Problems and solutions in myoblast transfer therapy

Journal of Cellular and Molecular Medicine

Volumn 5, Issue 1, 2001, Pages 33-47

  Smythe, Gayle M ^a   Hodgetts, Stuart I ^a   Grounds, Miranda D ^b  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

Duchenne muscular dystrophy; Dystrophin; Dystrophin gene; Gene therapy; Myoblast; Myoblast transfer therapy; Stem cells

Indexed keywords

ANIMAL; CELL DIVISION; CELL FUSION; CELL MOTION; CELL TRANSPLANTATION; CYTOLOGY; DUCHENNE MUSCULAR DYSTROPHY; GRAFT REJECTION; HISTOCOMPATIBILITY; HUMAN; IMMUNOLOGY; MUSCLE; PATHOLOGY; REVIEW; TRANSPLANTATION;

ANIMALS; CELL DIVISION; CELL FUSION; CELL MOVEMENT; CELL TRANSPLANTATION; HISTOCOMPATIBILITY; HOST VS GRAFT REACTION; HUMANS; MUSCLES; MUSCULAR DYSTROPHY, DUCHENNE;

EID: 0035074378     PISSN: 15821838     EISSN: None     Source Type: Journal    
DOI: 10.1111/j.1582-4934.2001.tb00136.x     Document Type: Article

References (93)

