Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles

Gene Therapy

Volumn 5, Issue 1, 1998, Pages 19-30

  Floyd Jr , S S ^a   Clemens, P R ^b   Ontell, M R ^b   Kochanek, S ^d   Day, C S ^a   Yang, J ^b   Hauschka, S D ^e   Balkir, L ^c   Morgan, J ^f   Moreland, M S ^a   Feero, G W ^c   Epperly, M ^b   Huard, J ^a  

^a CHILDREN S HOSPITAL OF PITTSBURGH   (United States)

^b UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF PITTSBURGH   (United States)

^d BAYLOR COLLEGE OF MEDICINE   (United States)

^e UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^f IMPERIAL COLLEGE SCHOOL OF MEDICINE   (United Kingdom)

Author keywords

Adenovirus; Dystrophin; Dystrophin associated proteins; Ex vivo approach; Mdx mouse

Indexed keywords

BETA GALACTOSIDASE; DYSTROPHIN;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL TRANSPLANTATION; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXPRESSION VECTOR; GENE TRANSFER; MOUSE; MYOBLAST; NONHUMAN; PRIORITY JOURNAL; RECOMBINANT GENE; TRANSGENIC MOUSE;

ADENOVIRIDAE; ANIMALIA; MUS MUSCULUS;

EID: 6844257545     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300549     Document Type: Article

References (77)

