Gene Ther 2002; 13: 243-260.
23. Park F, Ohashi K, Kay MA. The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo. Mol Ther 2003; 8: 314-323.
24. Worsham DN, Schuesler T, von Kalle C, Pan D. In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vector. Mol Ther 2006; 14: 514-524.
25. Baum C, Hunt N, Hildinger M, et al. cis-Active elements of Friend spleen focus-forming virus: from disease induction to disease prevention. Acta Haematol 1998; 99: 156-164.
26. Pan D, Aronovich E, McIvor RS, Whitley CB. Retroviral vector design studies toward hematopoietic stem cell gene therapy for mucopolysaccharidosis type I. Gene Ther 2000; 7: 1875-1883.
27. Weber-Benarous A, Decaux JF, Bennoun M, Allemand I, Briand P, Kahn A. Retroviral infection of primary hepatocytes from normal mice and mice transgenic for SV40 large T antigen. Exp Cell Res 1993; 205: 91-100.
28. Petersen BE, Bowen WC, Patrene KD, et al. Bone marrow as a potential source of hepatic oval cells. Science 1999; 284: 1168-1170.
29. Oertel M, Rosencrantz B, Chen YQ, et al. Repopulation of rat liver by fetal hepatoblasts and adult hepatocytes transduced ex vivo with lentiviral vectors. Hepatology 2003; 37: 994-1005.
30. Nguyen TH, Oberholzer J, Birraux J, Majno P, Morel P, Trono D. Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes. Mol Ther 2002; 6: 199-209.
31. Baum C, Dullmann J, Li Z, et al. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 2003; 101: 2099-2114.
32. Modlich U, Bohne J, Schmidt M, et al. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 2006; 108: 2545-2553.
33. Mitra S, Kaina. B. Regulation of repair of alkylation damage in mammalian genomes. Prog Nucleic Acid Res Mol Biol 1993; 44: 109-142.
34. Mingozzi F, Liu YL, Dobrzynski E, et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepaticgene transfer. J Clin Invest 2003; 111: 1347-1356.
35. Moritz T, Mackay W, Glassner BJ, Williams DA, Samson L. Retrovirus-mediated expression of a DNA repair protein in bone marrow protects hematopoietic cells from nitrosourea-induced toxicity in vitro and in vivo. Cancer Res 1995; 55: 2608-2614.
36. Maze R, Carney JP, Kelley MR, Glassner BJ, Williams DA, Samson L. Increasing DNA repair methyltransferase levels via bone marrow stem cell transduction rescues mice from the toxic effects of 1,3-bis(2-cloroethyl)-1-nitrosourea, a chemotherapeutic alkylating agent. Proc Natl Acad Sci USA 1996; 93: 206-210.
37. Demaison C, Parsley K, Brouns G, et al. High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum Gene Ther 2002; 13: 803-813.
38. Hammond SM, Crable SC, Anderson KP. Negative regulatory elements are present in the human LM02 oncogene and may contribute to its expression in leukemia. Leuk Res 2005; 29: 89-97.
39. Forbes SJ, Themis M, Alison MR, et al. Tri-iodothyronine and a deleted form of hepatocyte growth factor act synergistically to enhance liver proliferation and enable in vivo retroviral gene transfer via the peripheral venous system. Gene Ther 2000; 7: 784-789.
40. Minami H, Tada K, Chowdhury NR, Chowdhury JR, Onji M. Enhancement of retrovirus-mediated gene transfer to rat liver in vivo by infusion of hepatocyte growth factor and triiodothyronine. J Hepatol 2000; 33: 183-188.
41. Patijn GA, Lieber A, Schowalter DB, Schwall R, Kay MA. Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice. Hepatology 1998; 28: 707-716.