Lack of persistence of E1- recombinant adenoviral vectors containing a temperature-sensitive E2A mutation in immunocompetent mice and hemophilia B dogs

Gene Therapy

Volumn 3, Issue 3, 1996, Pages 217-222

  Fang, B ^a   Wang, H ^a   Gordon, G ^b   Bellinger, D A ^b   Read, M S ^b   Brinkhous, K M ^b   Woo, S L C ^a   Eisensmith, R C ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

^b UNIVERSITY OF NORTH CAROLINA   (United States)

Author keywords

Adenoviral vector; Alpha1 antitrypsin; Factor IX; Gene therapy; Hemophilia B; Temperature sensitive

Indexed keywords

ALPHA 1 ANTITRYPSIN; BLOOD CLOTTING FACTOR 9; DNA BINDING PROTEIN; VIRUS PROTEIN;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DOG; EXPRESSION VECTOR; GENE EXPRESSION; GENE MUTATION; GENE TRANSFER; HEMOPHILIA B; HUMORAL IMMUNITY; MOUSE; NONHUMAN; PRIORITY JOURNAL; TEMPERATURE SENSITIVE MUTANT; TRANSGENE; VIRUS RECOMBINANT;

ADENOVIRIDAE; ADENOVIRUS E1 PROTEINS; ADENOVIRUS E2 PROTEINS; ALPHA 1-ANTITRYPSIN; ANIMALS; BASE SEQUENCE; DNA PRIMERS; DOGS; FACTOR IX; GENETIC VECTORS; HEMOPHILIA A; HUMANS; IMMUNOCOMPETENCE; MICE; MICE, INBRED BALB C; MOLECULAR SEQUENCE DATA; MUTATION; RECOMBINATION, GENETIC; TRANSGENES;

EID: 0029670374     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (33)

1. Kay MA, Woo SLC. Gene therapy for metabolic disorders. Trends Genet 1994; 10: 253-257.
2. Englehardt JF, Litzky L, Wilson JM. Prolonged transgene expression in Cotton rat lung with recombinant adenoviruses defective in E2a. Hum Gene Ther 1994; 5: 1217-1229.
3. Englehardt JF, Ye X, Doranz B, Wilson JM. Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc Natl Acad Sci USA 1994; 91: 6196-6200.
4. Yang Y et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 1994; 91: 4407-4411.
5. Yang Y et al. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat Genet 1994; 7: 362-369.
6. Fang B et al. Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression. Hum Gene Ther 1995; 6: 1039-1044.
7. Ensinger MJ, Ginsberg HS. Selection and preliminary characterization of temperature sensitive mutants of type 5 adenovirus. J Virol 1972; 10: 328-339.
8. Kay MA, Graham F, Leland F, Woo SLC. Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology 1995; 21: 815-819.
9. Kay MA et al. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc Natl Acad Sci USA 1994; 91: 2353-2357.
10. Stratford-Perricaudet LD, Makeh I, Perricaudet M, Briand P. Widespread long-term gene transfer to mouse skeletal muscles and heart. J Clin Invest 1992; 90: 626-630.
11. Stratford-Perricaudet LD et al. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum Gene Ther 1990; 1: 214-256.
12. Fang B et al. Gene therapy for phenylketonuria: phenotypic correction in a genetically deficient mousse model by adenovirus-mediated hepatic gene transfer. Gene Ther 1994: 1; 247-254.
13. Shenk T, Jones N, Colby W, Fowlkes D. Functional analysis of adenovirus-5 host-range deletion mutants defective for transformation of rat embryo cells. Cold Spring Harbor Symp Quant Biol 1980; 44: 367-375.
14. Imperiale MJ et al. Common control of the heat shock gene and early adenovirus gene: evidence for a cellular E1a-like activity. Mol Cell Biol 1984; 4: 867-874.
15. Thangue NBL, Rigby PWJ. An adenovirus E1A like transcription factor is regulated during the differentiation of murine embryonal carcinoma stem cells. Cell 1987; 49: 507-513.
16. Onclercq R, Gilardi P, Lavenu A, Cremisi C. C-myc products trans-activate the adenovirus E4 promoter in EC stem cells by using the same target sequence as E1A products. J Virol 1988; 62: 4533-4537.
17. Freidel BR, Krevolin MD, Horwitz MS. Effects of the adenovirus H5ts125 and H5ts107 DNA binding proteins on DNA replication in vitro. Virology 1983; 124: 380-389.
18. Barr D et al. Strain-related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther 1995; 2: 151-155.
19. Carter TH, Blanton RA. Autoregulation of adenovirus type 5 early gene expression II. Effect of temperature-sensitive early mutations on virus RNA accumulation. J Virol 1978; 28: 450-156.
20. Nevins JR, Jensen-Winkler JJ. Regulation of early adenovirus transcription: a protein product of early region 2 specifically represses region 4 transcription. Proc Natl Acad Sci USA 1980; 77: 1893-1897.
21. Rice SA, Klessig DF. Isolation and analysis of adenovirus type 5 mutants containing deletions in the gene encoding the DNA-binding protein. J Virol 1985; 56: 767-778.
22. Williams KP, Smith JA. Processing and presentation of viral antigens. In: Van Regenmortel MHV, Neurath AR (eds). Immunochemistry of Virus II: The Basis for Serodiagnosis and Vaccines. Elsevier: Amsterdam, 1990, pp 39-51.
23. Germain RN. The biochemistry and cell biology of antigen processing and presentation. Annu Rev Immunol 1993; 11: 403-450.
24. Rammensee H-G, Falk K, Roetzschke O. Peptides naturally presented by MHC class I molecules. Annu Rev Immunol 1993; 11: 213-244.
25. Dai F et al. Cellular and humoral immune responses to adenoviral vector containing factor IX gene. Tolerization of factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 1995; 92: 1401-1405.
26. Halbert DN, Cutt JR, Shenk T. Adenovirus early region 4 encodes functions required for efficient DNA replication, late gene expression, and host cell shutoff. J Virol 1985; 56: 250-257.
27. Graham FL, Prevec L. Manipulation of adenovirus vector. In: Murray EJ (ed). Methods in Molecular Biology Gene Transfer and Expression Protocols. Vol 7. Humana Press: Clifton, NJ, 1991, pp 109-128.
28. Li Q et al. Assessment of recombinant adenoviral vectors for hepatic gene therapy. Hum Gene Ther 1993; 4: 403-409.
29. Langdell RD, Wagner RH, Brinkhous KM. Effect of antihemophilic factor on one-stage clotting tests. J Lab Clin Med 1953; 41: 637-647.
30. Brinkhous KM et al. Effect of recombinant factor VIIa on the hemostatic defect in dogs with hemophilia A, hemophilia B and von Willebrand disease. Proc Natl Acad Sci USA 1989; 86: 1382-1386.
31. Kay MA et al. Hepatic gene therapy: persistent expression of human alpha-1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther 1993; 3: 641-647.
32. Hornbeck P. Antibody detection and preparation. In: Coligan JE et al (eds). Current Protocols in Immunology. Vol 1. John Wiley: New York, 1994, pp 2.1.1-2.1.22.
33. Kasper CK, Pool JG. Measurement of mild factor VIII inhibitors in Bethesda units. Thrombos Diathes Haemorrh 1975; 34: 875-876.