Gene therapy for hemophiliacs - Therapeutic possibilities and technological limits;Therapie genique des hemophilies - Potentialites therapeutiques et limitations technologiques

Transfusion Clinique et Biologique

Volumn 4, Issue 3, 1997, Pages 251-261

  Michou, A I ^a   Christ, M ^a   Pavirani, A ^a   Mehtali, M ^a  

^a TRANSGENE S A   (France)

Author keywords

adenovirus vectors; gene therapy; hemophilia A and B

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG; VIRUS VECTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9;

ADENOVIRUS; CONFERENCE PAPER; GENE THERAPY; HEMOPHILIA; HUMAN; ANIMAL; C57BL MOUSE; CHEMICAL STRUCTURE; GENE TRANSFER; GENE VECTOR; GENETICS; HEMOPHILIA A; METHODOLOGY; MOUSE; REVIEW;

ADENOVIRIDAE; ANIMALS; FACTOR IX; FACTOR VIII; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMOPHILIA A; HUMANS; MICE; MICE, INBRED C57BL; MODELS, MOLECULAR;

EID: 0030917365     PISSN: 12467820     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1246-7820(97)80049-3     Document Type: Conference Paper

References (25)

1. Anderson WF. Human gene therapy. Science 1992; 256: 808-813.
2. Roe T, Reynolds TC, Yu G, Brown PO. Integration of murine leukemia virus DNA depends on mitosis. EMBO J 1993; 12: 2099-2108.
3. Fallaux FJ, Hoeben RC, Cramer SJ, van den Wollenberg DJM, Briet E, van Ormondt H, van der Eb AJ. The human clotting factor VIII cDNA contains an autonomously replicating sequence consensus and matrix attachment region-like sequence that binds a nuclear factor, represses heterologous gene expression and mediates the transcriptionnal effects of sodium butyrate. Mol Cel Biol 1996; 16: 4264-4272.
4. Palmer TD, Thompson AR, Miller AD. Production of human factor IX in animals by genetically modified skin fibroblasts: potential gene therapy for hemophilia B. Blood 1989; 73: 438-445.
5. Yao SN, Smith KJ, Kurachi K. Primary myoblast-mediated gene transfer: persistent expression of human factor IX in mice. Gene Ther 1994; 1: 99-107.
6. Gerrard AJ, Hudson DL, Brownlee GG, Watt FM. Towards gene therapy for hemophilia B using primary human keratinocytes. Nat Genet 1993; 3: 180-183.
7. Lu DR, Zhou JM, Zheng B, Qiu X., Xue JL, Wang JM et al. Stage I clinical trial of gene therapy for Hemophilia B. Sci China 1993; 36: 1342-1351.
8. Gordon EM, Anderson WF. Gene therapy using retroviral vectors. Curr Opin Biotechnol 1994; 5: 611-616.
9. Rosenthal FM, Zier KS, Gansbacher B. Human tumor vaccines and genetic engineering of tumors with cytokine and histocompatibility genes to enhance immunogenicity. Curr Opin Oncol 1994; 6: 611-615.
10. Horwitz MS. Adenoviridae and their replication. In: Fields BN, ed. Virology, Raven Press Ltd, New York. 1990: 1679-1721.
11. Chroboczek J, Ruigrok R, Cusack S. Adenovirus fiber. In: Doerfler W and Boehm P, ed. The molecular repertoire of adenoviruses I. Springer Verlag Berlin. 1995: 163-200.
12. Graham FL, Smiley J, Russell WC, Nairn R. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J Gen Virol 1977; 36: 59-72.
13. Trapnell BC, Gorziglia M. Gene therapy using adenoviral vectors. Curr Opin Biotechnol 1994; 5: 617-625.
14. Crystal RG, McElvaney NG, Rosenfeld MA, Chu CS, Mastrangeli A, Hay JG et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tracts of individuals with cystic fibrosis. Nat Genet 1994; 8: 42-51.
15. Zabner J, Petersen DM, Puga AP, Graham SM, Couture LA, Keyes LD et al. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 1993; 75: 207-216.
16. Kass-Eisler A, Falck-Pedersen E, Elfenbein DH, Alvira M, Buthick PM, Leinwand LA. The impact of developmental stage, route of administration and the immune system on adenovirus mediated gene transfer. Gene Ther 1994; 1: 395-402.
17. Yang Y, Ertl HCJ, Wilson JM, MHC class I restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with El-deleted recombinant adenoviruses. Immunity 1994; 1: 433-442.
18. Yang Y, Il Q, Ertl HJC, Wilson JM. Cellular and humoral immune response to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 1995; 69: 2004-2015.
19. Yang Y, Jooss KU, Su Q, Ertl HJC, Wilson JM. Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo. Gene Ther 1996; 3: 137-144.
20. Yang Y, Haecker SE, Su Q, Wilson JM. Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle. Hum Mol Genet 1196; 5: 1703-1712.
21. Barr D, Tubb J, Ferguson D, Scaria A, Lieber A, Wilson C et al. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparison between immunocompetent and immunodeficient inbred strains. Gene Ther 1995; 2: 151-155.
22. Tripathy SK, Black HB, Goldwasser E, Leiden JM. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nat Med 1996; 2: 545-550.
23. Yang Y, Nunes FA, Berencsi K, Furth EA, Gonczol E, Wilson JM. Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc Natl Acad Sci (USA) 1994; 91: 4407-4411.
24. Evans JP, Brinkhous KM, Brayer GD, Reisner HM, High KA. Canine hemophilia B resulting from a point mutation with unusual consequences. Proc Natl Acad Sci (USA) 1989; 86: 10095-10099.
25. Guo ZS, Wang LH, Eisensmith RC, Woo SLC. Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer. Gene Ther 1996; 3: 802-810.