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Development of Viral Vectors for Human Gene Therapy: Retrovirus and Adenovirus (Part I)

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Gene Transfer in the Cardiovascular System

Part of the book series: Developments in Cardiovascular Medicine ((DICM,volume 189))

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Abstract

Gene therapy is one of the most exciting and rapidly advancing fields in modern medical science. Increasing investigative activity in laboratories and clinics throughout the world has led to conceptualization and evaluation of strategies for gene therapy for a number of genetic and acquired disorders. At the time of this writing, 136 clinical trials have been approved by the National Institutes of Health (NIH) Recombinant DNA Activities Committee (RAC) and more than 600 individuals have received transferred genes [1]. It is remarkable to note that the gene-enzyme basis of disease was first hypothesized by Garrod only 86 years ago.

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References

  1. Tolstoshev P. Gene Therapy. In Mendelsohn J, Howley PM, Israel MA and Liotta LA, editors. The Molecular Basis of Cancer. W.B. Saunders Company, 1995:534–557.

    Google Scholar 

  2. Rosenberg SA, Aebersold P, Cometta K, et al. Gene transfer into humans: Immunotherapy of patients with advanced melanoma, using tumor infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990;326:370–378.

    Google Scholar 

  3. Trapnell BC. Adenoviral vectors for gene transfer. Adv Drug Del Rev 1994;12:185–199.

    Article  Google Scholar 

  4. Zabner J, Couture LA, Gregory RJ, Graham SM, Smith AE, Welsh MJ. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 1993;75:207–216.

    Article  PubMed  CAS  Google Scholar 

  5. Crystal RG, McElvaney NG, Rosenfeld MA, et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Gene 1994;8:42–51.

    Article  CAS  Google Scholar 

  6. Knowles MR, Hohoneker KW, Zhou Z, et al. A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N Engl J Med 1995;333:823–831.

    Article  PubMed  CAS  Google Scholar 

  7. Boucher RC, Knowles MR, Johnson LG, et al. Gene therapy for cystic fibrosis using El-deleted adenovirus: A phase I trial in the nasal cavity. Hum Gene Ther 1994;5:615–639.

    Article  PubMed  CAS  Google Scholar 

  8. Wilmott RW, Whitsett JA, Trapnell BC. Gene Therapy for cystic fibrosis utilizing a replication deficient recombinant adenovirus vector to deliver the human cystic fibrosis transmembrane conductance regulator cDNA to the airways, A phase I study. Hum Gene Ther 1994;5:1019–1057.

    Article  Google Scholar 

  9. Jones N, Shenk T. An adenovirus type 5 early gene function regulates expression of other early viral genes. Proc Natl Acad Sci USA 1979;76:3665–3669.

    Article  PubMed  CAS  Google Scholar 

  10. Trapnell, B.C., Gorziglia, M. Gene therapy using adenoviral vectors. Current Opinion in Biotechnology 1994;5:617–625.

    Article  PubMed  CAS  Google Scholar 

  11. Coffin JM. Structure and classification of retroviruses. In Levy JA, editor. The Retroviridae, Vol. 1. New York: Plenum Press, 1992:19–49.

    Chapter  Google Scholar 

  12. Morgan RA, Anderson WF. Human gene therapy. Annu Rev Biochem 1993, 62:191–217.

    Article  PubMed  CAS  Google Scholar 

  13. Varmus H, Brown P. Retroviruses. In Berg, DE, Howe MM, editors. Mobile DNA. Washington DC: ASM, 1989:53–108.

    Google Scholar 

  14. Speck NA, Baltimore D. Six distinct nuclear factors interact with the 75-base-pair repeat of the Moloney murine leukemia virus enhancer. Mol Cell Biol 1987;7:1101–1110.

    PubMed  CAS  Google Scholar 

  15. Morgan RA. Retroviral vectors in human gene therapy. In Vos JH, editor. Viruses in Human Gene Therapy. North Carolina: Carolina Academic Press, 1995:77–107.

    Chapter  Google Scholar 

  16. Armentano D, Yu S, Kantoff PW, von Ruden T, Anderson WF, Gilboa E. Effect of internal viral sequences on the utility of retroviral vectors. J Virol 1987, 61:1647–1650.

    PubMed  CAS  Google Scholar 

  17. Bender MA, Palmer TD, Gelinas RE, Miller AD. Evidence that the packaging signal of Moloney murine leukemia virus extends into the gag regioa J Virol 1987;61:1639–1646.

