Respiratory muscles as a target for adenovirus-mediated gene therapy

European Respiratory Journal

Volumn 11, Issue 2, 1998, Pages 492-497

  Petrof, B J ^a,b  

^a ROYAL VICTORIA HOSPITAL   (Canada)

^b MCGILL UNIVERSITY   (Canada)

Author keywords

Adenovirus; Diaphragm function; Duchenne muscular dystrophy; Dystrophin; Gene transfer; Immunosuppression

Indexed keywords

DYSTROPHIN; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; BREATHING MUSCLE; CONTROLLED STUDY; DIAPHRAGM; DUCHENNE MUSCULAR DYSTROPHY; GENE THERAPY; GENE TRANSFER; IMMUNOSUPPRESSIVE TREATMENT; INJECTION; MOUSE; PRIORITY JOURNAL; REVIEW; VIRUS RECOMBINANT;

ADENOVIRIDAE; ANIMALS; GENE TARGETING; GENE THERAPY; GENETIC VECTORS; HUMANS; MUSCULAR DYSTROPHIES; RESPIRATORY MUSCLES;

EID: 0031909157     PISSN: 09031936     EISSN: None     Source Type: Journal    
DOI: 10.1183/09031936.98.11020492     Document Type: Review

References (46)

1. Smith PEM, Calverley PMA, Edwards RHT, Evans GA, Campbell EJM. Practical problems in the respiratory care of patients with muscular dystrophy. N Engl J Med 1987; 316: 1197-1204.
2. Khan MA. Corticosteroid therapy in Duchenne muscular dystrophy. J Neurol Sci 1993; 120: 8-14.
3. Hoffman EP, Brown RHJ, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51: 919-928.
4. Menke A, Jockusch H. Decreased osmotic stability of dystrophin-less muscle cells from the mdx mouse. Nature 1991; 349: 69-71.
5. Petrof BJ, Shrager JB, Stedman HH, Kelly AM, Sweeney HL. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc Natl Acad Sci USA 1993; 90: 3710-3714.
6. 2+. J Physiol 1990; 420: 337-353.
7. Williams DA, Head SI, Lynch GS, Stephenson DG. Contractile properties of skinned muscle fibres from young and adult normal and dystrophic (mdx) mice. J Physiol 1993; 460: 51-67.
8. Kozarsky KF, Wilson JM. Gene therapy: adenovirus vectors. Current Opinion in Genetics and Development 1993; 3: 499-503.
9. Horwitz MS. Adenoviridae and Their Replication. In: Fields BN, ed. Virology, 2nd Edition. New York, Raven Press Ltd. 1990; pp. 1679-1721.
10. Wold WSM, Gooding LR. Region E3 of adenovirus: A cassette of genes involved in host immunosurveillance and virus-cell interactions. Virology 1991; 184: 1-8.
11. Yang Y, Nunes FA, Berencsi K, Furth EE, Gonczol E, Wilson JM. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 1994; 91: 4407-4411.
12. Yang Y, Li Q, Ertl HCJ, Wilson JM. Cellular and humoral immune response to viral antigens create barriers to lung-directed gene therapy with recombinant adenovirus. J Virol 1995; 69: 2004-2015.
13. Engelhardt 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.
14. Yang Y, Nunes FA, Berencsi K, Gonczol E, Engelhardt JF, Wilson JM. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nature Gene 1994; 7: 362-369.
15. Dai Y, Schwarz EM, Gu D, Zhang WW, Sarvetnick N, Verma IM. Cellular and humoral immune responses to adenoviral vectors 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.
16. Whitton JL, Oldstone MBA. Virus-induced immune response interactions. In: Fields BN, ed. Virology, 2nd Edition. New York, Raven Press, Ltd. pp. 369-381.
17. Brutkiewicz RR, Welsh RM. Major histocompatibility complex class I antigens and the control of viral infections by natural killer cells. J Virol 1995; 69: 3967-3971.
18. Ginsberg HS, Moldawer LL, Sehgal PB, et al. A mouse model for investigating the molecular pathogenesis of adenovirus pneumonia. Proc Natl Acad Sci USA 1991; 88: 1651-1655.
19. Dalakas MC. Basic aspects of neuroimmunology as they relate to immunotherapeutic targets: Present and future prospects. Ann Neurol 1995; 37: S1-S13.
20. Kuiper M, Peakman M, Farzaneh F. Ovarian tumour antigens as potential targets for immune gene therapy. Gene Ther 1995; 2: 7-15.
21. 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. Nature Med 1996; 2: 545-550.
22. Tripathy SK., Goldwasser E, Lu M-M, Barr E, Leiden JM. Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus. Proc Natl Acad Sci USA 1994; 91: 11557-11561.
