Myoblast transplantation for inherited myopathies: a clinical approach

Bibliography

• PARTRIDGE TA, GROUNDS M, SLOPER JC: Evidence of fusion between host and donor myoblasts in skeletal muscle grafts. Nature (1978) 273:306–308.
   PubMed Web of Science ®Google Scholar
• JEPPESEN J, GREEN A,STEFFENSEN BE RAHBEK J: The Duchenne muscular dystrophy population in Denmark, 1977-2001: prevalence, incidence and survival in relation to theintroduction of ventilator use. Neureinuscul. Diserd. (2003) 13:804–812.
   PubMed Web of Science ®Google Scholar
• LIPTON BH, SCHULTZ E: Developmental fate of skeletal muscle satellite cells. Science (1979) 205:1292–1294.
   PubMed Web of Science ®Google Scholar
• •This pioneer paper (one of the first two publications on MT) initially described that following intramuscular injection, donor myoblasts either fuse together to form new small myofibres or fuse with the recipient's myofibres to be incorporated in large myofibres.
   Google Scholar
• 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. Neurel. Sci. (1982) 57:319–331.
   PubMed Web of Science ®Google Scholar
• •Another pioneer paper on MT, which demonstrated that the expression of donor and host proteins coexist in skeletal muscles of mice following MT.
   Google Scholar
• PARTRIDGE TA, MORGAN JE,COULTON GR, HOFFMAN EP,KUNKEL LM: Conversion of mdxmyofibres from dystrophin-negative to-positive by injection of normal myoblasts. Nature (1989) 337:176–179.
   PubMed Web of Science ®Google Scholar
• ••A seminal paper establishing theprinciple of cell transplantation as a means of introducing dystrophin into dystrophic muscle. This paper also identified some of the main problems of myoblast transplantation.
   Google Scholar
• SKUK D, ROY B, GOULET M et al.: Dystrophin expression in myofibers of Duchenne muscular dystrophy patients following intramuscular injections of normal myogenic cells. Ma The]: (2004) 9:475–482.
   PubMed Web of Science ®Google Scholar
• ••This report was the first demonstration inhumans that normal MT can systematically induce dystrophin expression in significant percentages of myofibres in DMD patients, if an appropriate donor cell delivery and immunosuppression are used.
   Google Scholar
• MENDELL JR, KISSEL JT, AMATO AA et al.: Myoblast transfer in the treatment of Duchenne's muscular dystrophy. N Engl. J. Med. (1995) 333:832–838.
   PubMed Web of Science ®Google Scholar
• PAVLATH GK, RICH K, WEBSTER SG, BLAU HM: Localization of muscle gene products in nuclear domains. Nature (1989) 337:570–573.
   PubMed Web of Science ®Google Scholar
• •This study determined that proteins coded by a single nucleus in a myofibre are expressed throughout a limited region of the syncytium, which is called the 'nuclear domain'. This fmding implies that the expression of donor proteins in a myofibre, following the fusion of a donor myoblast, is restricted to a limited region and not to the entire sarcolemma.
   Google Scholar
• RALSTON E, HALL ZW: Restricteddistribution of mRNA produced from a single nucleus in hybrid myotubes. J. Cell Biol. (1992) 119:1063–1068.
   PubMed Web of Science ®Google Scholar
• •A study that determined the short diffusion of the mRNA produced in the nucleus of a myofibre, which was estimated to be 1001.1m.
   Google Scholar
• HALL ZW, RALSTON E: Nuclear domains in muscle cells. Cell (1989) 59:771–772.
   PubMed Web of Science ®Google Scholar
• KARPATI G, POULIOT Y, ZUBRZYCKA-GAARN E et al.: Dystrophin is expressed in mdx skeletal muscle fibers after normal myoblast implantation. Am. Pathol (1989) 135:27–32.