    PubMed  CAS  Google Scholar 

  18. Miller AD, Rosman GJ. Improved retroviral vectors for gene transfer and expression. Bio Techniques 1989;7:980–986.

    CAS  Google Scholar 

  19. McLachlin JR, Mittereder N, Daucher MB, Kadan M, Eglitis MA. Factors affecting retroviral vector function and structural integrity. Virology 1993;195:1–5.

    Article  PubMed  CAS  Google Scholar 

  20. Gilboa E, Eglitis MA, Kantoff PW, Anderson WF. Transfer and expression of cloned genes using retroviral vectors. Bio Techniques 1986;4:504–512.

    CAS  Google Scholar 

  21. Yee J, Moores JC, Jolly DJ, Wolff JA, Respess JG, Friedmann T. Gene expression from transcriptionally disabled retroviral vectors. Proc Natl Acad Sci USA 1987;84:5197–5201.

    Article  PubMed  CAS  Google Scholar 

  22. Challita P, Skelton D, El-Khoueiry A, Yu X, Weinberg K, Kohn DB. Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells. J Virol 1995;69:748–755.

    PubMed  CAS  Google Scholar 

  23. Ferry N, Duplessis O, Houssin D, Danos O, Heard J. Retro viral-mediated gene transfer into hepatocytes in vivo.Pro Natl Acad Sci USA 1991;88:8377–8381.

    Article  CAS  Google Scholar 

  24. Krall, WJ, Skelton DC, Kohn DB. Enhanced expression from the MFG vector backbone is due to increased splicing efficiency. J Cellular Biochemistry 1995;Supp. 21A:409.

    Google Scholar 

  25. Morgan RA, Couture L, Elroy-Stein O, Ragheb, Moss B, Anderson WF. Retroviral vectors containing putative internal ribosome entry sites: development of a polycistronic gene transfer system and applications to human gene therapy. Nucleic Acids Res 1992;20:1293–1299.

    Article  PubMed  CAS  Google Scholar 

  26. Miller AD, Buttimore C. Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol Cell Biol 1986:6:2895–2902.

    PubMed  CAS  Google Scholar 

  27. Markowitz D, Goff S, Bank A. A safe packaging line for gene transfer: separating viral genes on two different plasmids. J Virol 1988, 62:1120–1124.

    PubMed  CAS  Google Scholar 

  28. Miller AD. Retrovirus packaging cells. Hum Gene Ther 1990;1:5–14.

    Article  PubMed  CAS  Google Scholar 

  29. Danos O, Mulligan RC. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc Natl Acad Sci USA 1988;85:6460–6464.

    Article  PubMed  CAS  Google Scholar 

  30. Miller DC, Adam MA, Miller AD. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 1990; 10:4239.

    PubMed  CAS  Google Scholar 

  31. Lewis P, Hensel M, Emerman M. Human immunodeficiency virus infection of cells arrested in the cell cycle. EMBO 1992;11:3053–3058.

    CAS  Google Scholar 

  32. Brenner MK. Current gene marking and gene therapy protocols for human bone marrow transplantation. In Chang PL, editor. Somatic Gene Therapy. Tokyo: CRC Press, 1995:225–242.

    Google Scholar 

  33. Culver KW, Van Gilder J, Carlstrom T, Prados M, Link CJ, Jr. Gene therapy for brain tumors. In Chang PL, editor. Somatic Gene Therapy. Tokyo: CRC Press, 1995,243–262.

    Google Scholar 

  34. Wells S, Malik P, Pensiero M, Kohn DB, Nolta JA. The presence of an autologous marrow stromal cell layer increases glucocerebrosidase gene transduction of long-term culture initiating cells (LTCICs) from the bone marrow of a patient with Gaucher disease. Gene Therapy 1995;2:512–520.

    PubMed  CAS  Google Scholar 

  35. Ginsberg, HS. The Adenoviruses. New York-London: Plenum Press, 1984.

    Book  Google Scholar 

  36. Horwitz MS. Adenoviridae and their replication. In Fields BN, Knipe DM, et al., editors. Virology. New York: Raven Press Ltd., 1990:1723–1740.

    Google Scholar 

  37. Straus SE. Adenovirus infections in humans. In Ginsburg HS, editor. The Adenoviruses. New York: Plenum Press, 1984:451–496.

