Mini-dystrophin gene transfer in mdx4(cv) diaphragm muscle fibers increases sarcolemmal stability

Gene Therapy

Volumn 4, Issue 5, 1997, Pages 401-408

  Decrouy, A ^a   Renaud, J M ^a   Davis, H L ^a,b   Lunde, J A ^a   Dickson, G ^c   Jasmin, B J ^a  

^a UNIVERSITY OF OTTAWA   (Canada)

^b OTTAWA HOSPITAL RESEARCH INSTITUTE   (Canada)

^c UNIVERSITY OF LONDON   (United Kingdom)

Author keywords

Duchenne muscular dystrophy; Gene therapy; Sarcolemma

Indexed keywords

DYSTROPHIN; PLASMID DNA;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DIAPHRAGM MUSCLE; DUCHENNE MUSCULAR DYSTROPHY; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HINDLIMB; IMMUNOFLUORESCENCE TEST; IMMUNOPEROXIDASE STAINING; MOUSE; MUSCLE ATROPHY; MUSCLE CONTRACTION; MUSCLE REGENERATION; MUSCLE TETANIC CONTRACTION; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; SARCOLEMMA;

ANIMALIA;

EID: 0030914424     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300407     Document Type: Article

References (45)

1. Hoffman EP, Brown RH, Kunkel LM. Dystrophin: the protein product of the Duchenne Muscular Dystrophy locus. Cell 1987; 51: 919-928.
2. Koenig M, Monaco AP, Kunkel LM. The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein. Cell 1988; 53: 219-228.
3. Watkins SC, Hoffman EP, Slayter HS, Kunkel LM. Immunoelectron microscopic localization of dystrophin in myofibres. Nature 1988; 333: 863-866.
4. Zubrzycka-Gaarn EE et al. The Duchenne muscular gene product is localized in sarcolemma of human skeletal muscle. Nature 1988; 333: 466-469.
5. Levine BA, Moir AJG, Patchell VB, Perry SV. The interaction of actin with dystrophin. FEBS Lett 1990; 263: 159-162.
6. Way M et al. Expression of the N-terminal of dystrophin in E. coli and demonstration of binding to F-actin. FEBS Lett 1992; 301: 243-245.
7. Ohlendieck K, Ervasti JM, Snook JB, Campbell KP. Dystrophin-glycoprotein complex is highly enriched in isolated skeletal muscle sarcolemma. J Cell Biol 1991; 112: 135-148.
8. Campbell KP, Kahl SD. Association of dystrophin and an integral membrane glycoprotein. Nature 1989; 338: 259-261.
9. Ervasti JM, Campbell KP. Dystrophin and the membrane skeleton. Curr Opin Cell Biol 1993; 5: 82-87.
10. Pasternak C, Wong S, Elson EL. Mechanical function of dystrophin in muscle cells. J Cell Biol 1995; 128: 355-361.
11. Fabbrizio E et al. Dystrophin, the protein that promotes membrane resistance. Biochem Biophys Res Commun 1995; 213: 295-301.
12. Menke A, Jockush H. Decreased osmotic stability of dystrophinless muscle cells from the mdx mouse. Nature 1991; 349: 69-71.
13. Weller B, Karpati G, Carpenter S. Dystrophin-deficient mdx muscle fibers are preferentially vulnerable to necrosis induced by experimental lengthening contractions. J Neurol Sci 1990; 100: 9-13
14. Moens P, Baatsen PHWW, Maréchal G. Increased susceptibility of EDL muscles from mdx mice to damage induced by contractions with stretch. J Musc Res Cell Mot 1993; 14: 446-451.
15. Petrof BJ et al Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc Natl Acad Sci USA 1993; 90: 3710-3714.
16. Yang B et al. SHE domain-mediated interaction of dystroglycan and Grb2 J Biol Chem 1995, 270: 11711-11714.
17. Brenman JE et al. Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy. Cell 1995; 82: 743-752.
18. Brenman JE et al. Interaction of nitric oxide synthase with the postsynaptic density protein PSD-95 and α1-syntrophin mediated by PDZ domains. Cell 1996; 84. 757-767.