1. Hoffman E.P., Brown R.H., Kunkel L.M., Dystrophin: the protein product of the Duchenne muscular dystrophy locus, Cell, 51: 919, 1987
2. Koenig M., Hoffman E.P., Bertelson C.J., Monaco A.P., Feener C., Kunkel L.M., Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals, Cell, 50: 509, 1987
3. Boniila E., Samitt C.E., Miranda A.F., Hays A.P., Salviati GL, DiMauro S., Kunkel L.M., Hoffman E.P., Rowland L.P., Duchenne muscular dystrophy: deficiency of dystrophin at the muscle cell surface, Cell, 54: 447, 1998
4. Tremblay J.P., Guerette B., Myoblast transplantation: a brief review of the problems and some solutions, Basic Appl. Myol, 7: 221, 1997
5. Law P.K., Goodwill T.G., Fang Q., Duggirala V., Larkin C., Florendo J.A., Kirby D.S., Deering M.B., Li HJ., Chen M., Yoo T.J., Cornett J., Li L.M., Shirzad A., Quinley T., Holcomb R.L. Feasibility, safety, and efficacy of myoblast transfer therapy on Duchenne muscular dystrophy boys, Cell Transplant., 1: 235, 1992
6. Partridge T., Beauchamp J.R., Morgan J., Tremblay J.P., Huard J., Watt D., Wernig A., Irintchev A., Grounds M., Springer M.L., Bartlett R.J., Mendell J., Vilquin J.T., Bower J.J., Letter to the editor, Cell Transplant., 6: 195, 1997
7. Fan Y., Maley M., Beilharz M., Grounds M., Rapid death of injected myoblasts in myoblast transfer therapy, Muscle Nerve, 19: 853, 1996
8. Beauchamp J.R., Pagel C.N., Partridge T.A., A dual-marker system for quantitative studies of myoblast transplantation in the mouse, Transplant., 63: 1794, 1997
9. Qu Z., Balkir L., van Deutekom J., Robbins P., Pruchnic R., Huard J., Development of approaches to improve cell survival in myoblast transfer therapy, J.CellBioL, 142: 1257, 1998
10. Hodgetts S.I., Beilharz M.W., Scalzo T., Grounds M.D., Why do cultured transplanted myoblasts die in vivo! DNA quantification shows enhanced survival of donor myoblasts in host mice depleted of CD4+/CD8+ or NK1.1+ cells, Cell Transplant., 9: 489, 2000
11. Smythe G.M., Hodgetts S.I., Grounds M.D., Immunobiology and the future of. myoblast transfer therapy, Mol. Then, 1: 304, 2000
12. Guerette B., Asselin L, Skuk D., Entman M., Tremblay J., Control of inflammatory damage by anti-LFA-1: Increased success of myoblast transplantation, Cell Transplant., 6: 101, 1997
13. Fan Y., Grounds M.D., Garlepp M.J., Beilharz M.W., Increased survival, movement and fusion of myoblasts from sliced muscle grafts into skeletal muscles of T-cell depleted and tolerised dystrophic host mice, Basic Appl. Myol., 7: 231, 1997
14. Smythe G.M., Fan Y., Grounds M.D., Enhanced migration and fusion of donor myoblasts in dystrophic and normal host muscle, Muscle Nerve, 23: 560, 2000
15. CobboId S., Adams E., Marshall S., Davies J., Waldmann H., Mechanisms of peripheral tolerance and suppression induced by monoclonal antibodies to CD4 and CDS, Immunol Rev., 149: 5, 1996
16. Guerette B., Gingras M., Wood K., Roy R., Tremblay J., Immunosuppression with monoclonal antibodies and CTLA4-Ig after myoblast transplantation in mice, Transplant., 62: 962, 1996
17. Beauchamp J.R., Abraham D.J., Bou-Gharios G, Partridge T.A., Olsen I., Expression and function of heterotypic adhesion molecules during differentiation of human skeletal muscle in culture, Am. J. Pathol., 140: 387, 1992
18. Pavlath GK., Rando T.A., Blau H.M., Transient immunosuppressive treatment leads to long-term retention of allogeneic myoblasts in hybrid myofibers, J. Cell BioL, 127: 1923, 1994
19. Pimorady-Esfahani A., Grounds M.D., McMenamin P.G, Macrophages and dendritic cells in normal and regenerating murine skeletal muscle, Muscle Nerve 20: 158, 1997
20. Banchereau J., Briere F., Caux C., Davoust J., Lebecque S., Liu Y.J., Pulend ran B., Palucka K., Immunobiology of dendritic cells, Anmi. Rev. ImimmoL, 18: 767, 2000
21. Doyle N.A., Bhagwan S.B., Meek B.B., Kutkoski GJ., Steeber D.A., Tedder T.R, Doerschhuk C.M., Neutrophil margination, sequestration, and emigration in the lungs of L-selectin-deficient mice, J. Clin. Invest., 99: 526, 1997
22. Gorospe J., Tharp M., Hinckley J., Kornegay J., Iloffman E.P., A role for mast cells in the progression of Duchenne muscular dystrophy: correlations in dystrophin-deficient humans, dogs, and mice, J. Neurol. Sci., 122: 44, 1994
