Dystrophin expression in muscles of Duchenne muscular dystrophy patients after high-density injections of normal myogenic cells

Journal of Neuropathology and Experimental Neurology

Volumn 65, Issue 4, 2006, Pages 371-386

  Skuk, Daniel ^a   Goulet, Marlyne ^a   Roy, Brigitte ^a   Chapdelaine, Pierre ^a   Bouchard, Jean Pierre ^a   Roy, Raynald ^a   Dugré, Francine J ^a   Sylvain, Michel ^a   Lachance, Jean Guy ^a   Deschênes, Louise ^a   Senay, Hélène ^a   Tremblay, Jacques P ^a  

^a LAVAL UNIVERSITY   (Canada)

Author keywords

Cell transplantation; Clinical trial; Duchenne muscular dystrophy; Immune reaction; Muscle precursor cell; Tacrolimus

Indexed keywords

DYSTROPHIN; IMMUNOSUPPRESSIVE AGENT; TACROLIMUS; HISTOCOMPATIBILITY ANTIGEN;

ACUTE GRAFT REJECTION; ADOLESCENT; ALLOTRANSPLANTATION; ARTICLE; CELL CULTURE; CELL DENSITY; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; FEMALE; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOPROPHYLAXIS; IMMUNOSUPPRESSIVE TREATMENT; INJECTION SITE; MALE; MUSCLE BIOPSY; MUSCLE CELL; PRIORITY JOURNAL; PROTEIN DETERMINATION; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SCHOOL CHILD; SKELETAL MUSCLE; STEM CELL TRANSPLANTATION; TIBIALIS ANTERIOR MUSCLE; ANIMAL; BIOSYNTHESIS; CHILD; FLUORESCENCE IN SITU HYBRIDIZATION; FLUORESCENT ANTIBODY TECHNIQUE; GRAFT REJECTION; IMAGE PROCESSING; IMMUNOLOGY; METABOLISM; MOUSE; MOUSE MUTANT; PATHOLOGY; TRANSPLANTATION;

ADOLESCENT; ANIMALS; CHILD; DYSTROPHIN; FLUORESCENT ANTIBODY TECHNIQUE; GRAFT REJECTION; HISTOCOMPATIBILITY ANTIGENS; HUMANS; IMAGE PROCESSING, COMPUTER-ASSISTED; IMMUNOSUPPRESSIVE AGENTS; IN SITU HYBRIDIZATION, FLUORESCENCE; MICE; MICE, SCID; MUSCLE CELLS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; STEM CELL TRANSPLANTATION; TACROLIMUS;

EID: 33744983289     PISSN: 00223069     EISSN: None     Source Type: Journal    
DOI: 10.1097/01.jnen.0000218443.45782.81     Document Type: Article

References (34)