23. Acsadi G, Lochmuller H, Jani A, et al. Dystrophin expression in muscles of mdx mice after adenovirus-medialed in vivo gene transfer. Hum Gene Ther 1996; 7: 129-140.
24. Petrof BJ, Acsadi G, Jani A, et al. Efficiency and functional consequences of adenovirus-mediated in vivo gene transfer to normal and dystrophic (mdx) mouse diaphragm. Am J Respir Cell Mol Biol 1995; 13: 508-517.
25. 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.
26. Davis HL, Demeneix BA, Quantin B, Coulombe J, Whalen RG. Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal muscle. Hum Gene Ther 1993; 4: 733-740.
27. Acsadi G, Jani A, Massie B, Simoneau M, Holland P, Karpati G. A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity. Hum Mol Genet 1994; 3: 579-584.
28. Wickham TJ, Mathias P, Cheresh DA, Nemerow GR. Integrins αvβ3 and αvβ5 promote adenovirus internalization but not virus attachment. Cell 1993; 73: 309-319.
29. Stedman HH, Sweeney HL, Shrager JB, et al. The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 1991; 352: 536-539.
30. Petrof BJ, Stedman HH, Shrager JB, Eby J, Sweeney HL, Kelly AM. Adaptations in myosin heavy chain expression and contractile function in the dystrophic (MDX) mouse diaphragm. Am J Physiol 1993; 265: C834-C841.
31. Petrof BJ, Lochmüller H, Massie B, et al. Impairment of force generation after adenovirus-mediated gene transfer to muscle is alleviated by adenoviral gene inactivation and host CD8+ T cell deficiency. Hum Gene Ther 1996; 7: 1813-1826.
32. Wilcox P, Osborne S, Bressler B. Monocyte inflammatory mediators impair in vitro hamster diaphragm contractility. Am Rev Respir Dis 1992; 146: 462-466.
33. Lochmuller H., Petrof BJ, Pari G, et al. Transient immunosuppressive by FK506 permits a sustained high-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscles of adult dystrophin (mdx) mice. Gene Ther 1996; 3: 706-716.
34. Ungureanu-Longrois, D, Balligand J-L, Okada I, et al. Contractile responsiveness of ventricular myocytes to isoproterenol is regulated by induction of nitric oxide synthase activity in cardiac microvascular endothelial cells in heterotypic primary culture. Circ Res 1995; 77: 486-493.
35. Kobzik L, Reid MB, Bredt DS, Stamler JS. Nitric oxide in skeletal muscle. Nature 1994; 372: 546-548.
36. Boczkowski J, Lanone S, Ungureanu-Longrois D, Danialou G, Fournier T, Aubier M. Induction of diaphragmatic nitric oxide synthase after endotoxin administration in rats. J Clin Invest 1996; 98: 1550-1559.
37. 2-Microglobulin deficient mice lack CD4-8+ cytolytic T cells. Nature 1990; 344: 742-746.
38. Matloubian M, Concepcion RJ, Ahmed R. CD4+ T cells are required to sustain CD8+ cytotoxic T-cell responses during chronic viral infection. J Virol 1994; 68: 8056-8063.
39. Yang Y, Xiang Z, Ertl HCJ, Wilson JM. Upregulation of class I major histocompatibility complex antigens by interferon gamma is necessary for T-cell-mediated elimination of recombinant adenovirus-infected hepatocytes in vivo. Proc Natl Acad Sci USA 1995; 92: 7257-7261.
40. 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.
41. Dobner T, Horikoshi N, Rubenwolf S, Shenk I. Blockage by adenovirus E4orf6 of transcriptional activation by the p53 tumor suppressor. Science 1996; 272: 1470-1473.
42. Cotten M, Saltik M, Kursa M, Wagner E, Maass G, Birnstiel ML. Psoralen treatment of adenovirus particles eliminates virus replication and transcription while maintaining the endosomolytic activity of the virus capsid. Virology 1994; 205: 254-261.
43. McCoy RD, Davidson BL, Roessler BJ, et al. Pulmonary inflammation induced by incomplete or inactivated adenoviral particles. Hum Gene Ther 1995; 6: 1533-1560.
44. Kochanek S, Clemens PR, Mitani K, Chen H-H, Chan S, Caskey CT. A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc Natl Acad Sci USA 1996; 93: 5731-5736.
45. Haecker SE, Stedman HH, Balice-Gordon RJ, et al. In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes. Hum Gene Ther 1996; 7: 1907-1914.
46. Kay MA, Holterman A-X, Meuse L, et al. Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration. Nature Gene 1995; 11: 191-197.