23. Tidball J.G., Berchenko E., Frenette J., Macrophage invasion does not contribute to muscle membrane injury during inflammation, J. Lenk. BioL, 65: 192, 1999
24. Skuk D., Tremblay J.P., Complement deposition and cell death after myoblast transplantation, Cell Transplant., 7: 427, 1998
25. Hodgetts S.I., Grounds M.D., Complement and myoblast transfer therapy: Donor myoblast survival is enhanced following depletion of host complement C3 using cobra venom factor but not in the absence of C5, Immunol. Cell BioL, In press, 2001
26. Gasque P., Morgan B.P., Legoedec J., Chan P., Fontaine M., Human skeletal myoblasts spontaneously activate allogeneic complement but are resistant to killing, J. Immunol., 156: 3402, 1996
27. Vilquin J.T., Wagner E., Kinoshita I., Roy R., Tremblay J.P., Successful histocompatible myoblast transplantation in dystrophin-deficient mdx mouse despite the production of antibodies against dystrophin.J. Cell BioL, 131: 975, 1995
28. Tremblay J.P., Malouin F., Roy R., Huard J., Bouchard J.P., Satoh A., Richards C.L., Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy, Cell Transplant., 2: 99, 1993
29. Kong J., Anderson J.E., Dynamic restoration of dystrophin to dystrophin-deficient myotubes, Muscle Nerve, 24: 77, 2001
30. Partridge T., Lu Q.L., Morris G, Hoffman E., Is myoblast transplantation effective?, Nature Med., 4: 1208, 1998
31. Lu Q.L., Morris G.E., Wilton S.D., Ly T., Artem'yeva O.V., Strong P., Partridge T.A., Massive idiosyncratic exon skipping corrects thé nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion, J. Cell BioL, 148: 985, 2000
32. Boulanger A., Asselin L, Roy R., Tremblay J., Role of non-major histocompatibility complex antigens in the rejection of transplanted myoblasts, Transplant., 63: 893, 1997
33. Miller S., Ito H., Blau H., Torti F., Tumor necrosis factor inhibits human myogenesis in vitro, Mol. Cell. BioL, 8: 2295, 1998
34. Zamir O., Hasseigren P., Kunkel S., Frederick J., Higashiguchi T., Fischer J., Evidence that tumour necrosis factor participates in the regulation of muscle proteolysis during sepsis, Arch. Surg., 127: 170, 1992
35. Brunda M.J., Interleukin-12, J. Leuk. BioL, 55: 280, 1994
36. Irintchev A., Zweyer M., Wernig A., Cellular and molecular reactions in mouse muscles after myoblast implantation, J. NeurocytoL, 24: 319, 1995
37. De Rossi M., Bernasconi P., Baggi F., de Waal Malefyt R., Mantegazza R., Cytokines and chemokines are expressed by human myoblasts: possible relevance for the immune pathogenesis of muscle inflammation, Int. ImmiuwL, 12: 1329, 2000
38. Fan Y., Beilharz M.W., Grounds M.D., A potential alternative strategy for myoblast transfer therapy: the use of sliced muscle grafts, Cell Transplant., 5: 421, 1996
39. Smythe GM., Grounds M.D., Exposure to tissue culture conditions can adversely affect myoblast behavior in vivo in whole muscle grafts: implications for myoblast transfer therapy, Cell Transplant., 9: 379,2000
40. Irintchev A., Langer M., Zweyer M., Wernig A., Myoblast transplantation in the mouse: what cells do we use?, Basic Appl. Myol., 7: 161, 1997
41. Bower J., White J.D., Kurek J.B., Muldoon C.M., Austin L., The role of growth factors in myoblast transfer therapy, Basic Appl. Myol., 7: 177, 1997
42. Burkin D.J., Kaufman S.J., The alpha 7 beta 1 integrin in muscle development and disease, Cell Tiss. Res., 296: 183, 1999
43. Taverna D., Disatnik M-H., Rayburn H., Bronson R.T., Yang J., Rando T.A., Hynes R.O., Dystrophic muscle in mice chimeric for expression of α5 integrin.y. Cell Biol., 143: 849, 1998
44. Grounds M.D., McGeachie J.K., Davies M.J., Sorokin L., Maley M.A.L., The expression of extracellular matrix during adult skeletal muscle regeneration: how the basement membrane, interstitium, and myogenic cells collaborate, Basic Appl. Myol, 8: 129, 1998
45. Ringelmann B., Roder C., Hallmann R., Maley M., Davies M., Grounds M., Sorokin L., Expression of laminin al, o2, a4 and o5 chains, fibronectin and tenascin-C in skeletal muscle of dystrophic 129REJ dy/dy mice, Exp. Cell Res., 246: 165, 1999
46. Yokoyama W.M., Natural killer cell receptors, Curr. Opin. Cell Biol., 10: 298, 1998
47. Weindl H., Behrens L., Maier S., Johnson M.A., Weiss E.H., Hohlfeld R., Muscle fibers in inflammatory myopathies and cultured myoblasts express the non-classical major histocompatibility antigen HLA-G, Ann. Neurol., 48: 679,2000