   PubMed Web of Science ®Google Scholar
• WERNIG A, IRINTCHEV A, LANGE G: Functional effects of myoblast implantation into histoincompatible mice with orwithout immunosuppression. I Physiol (Lond) (1995) 484:493–504.
   PubMed Web of Science ®Google Scholar
• •A detailed study on the potential of MT to restore muscular mass in mice following acute irreversible damage, with special interest in immunological aspects.
   Google Scholar
• IRINTCHEV A, LANGER M,ZWEYER M, THEISEN R, WERNIG A: Functional improvement of damaged adult mouse muscle by implantation of primary myoblasts. Physiol. (Lond) (1997) 500:775–785.
   Google Scholar
• •A study on the potential of MT to restore muscular mass in mice following acute irreversible damage.
   Google Scholar
• ALAMEDDINE HS, LOUBOUTIN JP, DEHAUPAS M, SEBILLE A,FARDEAU M: Functional recovery induced by satellite cell grafts in irreversibly injured muscles. Cell Transplant. (1994) 3:3–14.
   PubMed Web of Science ®Google Scholar
• •One of the first studies on mice demonstrating that MT can restore mass and force in acutely damaged skeletal muscles in which endogenous regeneration was inhibited.
   Google Scholar
• WERNIG A, ZWEYER M, IRINTCHEV A: Function of skeletal muscle tissue formed after myoblast transplantation into irradiated mouse muscles. J. Physic] (Lond) (2000) 522:333–345.
   PubMed Web of Science ®Google Scholar
• YAO SN, KURACHI K: Implanted myoblasts not only fuse with myofibers but also survive as muscle precursor cells. J. Cell Sci. (1993) 105:957–963.
   PubMed Web of Science ®Google Scholar
• •The first study to demonstrate that mice donor myoblasts can remain as dormant mononuclear muscle-precursor cells in the host muscle following MT. This fmding implies that, if the same could be obtained in humans, clinical MT could provide a source of normal myogenic cells able to participate later in hypertrophy and muscle regeneration.
   Google Scholar
• GROSS JG, MORGAN JE: Muscleprecursor cells injected into irradiated mdxmouse muscle persist after serial injury. Muscle Nerve (1999) 22:174–185.
   PubMed Web of Science ®Google Scholar
• •This study provided further evidence that donor myoblasts can remain as mononuclear muscle-precursor cells in the host muscle following MT in mice.
   Google Scholar
• HESLOP L, BEAUCHAMP JR,TAJBAKHSH S, BUCKINGHAM ME, PARTRIDGE TA, ZAMMIT PS: Transplanted primary neonatal myoblasts can give rise to functional satellite cells asidentified using the Myf5(nlacZ1+) mouse. Gene The]: (2001) 8:778–783.
   PubMed Web of Science ®Google Scholar
• ••This paper demonstrated for the first timethat at least some of the donor myoblasts, which remain as mononudeated precursor cells in host muscles, stay specifically in the form of satellite cells. If this is confirmed in humans, it implies that MT could not induce the expression ofnormal proteins in the myofibres of myopathic patients, but could restore a pool of functional satellite cells (at this time normal).
   Google Scholar
• MAURO A: Satellite cell of skeletal musclefibers. J. Biophys. Biochetn. Cytol. (1961) 9:493–495.
   PubMed Web of Science ®Google Scholar
• •A landmark paper that reported the discovery of satellite cells.
   Google Scholar
• LEWIS MR: Rhythmical contraction of the skeletal muscle tissue observed in tissue cultures. Am. J. Physic] (1915) 38:153–161.
   Google Scholar
• KONIGSBERG IR: The differentiation of cross-striated myofibrils in short term cell culture. Exp. Cell Res. (1960) 21:414–420.
   PubMed Web of Science ®Google Scholar
• KINOSHITA I, ROY R, DUGRE FJ et al.: Myoblast transplantation in monkeys: control of immune response by FK506. Neuropathol Exp. Neurol (1996) 55:687–697.
   PubMed Web of Science ®Google Scholar
• •This was the first approach to the study of the acute rejection response following MT in non-human primates.
