Sustained transgene expression by human cord blood derived CD34+ cells transduced with simian immunodeficiency virus agmTYO1-based vectors carrying the human coagulation factor VIII gene in NOD/SCID mice

Journal of Gene Medicine

Volumn 6, Issue 10, 2004, Pages 1049-1060

  Kikuchi, Jiro ^a   Mimuro, Jun ^a   Ogata, Kyoichi ^a   Tabata, Toshiaki ^b   Ueda, Yasuji ^b   Ishiwata, Akira ^a   Kimura, Konzoh ^a   Takano, Katsuhiro ^a   Madoiwa, Seiji ^a   Mizukami, Hiroaki ^a   Hanazono, Yutaka ^a   Kume, Akihiro ^a   Hasegawa, Mamoru ^b   Ozawa, Keiya ^a   Sakata, Yoichi ^a  

^a JICHI MEDICAL UNIVERSITY   (Japan)

^b DNAVEC Research Inc   (Japan)

Author keywords

Gene therapy; Hematopoietic stem cell; Hemophilia; Simian immunodeficiency virus

Indexed keywords

BLOOD CLOTTING FACTOR 8; CD34 ANTIGEN; COMPLEMENTARY DNA; ENHANCED GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIGEN DETECTION; ARTICLE; AUTOLOGOUS BONE MARROW TRANSPLANTATION; BONE MARROW CELL; CONTROLLED STUDY; CORD BLOOD STEM CELL TRANSPLANTATION; DNA MODIFICATION; GENE EXPRESSION; GENE THERAPY; GENETIC ENGINEERING; GENETIC TRANSCRIPTION; GENETIC TRANSDUCTION; HEMOPHILIA A; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; INCUBATION TIME; MOUSE; MOUSE STRAIN; NONHUMAN; PRIORITY JOURNAL; RISK ASSESSMENT; SCID MOUSE; SIMIAN IMMUNODEFICIENCY VIRUS; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS GENOME; VIRUS INFECTION;

ANIMALS; ANTIGENS, CD34; BONE MARROW CELLS; DNA, COMPLEMENTARY; DOSE-RESPONSE RELATIONSHIP, DRUG; ENZYME-LINKED IMMUNOSORBENT ASSAY; FACTOR VIII; FETAL BLOOD; FLOW CYTOMETRY; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELLS; HUMANS; MICE; MICE, INBRED NOD; MICE, SCID; MICROSCOPY, FLUORESCENCE; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; SIMIAN IMMUNODEFICIENCY VIRUS; TIME FACTORS; TRANSGENES;

MURINAE; SIMIAE; SIMIAN IMMUNODEFICIENCY VIRUS;

EID: 13644262651     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.609     Document Type: Article

References (35)