48. Davis D.M., Chiu L, Fassett M., Cohen GB., Mandelboim O., Strominger J.L., The human natural killer cell immune synapse, Proc. Natl. Acad. Sci. USA., 96: 15062, 1999
49. Beauchamp J.R., Morgan J.E., Pagel C.N., Partridge T.A., Dynamics of myoblast transplantation reveal a discrete minority of precursors with stem cell-like properties as the myogenic source, J. Cell Biol., 144: 1113, 1999
50. Lee J.Y., Qu-Petersen Z., Cao B., Kimura S., Jankowski R., Cummins J., Usas A., Gates C., Robbins P., Wernig A., Huard J., Clonal isolation of muscle-derived cells capable of enhancing muscle regeneration and bone healing, J. CellBiol., 150:1085,2000
51. Beauchamp J., Heslop L., Yu D.S.W., Tajbakhsh S., Kelly R.G, Wernig A., Buckingham M.E., Partridge T.A., Zammit P.S., Expression of CD34 and myf5 defines the majority of quiescent adult skeletal muscle satellite cells, / CellBiol., 151: 1221,2000
52. Cossu G, Mavilio F., Myogenic stem cells for the therapy of primary myopathies: wishful thinking of therapeutic perspective?, J. Clin. Invest., 105: 1669,2000
53. Grounds M.D., Muscle regeneration: Molecular aspects and therapeutic implications, Curr. Opin. Neurol.. 12: 535, 1999
54. Grounds M.D., Davies M J., Chemotaxis in myogenesis, Basic Appl. Myol., 6:469, 1996
55. Bischoff R., Chemotaxis of skeletal muscle satellite cells, Dev. Dynam., 208: 505, 1997
56. Kinoshita L, Vilquin J., Roy R., Tremblay J., Successive injections in mdx mice of myoblasts grown with bFGF, Neuromnsc. Dlsord., 6: 187, 1996
57. Musaro A., McCulIagh K., Houghton L., Dobrowolny G, Molinaro M., Barton E.R., Sweeney H., Rosenthal N., Localized IGF-1 transgene expression sustains hypertrophy in senescent skeletal muscle, Nature Genetics, 27: 195,2001
58. Barton-Davis E.R., Shoturma D.I., Musaro A., Rosenthal N., Sweeney H.L., Viral mediated expression of insulin-like growth factor I blocks the agerelated loss of skeletal muscle function, Proc. Natl. Acad. Sci. USA., 95: 15603, 1998
59. Megeney L.A., Kablar B., Garrett K., Anderson J.E., Rudnicki M.A., MyoD is required for myogenic stem cell function in adult skeletal muscle, Genes Dev., 10: 1173, 1996
60. Yablonka-Reuveni Z., Rivera A.J., Rudnicki M.A., Primig M., Anderson J.E., Natanson P., The transition from proliferation to differentiation is delayed in satellite cells from mice lacking MyoD, Dev. Biol, 210: 440, 1999
61. White J.D., Scaffidi A., Davies M., McGeachie J., Rudnicki M.A., Grounds M.D., Myotube formation is delayed but not prevented in MyoD-deficient skeletal muscle: studies in regenerating whole muscle grafts of adult mice, J. Histochem. Cytochem., 48: 1531,2000
62. Ito H., Hallauer P.L., Hastings K.E.M., Tremblay J.P., Prior culture with concanavalin A increases intramuscular migration of transplanted myoblasts, Muscle Netve, 21: 291, 1998
63. Caron N J., Asselin I., Morel GL, Tremblay J.P., Increased myogenic potential and fusion of matrilysinexpressing myoblasts transplanted in mice, Cell Transplant., 8: 465, 1999
64. Torrente Y., El Fahime E., Caron N J., Bresolin N., Tremblay J.P., Intramuscular migration of myoblasts transplanted after muscle pretreatment with metalloproteinases, Cell Transplant., 9: 539, 2000
65. Karpati G., Zubrzj cka-Gaarn E.E., Carpenter S., Bulman D.E., Ray P.N., Worton R.G, Age-related conversion of dystrophin-negative to -positive fiber segments of skeletal but not cardiac muscle fibers in hétérozygote mdx mice, J. Nenropathol. Exp. Neurol., 49: 96, 1990
66. Chamberlain JS. Dystrophin levels required for genetic correction of Duchenne Muscular Dystrophy. Basic and Applied Myology, 7: 251,1997
67. Hauser M.A., Amalfltano A., Kumar-Singh R., Hauschka S.D., Chamberlain J.S., Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy, Neuromusc. Disord., 7: 277, 1997
68. Kinoshita I, Vilquin JT, Chamberlain J, Tremblay JP. Transplantation of myoblasts from a transgenic mouse overexpressing dystrophin produced only a relatively small increase of dystrophin-positive membrane. Muscle and Nerve, 21 (1): 91, 1998
69. Ahmad A., Brinson M., Hodges B.L., Chamberalain J.S., Amalfitano A., Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy, Hum. Mol. Gen. 12: 2507, 2000
70. Wang B., Li J., Xiao X., Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model, Proc. Natl. Acad. Sci. USA, 97: 13714, 2000