   Google Scholar
• KINOSHITA I, VILQUIN JT,GRAVEL C, ROY R, TREMBLAY JP: Myoblast allotransplantation in primates [letter]. Muscle Nerve (1995) 18:1217–1218.
   PubMed Web of Science ®Google Scholar
• •This was the first preclinical study of MT in non-human primates, which demonstrated that hybrid myofibres can be obtained in this model following MT under tacrolimus immunosuppression.
   Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• •This paper initially demonstrated that it was possible to obtain a high performance of MT in the muscles of a primate, thus indicating no inherent difference between the results of mouse MT and human MT, if similar parameters are applied.
   Google Scholar
• 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) 175:112–126.
   PubMed Web of Science ®Google Scholar
• •This paper showed the possibility to reach high percentages of hybrid myofibres in the skeletal muscles of non-human primates following the intramuscular implantation of myoblasts, without myotoxins. It was the first paper to establish a quantitative parameter in order to make MT a technique to provide reproducible results in large muscles of primates.
   Google Scholar
• SKUK D, GOULET M, ROY B, TREMBLAY JP: Myoblast transplantation in whole muscle of nonhuman primates. Neuropathol Exp. Neurol (2000) 59:197–206.
   PubMed Web of Science ®Google Scholar
• ••Through this study, it is shown that MTcan be successfully carried out throughout large-limb muscles in primates if a method of HDI is used. It also demonstrates that muscles tolerate this treatment well, in spite of the initial inflammatory reaction, and that muscle damage, if maintained under some limits, did not cause the systemic toxicity of a rhabdomyolysis. Finally, the beneficial properties of tacrolimus as an immunosuppressant to control acute rejection in MT, while preserving the success of the graft, were confirmed in experiments of up to 1 year.
   Google Scholar
• DEASY BM, JANKOWSKI RJ, HUARD J: Muscle-derived stem cells: characterization and potential for cell- mediated therapy. Blood Cells MM. Dis. (2001) 27:924–933.
   PubMed Web of Science ®Google Scholar
• QU-PETERSEN Z, DEASY B, JANKOWSKI R et al.: Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration. Cell Biol. (2002) 157:851–864.
   PubMed Web of Science ®Google Scholar
• MUELLER GM, O& DAY T,WATCHKO JF, ONTELL M: Effect of injecting primary myoblasts versus putative muscle-derived stem cells on mass and force generation in mdx mice. Hum. Gene Thec (2002) 13:1081–1090.
   PubMed Web of Science ®Google Scholar
• WERNIG A, IRINTCHEV A, HARTLING A, STEPHAN G, ZIMMERMANN K, STARZINSKI-POWITZ A: Formation of new muscle fibres and tumours after injection of cultured myogenic cells. Neurocytol (1991) 20:982–997.
   PubMedGoogle Scholar
• FRESHNEY RI: Culture of Animal Cells. A Manual of Basic Technique. Freshney RI (Ed.), Alan R Liss, Inc., New York, USA (1987).
   Google Scholar
• TREMBLAY JP, ROY B, GOULET M: Human myoblast transplantation: a simple assay for tumorigenicity. Neuromuscul Disord. (1991) 1:341–343.
   PubMedGoogle Scholar
• GRIGORIADIS AE, HEERSCHE JN, AUBIN JE: Differentiation of muscle, fat, cartilage, and bone from progenitor cells present in a bone-derived clonal cell population: effect of dexamethasone. Cell Biol. (1988) 106:2139–2151.
   PubMed Web of Science ®Google Scholar
• WAKITANI S, SAITO T, CAPLAN Al: Myogenic cells derived from rat bone marrow mesenchymal stem cells exposed to 5-azacytidine. Muscle Nerve (1995) 18:1417–1426.
   PubMed Web of Science ®Google Scholar
• FERRARI G, CUSELLA-DE ANGELIS G, COLETTA M et al.: Muscle regeneration by bone marrow-derived myogenic progenitors. Science (1998) 279:1528–1530.