19. Grozdanovic Z, Gosztonyi G, Gossrau R. Nitric oxide synthase I (NOS-I) is deficient in the sarcolemma of striated muscle fibres in patients with Duchenne muscular dystrophy, suggesting an association with dystrophin. Acta Histochem 1996; 98: 61-69.
20. Bulfield G, Siller WG, Wight PAL, Moore KJ. X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc Natl Acad Sci USA 1984; 81: 1189-1192.
21. Sicinski P et al. The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 1989, 244: 1578-1580.
22. Dunckley MG, Wells DJ, Walsh FS, Dickson G. Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vitro. Hum Mol Genet 1993; 2: 717-723.
23. Ragot T et al. Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 1993; 361: 647-650.
24. Vincent N et al. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nat Genet 1993; 5: 130-134.
25. Ascadi G et al Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs Nature 1991; 352: 815-818.
26. Danko I et al. Dystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injection. Hum Mol Genet 1993; 2: 2055-2061.
27. England SB et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 1990; 343: 180-182.
28. Deconinck N et al. Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene. Proc Natl Acad Sci USA 1996; 93: 3570-3574.
29. Stedman HH et al. The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 1991; 352: 536-539.
30. Dupont-Versteegden EE, McCarter RJ. Differential expression of muscular dystrophy m diaphragm versus hindlimb muscles of mdx mice. Muscle and Nerve 1992; 15: 1105-1110.
31. Boland B, Himpens B, Denef JF, Gillis JM. Site-dependent pathological differences in smooth muscles and skeletal muscles of the adult mdx mouse. Muscle and Nerve 1995; 18: 649-657.
32. Davis HL, Jasmin BJ. Direct gene transfer into mouse diaphragm. FEBS Lett 1993; 333: 146-150.
33. Tanabe Y, Esaki K, Nomura T. Skeletal muscle pathology in X chromosome-linked muscular dystrophy (mdx) mouse. Acta Neuropathol (Berlin) 1986; 69: 91-95.
34. Karpati G, Carpenter S, Prescott S. Small-caliber skeletal muscle fibers do not suffer necrosis in mdx mouse dystrophy. Muscle and Nerve 1988; 11: 795-803
35. Decrouy A et al. Direct gene transfer with mini-dystrophin gene results in rapid improvement of physiological properties of mdx diaphragm muscle. Mol Biol Cell 1995; 6: 152a.
36. McCully KK, Faulkner JA. Injury to skeletal muscle fibers of mice following lengthening contractions. J Appl Physiol 1985; 59: 119-126.
37. Alameddine HS et al. Expression of a recombinant dystrophin in mdx mice using adenovirus vector. Neuromusc Dis 1994; 4: 193-203
38. Wells DJ et al. Human dystrophin expression corrects the myopathic phenotype in transgenic mdx mice. Hum Mol Genet 1992; 1: 35-45.
39. Cox GA et al. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 1993; 364: 725-729.
40. Matsumura K, Lee CC, Caskey CT, Campbell KP. Restoration of dystrophin-associated proteins in skeletal muscle of mdx mice transgenic for dystrophin gene. FEBS Lett 1993; 320: 276-280.
41. Phelps SF et al. Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice. Hum Mol Genet 1995; 4: 1251-1258.
42. Wells D et al. Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy. Hum Mol Genet 1995; 4: 1245-1250.
43. Danko I, Chapman V, Wolff JA. The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy. Pediatr Res 1992; 32: 128-131.
44. Lidov HGW, Byers TJ, Watkins SC, Kunkel LM. Localization of dystrophin to postsynaptic regions of central nervous system cortical neurons. Nature 1990; 348: 725-728.
45. Cartaud A et al. Localization of dystrophin and dystrophin-related protein at the electromotor synapse and neuromuscular junction in Torpedo marmorata. Neuroscience 1992; 48: 995-1003.