1. Hoffman EP, Brown T, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51: 919-928.
2. Arahata K et al. Immunostaining of skeletal and cardiac muscle surface membrane with antibody against Duchenne muscular dystrophy peptide. Nature 1989; 333. 861-863.
3. Sugita H et al. Muscular dystrophy (DMD) and mdx muscle surface membrane with antibody against synthetic peptide fragment predicted from DMD cDNA. Proc Jpn Acad 1988; 64: 37-39.
4. Zubryzcka-Gaarn EE et al. The Duchenne muscular dystrophy gene is localized in the sarcolemma of human skeletal muscle Nature 1988; 333: 466-469.
5. Ervasti JM, Campbell KP. Membrane organization of the dystrophin-glycoprotein complex. Cell 1991; 66: 1121-1131.
6. Ibraghinov-Beskrovnaya et al. Primary structure of dystrophinassociated proteins linking dystrophin to the extracellular matrix. Nature 1992; 355: 696-702.
7. Matsumura K et al. Association of dystrophin-related protein with dystrophin-associated proteins in mdx mouse muscle. Nature 1992; 360: 588-591.
8. Matsumura K, Campbell KP. Dystrophin-glycoprotein complex: its role in the molecular pathogenesis of muscular dystrophies. Muscle Nerve 1994; 17: 2-15.
9. Ozawa E et al Dystrophin-associated proteins in muscular dystrophy. Hum Mol Genet 1995; 4: 1711-1716.
10. Watkins SC, Hoffman EP, Slayter HS, Kunkel LM. Immunoelectron microscopic localization of dystrophin in myofibers. Nature 1988; 333: 863-866.
11. Bonilla EC et al. Duchenne muscular dystrophy: deficiency of dystrophin at the muscle cell surface Cell 1988; 54: 447-452.
12. Menke A, Jokush H. Decreased osmotic stability of dystrophinless muscle cells from the mdx mice. Nature 1991; 349: 69-71.
13. Bulfield G, Siller WG, Wright PAL, Moore KJ. X chromosomelinked muscular dystrophy (mdx) in the mouse. Proc Natl Acad Sci USA 1984; 81: 1189-1192.
14. Sincinsky P et al. The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 1989; 244: 1578-1580.
15. Partridge TA. Myoblast transfer: a possible therapy for inherited myopathies. Muscle Nerve 1991; 14: 197-212.
16. Law PK, Goodwin TG, Li HJ. Histoincompatible myoblast injection improves muscle structure and function of dystrophic mice. Transplant Proc 1988, 20: 1014-1019.
17. Partridge TA et al. Conversion of mdx myofibers from dystrophin negative to positive by injection of normal myoblasts. Nature 1989; 337: 176-179.
18. Alamedine HS, Dehaupas M, Fardeau M. Regeneration of skeletal muscle fibers from autologous satellite cells multiplied in vitro Muscle Nerve 1989; 12: 544-555.
19. Karpati G et al. Dystrophin is expressed in mdx skeletal muscle fibers after normal myoblast implantation. Am J Pathol 1989; 135: 27-32.
20. Morgan JE, Hoffman EP, Partridge TA. Normal myogenic cells from newborn mice restore normal histology to degenerating muscle of the mdx mouse. J Cell Biol 1990; 111: 2437-2449.
21. Huard J et al. Dystrophm expression in myotubes formed by the fusion of normal and dystrophic myoblasts. Muscle Nerve 1991; 14: 178-182.
22. Huard J et al. Human myoblast transplantation in immunodeficient and immunosuppressed mice: evidence of rejection. Muscle Nerve 1992; 17: 224-234.
23. Huard J et al. High efficiency of muscle regeneration following human myoblast clone transplantation in SCID mice. J Clin Invest 1994; 93: 586-599.
24. Kinoshita I et al. Very efficient myoblast allotransplantation in mice under FK506 immunosuppression. Muscle Nerve 1994; 17: 1407-1415
25. Law PK et al. Pioneering development of myoblast transfer therapy. In: Angelini C, Darrieli GA, Fontanan D (eds). Muscular Dystrophy Research. Excerpta Medica. International Congress Series Elsevier: New York, 1991, pp 109-116.
26. Huard J et al. Human myoblast transplantation: preliminary results of four cases. Muscle Nerve 1992; 15: 550-560.
27. Huard J et al. Human myoblast transplantation between immunohistocompatible donors and recipients produces immune reactions Transplant Proc 1992; 24: 3049-3051.
28. Gussoni E et al. Normal dystrophin transcripts detected in DMD patients after myoblast transplantation. Nature 1992; 356: 435438.
29. Tremblay JP et al Results of a blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy. Cell Transplant 1993; 2: 99-112.
30. Karpati G et al Myoblast transfer in Duchenne muscular dystrophy. Ann Neural 1993; 34: 8-17.
31. Mendell JR et al. Myoblast transfer in the treatment of Duchenne muscular dystrophy. New Engl J Med 1995; 333: 832-838.
32. Vilquin JT et al. Successful histocompatible myoblast transplantation in the dystrophin-deficient mdx mouse despite the production of antibodies against dystrophin. J Cell Biol 1995; 131: 975-988.
33. Acsadi G et al. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 1991; 352: 815-818.
34. Wolff JA et al. Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle Hum Mol Genet 1992; 1. 363-369.
35. Katsumi A et al. Humoral and cellular immunity to an encoded protein induced by direct DNA injection. Hum Gene Ther 1994; 5: 1335-1339
36. Dunckley MG et al. Retrovirus-mediated transfer of a dystrophin mirugene into mdx mouse myoblasts in vitro. Febs Lett 1992; 296. 128-134
37. Dunckley MG, Wells DJ, Walsh FS, Dickson G. Direct retrovirusmediated transfer of a dystrophin minigene into mdx mouse muscle in vivo. Hum Mol Genet 1993; 6: 717-723
38. Quantin B Perricaudet LD, Tajbakhsh S, Mandell JL. Aden- ovirus as an expression vector in muscle cells in vivo Proc Natl Acad Sci USA 1992; 89: 2581-2584.