1. Hoyer LW. Hemophilia A. N Engl J Med 1994; 330: 38-47.
2. Kay MA, High K. Gene therapy for the hemophilias. Proc Natl Acad Sci U S A 1999; 96: 9973-9975.
3. Kume A, Hanazono Y, Mizukami H, Urabe M, Ozawa K. Hematopoietic stem cell gene therapy: a current overview. Int J Hematol 1999; 68: 227-233.
4. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vector. Science 1999; 283: 682-686.
5. Woods NB, Fahlman C, Mikkola H, et al. Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells. Blood 2000; 96: 3725-3733.
6. + NOD /SCID-repopulating cells. Blood 2002; 99: 709-712.
7. Naldini L, Blomer U, Gallay P, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
8. Honjo S, Narita T, Kobayashi R, et al. Experimental infection of African green monkeys and cynomolgus monkeys with a SIVAGM strain isolated from a healthy African green monkey. J Med Primatol 1990; 19: 9-20.
9. Nakajima T, Nakamaru K, Ido E, Terao K, Hayami M, Hasegawa M. Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system. Hum Gene Ther 2000; 11: 1863-1874.
10. Hoeben RC, Einerhand MP, Briet E, van Ormondt H, Valerio D, van der Eb AJ. Toward gene therapy in haemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost 1992; 67: 341-345.
11. Tonn T, Herder C, Becker S, Seifried E, Grez M. Generation and characterization of human hematopoietic cell lines expressing factor VIII. J Hematother Stem Cell Res 2002; 11: 695-704.
12. Kootstra NA, Matsumura R, Verma IM. Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther 2003; 7: 623-631.
13. Tiede A, Eder M, von Depka M, et al. Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction. Gene Ther 2003; 10: 1917-1925.
14. Ogata K, Mimuro J, Kikuchi J, et al. Expression of human coagulation factor VIII in adipocytes transduced with the simian immunodeficiency virus agmTYO1-based vector for haemophilia A gene therapy. Gene Ther 2004; 11: 253-259.
15. Furukawa Y, Kikuchi J, Nakamura M, Iwase S, Yamada H, Matsuda M. Lineage-specific regulation of cell cycle control gene expression during haematopoietic cell differentiation. Br J Haematol 2000; 110: 663-673.
16. Ueda T, Tsuji K, Yoshino H, et al. Expansion of human NOD/SCID-repopulating cells by stem cell factor, Flk2/Flt3 ligand, thrombopoietin, IL-6, and soluble IL-6 receptor. J Clin Invest 2000; 105: 1013-1021.
17. Yoshino H, Ueda T, Kawahata M, et al. Natural killer cell depletion by anti-asialo GM1 antiserum treatment enhances human hematopoietic stem cell engraftment in NOD/Shi-scid mice. Bone Marrow Transplant 2000; 26: 1211-1216.
18. Ma F, Wada M, Yoshino H, et al. Development of human lymphohematopoietic stem and progenitor cells defined by expression of CD34 and CD81. Blood 2001; 97: 3755-3762.
19. Yonemura H, Sugawara K, Nakashima K, Nakahara Y, Hamamoto T, Mimaki I. Production of recombinant human factor VIII by co-expression of the heavy and light chains. Protein Eng 1993; 6: 669-674.
20. Chomczynski P, Sacchi N. Single-step method of RNA isolation by acid guanidinium thiocyanate-phenol-chloroform extraction. Anal Biochem 1987; 162: 156-159.
21. Sutton RE, Reitsma MJ, Uchida N, Brown PO. Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent. J Virol 1999; 73 3649-3660.
22. Xu R, Reems JA. Umbilical cord blood progeny cells that retain a CD34+ phenotype after ex vivo expansion have less engraftment potential than unexpanded CD34+ cells. Transfusion 2001; 41: 213-218.
23. VandenDriessche T, Vanslembrouck V, Goovaerts I, et al. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc Natl Acad Sci U S A 1999; 96: 10379-10384.
24. Hoeben RC, Fallaux FJ, Van Tilburg NH, et al. Toward gene therapy for hemophilia A: long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice. Hum Gene Ther 1993; 4: 179-186.
25. Dwarki VJ, Belloni P, Nijjar T, et al. Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci U S A 1995; 92: 1023-1027.
26. Chuah MK, Van Damme A, Zwinnen H, et al. Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice. Hum Gene Ther 2000; 11: 729-738.
27. Naldini L, Verma IM. Lentiviral vectors. Adv Virus Res 2000; 55: 599-609.
28. Chao H, Walsh CE. Induction of tolerance to human factor VIII in mice. Blood 2001; 97: 3311-3312.
29. Roth DA, Tawa NE Jr, Proper JA, et al. The factor VIII transkaryotic therapy study group. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 2001; 344: 1782-1784.
30. Powell J, Ragni MV, White GC, et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood 2003; 102 2038-2045.
31. Zennou V, Petit C, Guetard D, Nerhbass U, Montagnier L, Charneau P. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 2000; 101: 173-185.
32. Sirven A, Pflumio F, Zennou V, et al. The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells. Blood 2000; 96: 4103-4110.
33. Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 28: 2410-2413.
34. 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.
35. Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348: 255-256.