   PubMed Web of Science ®Google Scholar
• GUSSONI E, SONEOKA Y, STRICKLAND CD et al: Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature (1999) 401:390–394.
   PubMed Web of Science ®Google Scholar
• GUSSONI E, BENNETT RR, MUSKIEWICZ KR et al.: Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation. J. Gin. Invest. (2002) 110:807–814.
   PubMed Web of Science ®Google Scholar
• CAMARGO FD, GREEN R, CAPETENAKI Y, JACKSON KA, GOODELL MA: Single hematopoietic stem cells generate skeletal muscle through myeloid intermediates. Nat. Med. (2003) 9:1520–1527.
   PubMed Web of Science ®Google Scholar
• ••An interesting study that raised thepossibility that the rare fusion of circulating bone marrow-derived cells with myofibres should be carried out by the occasional recruitment of a macrophage in the fusiogenic process during myofibre regeneration. The rationality of this study comes from the fact that macrophages are cells that have the capacity to fuse, and that they densely infiltrate necrosed myofibres, participating actively in the process of muscle regeneration.
   Google Scholar
• HUARD C, MOISSET PA, DICAIRE A et al.: Transplantation of dermal fibroblasts expressing MyoD1 in mouse muscles. Biochem. Biophys. Res. Commun. (1998) 248:648–654.
   PubMed Web of Science ®Google Scholar
• GOLDRING K, JONES GE, SEWRY CA, WATT DJ: The muscle-specific marker desmin is expressed in a proportion of human dermal fibroblasts after theirexposure to galectin-1. Neuromuscul Disord. (2002) 12:183–186.
   PubMed Web of Science ®Google Scholar
• LANDON DN: Skeletal muscle: normal morphology, development and innervation. In: Skeletal Muscle Pathology Mastaglia FL, Walton JN (Eds), Churchill Livingstone, Edinburgh, UK (1982):1–87.
   Google Scholar
• PARTRIDGE TA: Invited review: myoblast transfer: a possible therapy for inherited myopathies? Muscle Nerve (1991) 14:197–212.
   PubMed Web of Science ®Google Scholar
• ••Probably the best review on MT duringthe beginning of this technique.
   Google Scholar
• NEUMEYER AM, DIGREGORIO DM, BROWN RH JR: Arterial delivery of myoblasts to skeletal muscle. Neurology (1992) 42:2258–2262.
   PubMed Web of Science ®Google Scholar
• TORRENTE Y, TREMBLAY JP, PISATI F et al.: Intraarterial injection of muscle-derived CD34+Sca-1+ stem cells restores dystrophin in mdx mice. .1 Cell Biol. (2001) 152:335–348.
   Google Scholar
• SAMPAOLESI M, TORRENTE Y, INNOCENZI A et al.: Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science (2003) 301:487–492.
   PubMed Web of Science ®Google Scholar
• MILLER RG, SHARMA KR, PAVLATH GK et al.: Myoblast implantation in Duchenne muscular dystrophy: the San Francisco study. Muscle Nerve (1997) 20:469–478.
   PubMed Web of Science ®Google Scholar
• NEUMEYER AM, CROS D,MCKENNA-YASEK D et al.: Pilot study of myoblast transfer in the treatment of Becker muscular dystrophy. Neurology (1998) 51:589–592.
   Google Scholar
• 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) 5:291–295.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• TREMBLAY JP, BOUCHARD JP, MALOUIN F et al.: Myoblast transplantation between monozygotic twin girl carriers of Duchenne muscular dystrophy. Neuron/use& Disord. (1993) 3:583–592.
   PubMedGoogle Scholar
• KARPATI G, JOHNSTON W, AJDUKOVIC G et al: Myoblast transfer (MT) in McArdle's disease (McD). Neurology (1992) 42\(Suppl. 3):387.