    Chapter  Google Scholar 

  38. Buescher EL. Respiratory disease and the adenoviruses. Med Clin North Am 1967;51:769–779.

    PubMed  CAS  Google Scholar 

  39. Top FH, Dudding BA, Russell PK, Buescher EL. Control of respiratory disease in recruits with types 4 and 7 adenovirus vaccines. Am J Epidemiol 1971;94:142–146.

    PubMed  Google Scholar 

  40. Seth P, Fitzgerald D, Willingham M, Pastan I. Pathway of adenovirus entry into cells. Mol Cell Biol 1984;4:1528–1533.

    PubMed  CAS  Google Scholar 

  41. Berkner KL. Development of adenovirus vectors for the expression of heterologous genes. Bio Techniques 1988;6:616–624.

    CAS  Google Scholar 

  42. Smith TAG, Mehaffey MG, Kayda DB, et al. Adenovirus mediated expression of therapeutic plasma levels of human factor DC in mice. Nat Gene 1993;5:397–402.

    Article  CAS  Google Scholar 

  43. Simon RH, et al. Adeno-mediated transfer of the CFTR gene to lung of nonhuman primates: toxicity study. Hum Gene Ther 1993;4:771–780.

    Article  PubMed  CAS  Google Scholar 

  44. Yei S, Mittereder N, Wert S, Whitsett JA, Wilmott RW, Trapnell BC. In Vivo Evaluation of the safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lung. Hum Gene Ther 1994;5:733–746.

    Article  Google Scholar 

  45. Yang Y, Li Q, Ertl HCJ, Wilson JM. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 1995;69:2004–2015.

    PubMed  CAS  Google Scholar 

  46. Yang Y, Nunes FA, Berencsi K, Furth EE, 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.

    Article  PubMed  CAS  Google Scholar 

  47. Yei, S, Mittereder, N, Tank, K, O’Sullivan, C, Trapnell, BC. Adenovirus-mediated gene transfer for cystic fibrosis: quantitative evaluation of repeated in vivo vector administration to the lung. Gene Therapy 1994; 1:192–200.

    PubMed  CAS  Google Scholar 

  48. Mittereder N, Yei S, Bachurski C, et al. Evaluation of the efficacy and safety of in vitro, adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA. Hum Gene Ther 1994;5:719–731.

    Article  Google Scholar 

  49. Engelhardt JF, Ye S, 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.

    Article  PubMed  CAS  Google Scholar 

  50. Gorziglia MI, Kadan MJ, Yei S, et al. Elimination of both E1 and E2a from adenovirus vectors further improves prospects for in vivo human gene therapy. J Virol (In press).

    Google Scholar 

  51. Armentano D, Sookdeo CC, Hehir KM, et al. Characterization of an adenovirus gene transfer vector containing an E4 deletion. Hum Gene Ther 1995;6:1343–1353.

    Article  PubMed  CAS  Google Scholar 

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Author information

Authors and Affiliations

  1. Department of Virology, Genetic Therapy, Inc., Gaithersburg, MD, 20878, USA

    Bruce C. Trapnell M.D. & Michael N. Pensiero Ph.D.

Authors
  1. Bruce C. Trapnell M.D.
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  2. Michael N. Pensiero Ph.D.
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Editor information

Editors and Affiliations

  1. Krannert Institute of Cardiology, Indiana University Medical Center, Indianapolis, Indiana, USA

    Keith L. March M.D., Ph.D.

  2. R. L. Roudebush VA Medical Center, Indianapolis, Indiana, USA

    Keith L. March M.D., Ph.D.

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© 1997 Springer Science+Business Media New York

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Trapnell, B.C., Pensiero, M.N. (1997). Development of Viral Vectors for Human Gene Therapy: Retrovirus and Adenovirus (Part I). In: March, K.L. (eds) Gene Transfer in the Cardiovascular System. Developments in Cardiovascular Medicine, vol 189. Springer, Boston, MA. https://doi.org/10.1007/978-1-4615-6277-1_1

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  • DOI: https://doi.org/10.1007/978-1-4615-6277-1_1

  • Publisher Name: Springer, Boston, MA

  • Print ISBN: 978-1-4613-7881-5

  • Online ISBN: 978-1-4615-6277-1

  • eBook Packages: Springer Book Archive

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