71. Howell J.M., Lochmuller H., O'Hara A., Fletcher S., Kakulas B.A., Massie B., Nalbantoglou J., Karpati G, High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: prolongation of expression with immunosuppression, Human Gene Ther., 9: 629, 1998
72. Yang L., Lochmuller H., Luo J., Massie B., Nalbantoglou J., Karpati G., Petrof B.J., Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (MDX) mice, Gene Ther., 5: 369, 1998
73. Moisset P.A., Gagnon Y., Karpati G., Tremblay J.P., Expression of human dystrophin following the transplantation of genetically modified mdx myoblasts, Gene Ther., 5: 1340, 1998
74. Moisset P.A., Skuk D., Asselin L, Goulet M., Roy B., Karpati G, Tremblay J.P., Successful transplantation of genetically corrected DMD myoblasts following ex vivo transduction with the dystrophin minigene, Biochem. Biophys. Res. Comm., 247: 94, 1998
75. Grounds M.D., Myoblast transfer therapy in the new millennium, Cell Transplant., 9: 485, 2000
76. Heslop L., Morgan J., Partridge T., Evidence for a myogenic stem cell that is exhausted in dystrophic muscle, J. Cell Sci., 113: 2299, 2000
77. Seigneurin-Venin S., Bernard V., Moisset P-A., Ouellette M.M., Mouly V., Di Donna S., Wright W.E., Tremblay J.P., Transplantation of normal and DMD myoblasts expressing the telomerase gene in SCID mice, Biochem. Biophys. Res. Comm., 272: 362, 2000
78. Partridge T., The 'fantastic voyage' of muscle progenitor cells, Nature Medicine, 4: 554, 1998
79. Mann C.J., Honeyman K., Cheng A.J., Ly T., Lloyd F., Fletcher S., Morgan J.E., Partridge T.A., Wilton S.D., Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse, Proc. Natl. Acad. Sci. USA, 98: 42, 2001
80. Barton-Davis E.R., Cordier L., Shoturma D.I., Leland S.E., Sweeney L.H., Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice, J. Clin. Invest., 104: 375, 1999
81. Blake D., Tinsley J., Davies K., Utrophin: a structural and functional comparison to dystrophin, Brain PathoL, 6: 37, 1996
82. Gilbert R., Nalbantoglou J., Tinsley J.M., Massie B., Davies K.E., Karpati G., Efficient utrophin expression following adenovirus gene transfer in dystrophic muscle, Biochem. Biophys. Res. Comm., 242: 244, 1998
83. Ebihara S., Guibinga G.H., Gilbert R., Nalbantoglou J., Massie B., Karpati G., Petrof B J., Differential effects of dystrophin and utrophin gene transfer in immunocompetent muscular dystrophy (mdx) mice, Physiol. Genomics, 3: 133, 2000
84. Faulkner J.A., Brooks S.V., The functional status of dystrophic muscles and functional recovery by skeletal muscles following myoblast transfer, Basic Appl. Myoi, 7: 257, 1997
85. Radojevic V., Lin S., Burgunder J-M., Differential expression of dystrophin, utrophin, and dystrophinassociated proteins in human muscle culture, Cell 7755. Res., 300: 447, 2000
86. Chaubourt E., Voisin V, Fossier P., Baux G., Israel M., De la Porte S., The NO way to increase muscular utrophin expression, CR Academy of Sciences III, 323: 735, 2000
87. Cannon J.G., Fiatarone M.A., Fielding R.A., Evans W.J., Aging and stress-induced changes in complement activation and neutrophil mobilization, J. Appl Physiol., 76: 2616, 1994
88. Ito H., Vilquin J-T,. Skuk D., Roy B., Goulet M., Lille S., Dugre F.J., Asselin L, Roy R., Fardeau M., Treniblay J.P., Myoblast transplantation in non-dystrophic dog, Neuromusc. Disord., 8: 95, 1998
89. Guerette B., Asselin L, Vilquin J., Roy R., Tremblay J., Lymphocyte infiltration following alloand xenomyoblast transplantation in mdx mice, Muscle Nerve, 18: 39, 1995
90. Emslie-Smith A., Arahata K., Engel A., Major histocompatibility complex class I antigen expression, immunolocalization of interferon subtypes, and T cell-mediated cytotoxicity in myopathies, Hum. Pathol., 20: 224, 1989
91. Spencer M.J., Montecino-Rodriguez E,. Dorshkind K., Tidball J.G., Helper (CD4+) and cytotoxic (CD8+) T cells promote the pathology of dystrophin-deficient muscle, Clin. Immunol., 98: 235,2001
92. Nahirney P., Dow P., Ovalle W., Quantitative morphology of mast cells in skeletal muscle of normal and genetically dystrophic mice, Anat. Rec., 247: 341, 1997
93. Cai B., Spencer M., Nakamura G., Tseng-Ong L., Tidball J., Eosinophilia of dystrophin-deficient muscle is promoted by perforin-mediated cytotoxicity by T cell effectors, Aw. J. Pathol., 156: 1789, 2000