   Google Scholar
• KARPATI G, AJDUKOVIC D, ARNOLD D et al.: Myoblast transfer in Duchenne muscular dystrophy. Ann. Neurol (1993) 34:8–17.
   PubMed Web of Science ®Google Scholar
• HUARD J, BOUCHARD JP, ROY R et al:Human myoblast transplantation: preliminary results of 4 cases. Muscle Nerve (1992) 15:550–560.
   PubMed Web of Science ®Google Scholar
• GUSSONI E, PAVLATH GK,LANCTOT AM et al.: Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature (1992) 356:435–438.
   Google Scholar
• WARD MM: Factors predictive of acute renal failure in rhabdomyolysis. Arch. Intern. Med. (1988) 148:1553–1557.
   PubMed Web of Science ®Google Scholar
• EL FAHIME E, TORRENTE Y, CARON NJ, BRES OLIN MD, TREMBLAY JP: ha vivo migration of transplanted myoblasts requires matrix metalloproteinase activity. Exp. Cell Res. (2000) 258:279–287.
   PubMed Web of Science ®Google Scholar
• EL FAHIME E, MILLS P,LAFRENIERE JF, TORRENTE Y, TREMBLAY JP: The urokinase plasminogen activator: an interesting way to improve myoblast migration following their transplantation. Exp. Cell Res. (2002) 280:169–178.
   PubMed Web of Science ®Google Scholar
• ITO H, HALLAUER PL, HASTINGS KE,TREMBLAY JP: Prior culture with concanavalin A increases intramuscular migration of transplanted myoblast. Muscle Nerve (1998) 21:291–297.
   PubMed Web of Science ®Google Scholar
• CARON NJ, ASSELIN I, MOREL G, TREMBLAY JP: Increased myogenic potential and fusion of matrilysin-expressing myoblasts transplanted in mice. Cell Transplant. (1999) 8:465–476.
   PubMed Web of Science ®Google Scholar
• HUARD J, VERREAULT S, ROY R, TREMBLAY M, TREMBLAY JP: High efficiency of muscle regeneration after human myoblast clone transplantation in SCID mice. J. Gin. Invest. (1994) 93:586–599.
   PubMed Web of Science ®Google Scholar
• KINOSHITA I, VILQUIN JT, GUERETTE B, ASSELIN I, ROY R, TREMBLAY JP: Very efficient myoblast allotransplantation in mice under FK506 immunosuppression. Muscle Nerve (1994) 17:1407–1415.
   PubMed Web of Science ®Google Scholar
• •This paper reported the best results obtained with MT in dystrophic mice,attributed to the use of tacrolirnus for immunosuppression.
   Google Scholar
• VILQUIN JT, ASSELIN I, GUERETTE B,KINOSHITA I, ROY R, TREMBLAY JP: Successful myoblast allotransplantation in mdx mice using rapamycin. Transplantation (1995) 59:422–426.
   PubMed Web of Science ®Google Scholar
• •The benefits of raparnycin as anirnmunosuppressant for MT in dystrophic mice are reported in this paper. This was the only MT study in which raparnycin was used.
   Google Scholar
• SKUK D, FURLING D, BOUCHARD JP et al.: Transplantation of human myoblasts in SCID mice as a potential muscular model for myotonic dystrophy. J. Neuropathol Exp. Neurol (1999) 58:921–931.
   PubMed Web of Science ®Google Scholar
• PIN CL, MERRIFIELD PA: Developmental potential of rat L6 myoblasts in vivo following injection into regenerating muscles. Dev. Biol. (1997) 188:147–166.
   PubMed Web of Science ®Google Scholar
• CANTINI M, MASSIMINO ML, CATANI C, RIZZUTO R, BRINI M, CARRARO U: Gene transfer into satellite cell from regenerating muscle: bupivacaine allows beta-Gal transfection and expression in vitro and in vivo. In Vitro Cell. Dev. Biol. Amin. (1994) 30A:131–133.