39. Ragot T et al. Efficient adenovirus-mediated gene transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 1993; 361: 647-650.
40. Vincent M et al. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nat Genet 1993; 5: 130-134.
41. Acsadi G et al. A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells at different maturity. Hum Mol Genet 1994; 3: 579-584.
42. Acsadi G et al. Cultured human myoblasts and myotubes show markedly different transducibility by replication-defective adenovirus recombinant. Gene Therapy 1994; 1: 338-340.
43. Smith TAG et al. Adenovirus-mediated expression of therapeutic plasma levels of human factor IX in mice. Nat Genet 1993; 5: 397-402.
44. Yang Y et al. Cellular immunity to viral antigens limits E1deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 1994; 91: 4407-4411.
45. Engelhardt JF, Ye X, Doranz B, Wilson JM. Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory responses in mouse liver. Proc Natl Acad Sci USA 1994; 91: 6196-6200.
46. Dai Y et al. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene. Tolerization of factor IX and vector antigens allow for long-term expression. Proc Natl Acad Sci USA 1995; 92: 1401-1405.
47. Acsadi G et al. Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer. Hum Gene Ther 1996; 7: 129-140.
48. Kochanek S et al. 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.
49. Kumar-Singh R, Chamberlain JS. Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells. Hum Mol Genet 1996; 5: 913-921.
50. Haecker SE 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.
51. Clemens PR et al. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Therapy 1996; 3: 965-972.
52. Huard J, Coins B, Glorioso JC. Herpes simplex virus type 1 vector mediated gene transfer to muscle. Gene Therapy 1995; 2: 1-9.
53. Huard J et al. The basal lamina is a physical barrier to herpes simplex virus-mediated gene delivery to mature muscle fibers. J Virol 1996; 70: 8117-8123.
54. Huard J et al. LacZ gene transfer to skeletal muscle using a replication defective herpes simplex virus type 1 mutant vector. Hum Gene Ther 1997; 4: 439-452.
55. Huard J et al. Gene transfer into skeletal muscles by isogenic myoblasts. Hum Gene Ther 1994; 5: 949-958.
56. Salvatori G et al. Retroviral vector-mediated gene transfer into human primary myogenic cells lead to expression in muscle fibers in vivo. Hum Gene Ther 1993; 4: 713-723.
57. Morgan JE et al. Myogenic cell lines derived from transgenic mice carrying a thermolabile T antigen: a model system for the derivation of tissue-specific and mutation-specific cell lines. Devel Biol 1994; 162: 486-498.
58. Cox GA et al. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 1993; 364: 725-729.
59. Hoffman EP et al. Somatic reversion/suppression of the mouse mdx phenotype in vivo. J Neurol Sci 1990; 99: 9-25.
60. Labrecque C et al. A new technique to identify hybrid myotubes in vitro without culture fixation. J Histochem Cytochem 1991; 39: 139-143.
61. Satoh A, Huard J, Tremblay JP. Use of fluorescent latex microspheres (FLMs) to follow the fate of transplanted myoblasts. J Histochem Cytochem 1993; 41: 1579-1582.
62. Kinoshita I et al. Transplantation of myoblasts from a transgenic mouse overexpressing dystrophin produced only a relatively small increase of dystrophin-positive membrane. Muscle Nerve 1997 (in press).
63. Harris JB, Johnson MA, Karlsson F. Pathological responses of rat skeletal muscle to a single subcutaneous injection of a toxin isolated from the venom of the Australian tiger snake, Notechis scutatus scutatus. Clin Exp Pharmacol Physiol 1975; 2: 383-404.
64. Beauchamps JR, Morgan JE, Pagel CN, Partridge TA. Quantitative studies of efficacy of myoblast transplantation. Muscle Nerve 1994, 18: S261.
65. Fan Y, Maley M, Beilharz M, Grounds M. Rapid death of injected myoblasts in myoblast transfer therapy. Muscle Nerve 1996; 19: 853-860.
66. Booth DK et al. Myoblast mediated ex vivo gene transfer to mature muscle. Tissue Engin 1997; 3: 125-133.
67. van Deutekom JCT, Hoffman EP, Huard J. Muscle maturation: implication for gene therapy. Molec Med Today 1997 (submitted).
68. Blau HM, Webster C, Pavlath GK. Defective myoblasts identified in Duchenne muscular dystrophy. Proc Natl Acad Sci USA 1983; 81: 4856-4860.
69. dy/dy) muscle. J Neurobiol 1992; 23: 407-419.
70. Chen HH et al. Persistence in muscle of an adenoviral vector that lacks all viral genes Proc Natl Acad Sci USA 1997; 94: 1645-1650
71. Yang Y, Haecker SE, Su Q, Wilson JM. Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle. Hum Mol Genet 1996; 5: 1703-1712.
72. Vilquin JT et al. FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer. Hum Gene Ther 1995; 6: 1391-1401.
73. Engel A, Yamamoto M, Fischbeck KH. Chapter 41. In: Engle AG, Franzini-Armstrong C (eds). Muscular Dystrophies; Dystrophinopathies. Myology (2nd edn). McGraw-Hill: USA, 1994, pp 1133-1187.
74. Sanes JR, Rubenstein JLR, Nicolas JF. Use of a recombinant retrovirus to study post-implantation cell lineage in mouse embryos. Embryol J 1986; 5: 5133-5142.
75. Yoshida M et al. Dissociation of the complex of dystrophin and its associated proteins into several unique groups by n-octyl beta-D-glucosid. Eur J Biochem 1994; 222: 1055-1061.
76. Mizuno Y et al. Selective defect of sarcoglycan complex in severe childhood autosomal recessive muscular dystrophy muscle. Biochem Biophys Res Commun 1994; 203: 979-983.
77. Larsson LI. Immunohistochemistry Theory and Practice. CRC Press: Boca Raton, FL, 1988, pp 77-130.