Myoblast transplantion for inherited myopathies: A clinical approach

Expert Opinion on Biological Therapy

Volumn 4, Issue 12, 2004, Pages 1871-1885

  Skuk, Daniel ^a,b  

^a UNIVERSITÉ LAVAL   (Canada)

^b LAVAL UNIVERSITY   (Canada)

Author keywords

Clinical trial; Duchenne muscular dystrophy; Myoblast; Myopathy; Non human primates; Skeletal muscle

Indexed keywords

CELL SURVIVAL; CELL TRANSPLANTATION; CLINICAL TRIAL; DUCHENNE MUSCULAR DYSTROPHY; EXPERIMENTAL MODEL; GENETIC COMPLEMENTATION; GRAFT REJECTION; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INJECTION; MONKEY; MUSCLE ATROPHY; MYOBLAST; MYOFIBROSIS; MYOPATHY; NONHUMAN; REVIEW; SKELETAL MUSCLE; STEM CELL; TECHNIQUE; TISSUE ENGINEERING; TISSUE STRUCTURE; ANIMAL; COMPARATIVE STUDY; CYTOLOGY; METHODOLOGY; MUSCLE DISEASE; PATHOLOGY; STATISTICS; TRANSPLANTATION;

PRIMATES;

ANIMALS; CELL TRANSPLANTATION; CLINICAL TRIALS; HUMANS; MUSCLE, SKELETAL; MUSCULAR DISEASES; MYOBLASTS;

EID: 10344223978     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.12.1871     Document Type: Review

References (101)

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87. 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. The first demonstration in mice of the potential of MT to treat congenital muscular dystrophy (another myopathy).
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92. 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.
93. 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.
94. GILL RG, WOLF L: Immunobiology of cellular transplantation. Cell Transplant. (1995) 4:361-370.
95. 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-dassical MHC class I molecule, which is a key mediator in maternal tolerance to the fetus.
96. FLOYD SS JR, CLEMENS PR, ONTELL MR et al : Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles. Gene Ther. (1998) 5:19-30.
97. BUJOLD M, CARON N, CAMIRAN G et al.: Autotransplantation in mdx mice of mdx myoblasts genetically corrected by an HSV-1 amplicon vector. Cell Transplant. (2002) 11:759-767.
98. 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. This paper showed that it is possible to genetically correct human DMD myobtasts in vitro, being grafted back (in this case in SCID mice) to form human myofibtes expressing dystrophin.
99. 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.
100. 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. This was the first study on the potential immunogenicity of dystrophin alone, in the case of being expressed in dystrophin-deficient recipients.
101. 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. J. Immunol. (1998) 160:4635-4640. 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.