   Web of Science ®Google Scholar
• MORGAN JE, HOFFMAN EP, PARTRIDGE TA: Normal myogenic cells from newborn mice restore normal histology to degenerating muscles of the mdx mouse. J. Cell Biol. (1990) 111:2437–2449.
   PubMed Web of Science ®Google Scholar
• ••This study demonstrated that normalMT can improve the histology in the skeletal muscles of dystrophic mice, in which muscle degeneration was accelerated by pre-irradiation.
   Google Scholar
• KINOSHITA I, VILQUIN JT, ASSELIN I, CHAMBERLAIN J, TREMBLAY JP: Transplantation of myoblasts from a transgenic mouse overexpressing dystrophin produced only a relatively small increase of dystrophin-positive membrane. Muscle Nerve (1998) 21:91–103.
   PubMed Web of Science ®Google Scholar
• ZVIBEL I, SMETS F, SORIANO H: Anoikis: roadblock to cell transplantation? Cell Transplant. (2002) 11:621–630.
   Google Scholar
• BEAUCHAMP JR, MORGAN JE, PAGEL CN, PARTRIDGE TA: Dynamics of myoblast transplantation reveal a discrete minority of precursors with stem cell-like properties as the myogenic source. J. Cell Biol. (1999) 144:1113–1122.
   PubMed Web of Science ®Google Scholar
• ••This paper established a good method toanalyse early donor cell survival followingMT, discriminating donor cell death from donor cell differentiation. In fact, it was the first observation that both phenomena develop simultaneously during the first post-transplantation days in such a way that cell proliferation compensates cell death, therefore allowing good transplantation results.
   Google Scholar
• SKUK D, CARON NJ, GOULET M, ROY B, TREMBLAY JP: Resetting the problem of cell death following muscle-derived cell transplantation: detection, dynamics and mechanisms. J. Neuropathol Exp. Neurol (2003) 62:951–967.
   PubMed Web of Science ®Google Scholar
• •Evidence of apoptosis as a mechanism of early donor cell death is shown for the first time in this paper.
   Google Scholar
• GUERETTE B, SKUK D, CELESTIN F et al.: Prevention by anti-LFA-1 of acute myoblast death following transplantation. J. _lulu-ulna (1997) 159:2522–2531.
   Google Scholar
• SAMMELS LM, BOSIO E, FRAGALL CT, GROUNDS MD, VAN ROOIJEN N, BEILHARZ MW: Innate inflammatory cells are not responsible for early death of donor myoblasts after myoblast transfer therapy. Transplantation (2004) 77:1790–1797.
   PubMed Web of Science ®Google Scholar
• COUSINS JC, WOODWARD KJ, GROSS JG, PARTRIDGE TA,MORGAN JE: Regeneration of skeletal muscle from transplanted immortalised myoblasts is oligoclonal. J. Cell Sci. (2004) 117:3259–3269.
   PubMed Web of Science ®Google Scholar
• JONES PH: Implantation of culturedregenerate muscle cells into adult rat muscle. Exp. Neurol (1979) 66:602–610.
   PubMed Web of Science ®Google Scholar
• •This pioneer paper was one of the first publications on MT, and it described for the first time the acute rejection in this model.
   Google Scholar
• GUERETTE B, ASSELIN I, VILQUIN JT,ROY R, TREMBLAY JP: Lymphocyte infiltration following allo- and xenomyoblast transplantation in mdx mice. Muscle Nerve (1995) 18:39–51.
   PubMed Web of Science ®Google Scholar
• •This was one of the first characterisations of the specific immune cell reactions following myoblast allotransplantation in mice.
   Google Scholar
• IRINTCHEV A, ZWEYER M, WERNIG A: Cellular and molecular reactions in mouse muscles after myoblast implantation. J. Neurocytol (1995) 24:319–331.
   PubMedGoogle Scholar
• •This was one of the first characterisations of the specific immune cell reactions following myoblast allotransplantation in mice.