1. Partridge TA, Grounds M, Sloper JC. Evidence of fusion between host and donor myoblasts in skeletal muscle grafts. Nature 1978;273:306-08
2. Partridge TA, Morgan JE, Coulton GR, Hoffman EP, Kunkel LM. Conversion of mdx myofibres from dystrophin-negative to-positive by injection of normal myoblasts. Nature 1989;337:176-79
3. Kinoshita I, Vilquin JT, Guerette B, Asselin I, Roy R, Tremblay JP. Very efficient myoblast allotransplantation in mice under FK506 immunosuppression. Muscle Nerve 1994;17:1407-15
4. Vilquin JT, Asselin I, Guerette B, Kinoshita I, Roy R, Tremblay JP. Successful myoblast allotransplantation in mdx mice using rapamycin. Transplantation 1995;59:422-26
5. Skuk D, Goulet M, Roy B, Tremblay JP. Myoblast transplantation in whole muscle of nonhuman primates. J Neuropathol Exp Neurol 2000;59:197-206
6. Skuk D, Goulet M, Roy B, Tremblay JP. Efficacy of myoblast transplantation in nonhuman primates following simple intramuscular cell injections: Toward defining strategies applicable to humans. Exp Neurol 2002;175:112-26
7. Skuk D, Roy B, Goulet M, Tremblay JP. Successful myoblast transplantation in primates depends on appropriate cell delivery and induction of regeneration in the host muscle. Exp Neurol 1999;155:22-30
8. Kinoshita I, Vilquin JT, Gravel C, Roy R, Tremblay JP. Myoblast allotransplantation in primates. Muscle Nerve 1995;18:1217-18
9. Kinoshita I, Roy R, Dugre FJ, et al. Myoblast transplantation in monkeys: Control of immune response by FK506. J Neuropathol Exp Neurol 1996;55:687-97
10. Skuk D, Roy B, Goulet M, et al. Dystrophin expression in myofibers of Duchenne muscular dystrophy patients following intramuscular injections of normal myogenic cells. Mol Ther 2004;9:475-82
11. Tremblay JP, Roy B, Goulet M. Human myoblast transplantation: A simple assay for tumorigenicity. Neuromuscul Disord 1991;1:341-43
12. Chapdelaine P, Moisset PA, Campeau P, Asselin I, Vilquin JT, Tremblay JP. Functional EGFP-dystrophin fusion proteins for gene therapy vector development. Protein Eng 2000;13:611-15
13. Chapdelaine P, Vignola K, Fortier MA. Protein estimation directly from SDS-PAGE loading buffer for standardization of samples from cell lysates or tissue homogenates before Western blot analysis. Biotechniques 2001;31:478, 480, 482
14. Fanin M, Danieli GA, Cadaldini M, Miorin M, Vitiello L, Angelini C. Dystrophin-positive fibers in Duchenne dystrophy: Origin and correlation to clinical course. Muscle Nerve 1995;18:1115-20
15. Watt DJ, Lambert K, Morgan JE, Partridge TA, Sloper JC. Incorporation of donor muscle precursor cells into an area of muscle regeneration in the host mouse. J Neurol Sci 1982;57:319-31
16. Huang YC, Dennis RG, Larkin L, Baar K. Rapid formation of functional muscle in vitro using fibrin gels. J Appl Physiol 2005;98:706-13
17. Okano T, Matsuda T. Muscular tissue engineering: Capillary-incorporated hybrid muscular tissues in vivo tissue culture. Cell Transplant 1998;7:435-42
18. Yao SN, Kurachi K. Implanted myoblasts not only fuse with myofibers but also survive as muscle precursor cells. J Cell Sci 1993;105:957-63
19. Gross JG, Morgan JE. Muscle precursor cells injected into irradiated mdx mouse muscle persist after serial injury. Muscle Nerve 1999;22:174-85
20. Mendell JR, Kissel JT, Amato AA, et al. Myoblast transfer in the treatment of Duchenne's muscular dystrophy. N Engl J Med 1995;333:832-38
21. Miller RG, Sharma KR, Pavlath GK, et al. Myoblast implantation in Duchenne muscular dystrophy: The San Francisco study. Muscle Nerve 1997;20:469-78
22. Morandi L, Bernasconi P, Gebbia M, et al. Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer. Neuromuscul Disord 1995;5:291-95
23. Tremblay JP, Malouin F, Roy R, et al. Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy. Cell Transplant 1993;2:99-112
24. Karpati G, Ajdukovic D, Arnold D, et al. Myoblast transfer in Duchenne muscular dystrophy. Ann Neurol 1993;34:8-17
25. Huard J, Bouchard JP, Roy R, et al. Human myoblast transplantation: Preliminary results of 4 cases. Muscle Nerve 1992;15:550-60
26. Gussoni E, Pavlath GK, Lanctot AM, et al. Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 1992;356:435-38
27. Skuk D. Myoblast transplantation for inherited myopathies: A clinical approach. Expert Opin Biol Ther 2004;4:1871-85
28. Skuk D, Tremblay JP. Myoblast transplantation: The current status of a potential therapeutic tool for myopathies. J Muscle Res Cell Motil 2003;24:285-300
29. Hardiman O, Sklar RM, Brown RH Jr. Direct effects of cyclosporin A and cyclophosphamide on differentiation of normal human myoblasts in culture. Neurology 1993;43:1432-34
30. Hong F, Lee J, Song JW, et al. Cyclosporin A blocks muscle differentiation by inducing oxidative stress and inhibiting the peptidylprolyl-cis-trans isomerase activity of cyclophilin A: Cyclophilin A protects myoblasts from cyclosporin A-induced cytotoxicity. Faseb J 2002;16:1633-35
31. Vilquin JT, Kinoshita I, Roy R, Tremblay JP. Cyclophosphamide immunosuppression does not permit successful myoblast allotransplantation in mouse. Neuromuscul Disord 1995;5:511-17
32. Kinoshita I, Vilquin JT, Guerette B, et al. Immunosuppression with FK 506 insures good success of myoblast transplantation in MDX mice. Transplant Proc 1994;26:3518
33. McDouall RM, Dunn MJ, Dubowitz V. Nature of the mononuclear infiltrate and the mechanism of muscle damage in juvenile dermatomyositis and Duchenne muscular dystrophy. J Neurol Sci 1990;99:199-217
34. Arahata K, Engel AG. Monoclonal antibody analysis of mononuclear cells in myopathies. IV: Cell-mediated cytotoxicity and muscle fiber necrosis. Ann Neurol 1988;23:168-73