   Google Scholar
• WERNIG A, IRINTCHEV A: 'Bystander'damage of host muscle caused by implantation of MHC- compatible myogenic cells. J. Neurol Sci. (1995) 130:190–196.
   PubMed Web of Science ®Google Scholar
• GUERETTE B, ROY R, TREMBLAY M et al: Increased granzyme B mRNA after alloincompatible myoblast transplantation. Transplantation (1995) 60:1011–1016.
   PubMed Web of Science ®Google Scholar
• GUERETTE B, TREMBLAY G, VILQUIN JT et al.: Increased interferon-gamma mRNA expression following alloincompatible myoblast transplantation is inhibited by FK506. Muscle Nerve (1996) 19:829–835.
   PubMed Web of Science ®Google Scholar
• CAMIRAND G, CARON NJ, ASSELIN I, TREMBLAY JP: Combined immunosuppression of mycophenolate mofetil and FK506 for myoblast transplantation in mdx mice. Transplantation (2001) 72:38–44.
   PubMed Web of Science ®Google Scholar
• HARDIMAN 0, SKLAR RM,BROWN RH JR: Direct effects of cyclosporin A and cyclophosphamide on differentiation of normal human myoblasts in culture. Neurology (1993) 43:1432–1434.
   PubMed Web of Science ®Google Scholar
• HONG F, LEE J, SONG JVV et al.: Cyclosporin A blocks muscle differentiation by inducing oxidative stress and inhibiting the peptidyl-prolyl-cis-trans isomerase activity of cyclophilin A: cyclophilin A protects myoblasts from cyclosporin A-induced cytotoxicity. FASEB (2002) 16:1633–1635.
   PubMed Web of Science ®Google Scholar
• IRINTCHEV A, ZWEYER M,COOPER RN, BUTLER-BROWNE GS, WERNIG A: Contractile properties, structure and fiber phenotype of intact and regenerating slow-twitch muscles of mice treated with cyclosporin A. Cell Tissue Res. (2002) 308:143–156.
   PubMed Web of Science ®Google Scholar
• 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.
   Google Scholar
• SEMSARIAN C, WU MJ, JU YK et al.: Skeletal muscle hypertrophy is mediated by a Ca2+-dependent calcineurin signalling pathway. Nature (1999) 400:576–581.
   PubMed Web of Science ®Google Scholar
• •This paper raised the possibility that tacrolimus-based immunosuppression could inhibit the mechanism of hypertrophy in skeletal muscles by describing how calcineurin plays a role in the mechanism of skeletal muscle hypertrophy.
   Google Scholar
• VILQUIN JT, KINOSHITA I, ROY R, TREMBLAY JP: Cyclophosphamide immunosuppression does not permit successful myoblast allotransplantation in mouse. Neuromuscul Disord. (1995) 5:511–517.
   PubMed Web of Science ®Google Scholar
• VILQUIN JT, KINOSHITA I, ROY B et al.: Partial laminin alpha2 chain restoration in alpha2 chain-deficient dy/dy mouse by primary muscle cell culture transplantation. J. Cell Biol. (1996) 133:185–197.
   PubMed Web of Science ®Google Scholar
• •The first demonstration in mice of the potential of MT to treat congenital muscular dystrophy (another myopathy).
   Google Scholar
• WATT DJ, MORGAN JE,PARTRIDGE TA: Long term survival of allografted muscle precursor cells following a limited period of treatment with cyclosporin A. Clin. Exp. Immunol (1984) 55:419–426.
   PubMed Web of Science ®Google Scholar
• BRAZELTON TR, MORRIS RE: Molecular mechanisms of action of new xenobiotic immunosuppressive drugs: tacrolimus (FK506), sirolimus (rapamycin), mycophenolate mofetil and leflunomide. Curr. Opin. Immunol (1996) 8:710–720.
   PubMed Web of Science ®Google Scholar
• GUMMERT JF, IKONEN T,MORRIS RE: Newer immunosuppressive drugs: a review. J. Am. Soc. Nephrol (1999) 10:1366–1380.
   PubMed Web of Science ®Google Scholar
• PAVLATH GK, RANDO TA, BLAU HM: Transient immunosuppressive treatment leads to long-term retention of allogeneic myoblasts in hybrid myofibers. J. Cell Biol. (1994) 127:1923–1932.
   PubMed Web of Science ®Google Scholar
• CAMIRAND G, CARON NJ, TURGEON NA, ROSSINI AA, TREMBLAY JP: Treatment with anti-CD154 antibody and donor-specific transfusion prevents acute rejection of myoblast transplantation. Transplantation (2002) 73:453–461.
   PubMed Web of Science ®Google Scholar
• CAMIRAND G, ROUSSEAU J, DUCHARME ME, ROTHSTEIN DM, TREMBLAY JP: Novel Duchenne muscular dystrophy treatment through myoblast transplantation tolerance with anti-CD45RB, anti-CD154 and mixed chimerism. Am. J. Transplant. (2004) 4:1255–1265.
   PubMed Web of Science ®Google Scholar
• GILL RG, WOLF L: Immunobiology of cellular transplantation. Cell Transplant. (1995) 4:361–370.
   PubMed Web of Science ®Google Scholar
• WIENDL H, MITSDOERFFER M, HOFMEISTER V et al.: The non-classical MHC molecule HLA-G protects human muscle cells from immune-mediated lysis:implications for myoblast transplantation and gene therapy. Brain (2003) 126:176-185.An interesting in vitro study that proposed an innovative method to protect the transplanted myoblasts from acute rejection, by way of expressing HLA-G, a non-classical MHC class I molecule, which is a key mediator in maternal tolerance to the fetus.
   Google Scholar
• FLOYD SS JR, CLEMENS PR,ONTELL MR et al.: Ex vivo gene transferusing adenovirus-mediated full-length dystrophin delivery to dystrophic muscles. Gene Ther. (1998) 5:19–30.
   Google Scholar
• BUJOLD M, CARON N, CAMIRAN G et al.: Autotransplantation in mdx mice of mdx myoblasts genetically corrected by anHSV-1 amplicon vector. Cell Transplant. (2002) 11:759–767.
   PubMed Web of Science ®Google Scholar
• MOISSET PA, SKUK D, ASSELIN I et al: Successful transplantation of genetically corrected DMD myoblasts following ex vivo transduction with the dystrophin minigene. Biochem. Biophys. Res. Commun. (1998) 247:94–99.
   PubMed Web of Science ®Google Scholar
• •This paper showed that it is possible to genetically correct human DMD myoblasts in vitro, being grafted back (in this case in SCID mice) to form human myofibres expressing dystrophin.
   Google Scholar
• MOISSET PA, GAGNON Y, KARPATI G, TREMBLAY JP: Expression of human dystrophin following the transplantation of genetically modified mdx myoblasts. Gene Ther. (1998) 5:1340–1346.
   PubMed Web of Science ®Google Scholar
• VILQUIN JT, WAGNER E,KINOSHITA I, ROY R, TREMBLAY JP:Successful histocompatible myoblast transplantation in dystrophin- deficient mdx mouse despite the production of antibodies against dystrophin. J. Cell Biol. (1995) 131:975–988.
   PubMed Web of Science ®Google Scholar
• •This was the first study on the potential immunogenicity of dystrophin alone, in the case of being expressed in dystrophin-deficient recipients.
   Google Scholar
• OHTSUKA Y, UDAKA K, YAMASHIRO Y, YAGITA H, OKUMURA K: Dystrophin acts as a transplantation rejection antigen in dystrophin-deficient mice: implication for gene therapy. bronunol. (1998) 160:4635–4640.
   Google Scholar
• •This study defined some epitopes of dystrophin that could be the most antigenic in mice, raising the possibility that they led to a slow T lymphocyte reaction.
   Google Scholar