Correspondence Section: Myoblast transplantation produced dystrophin-positive muscle fibres in a 16-year-old patient with Duchenne muscular dystrophy

Huard, Johnny; Bouchard, Jean‐Pierre; Roy, Raynald; Labrecque, Claude; Dansereau, Guy; Lemieux, Bernard; Tremblay, Jacques P.

doi:10.1042/cs0810287

Cited by 61 publications

(34 citation statements)

References 4 publications

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“…Although researchers have extensively investigated various approaches to delivering dystrophin in dystrophic muscle (e.g., cell and gene therapy), no treatment is yet available to alleviate the muscle weakness associated with DMD. Transplantation of normal myoblasts into dystrophin-deficient muscle can create a reservoir of normal myoblasts that are capable of fusing with dystrophic muscle fibers and restoring dystrophin (Morgan et al, 1988(Morgan et al, , 1990(Morgan et al, , 1993Karpati et al, 1989;Huard et al, 1991Huard et al, , 1992aHuard et al, , b, 1994aPartridge, 1991;Gussoni et al, 1992Gussoni et al, , 1997Karpati and Worton, 1992;Morgan et al, 1993;Tremblay et al, 1993;Beauchamp et al, 1994Beauchamp et al, , 1999Kinoshita et al, 1994;Mendell et al, 1995;Vilquin et al, 1995;Fan et al, 1996;Guerette et al, 1997;Qu et al, 1998;Qu and Huard, 2000a, b). Although this method can transiently deliver dystrophin and improve the strength of injected dystrophic muscle, it has various limitations, including immune rejection, poor cellular survival rates, and limited spread of the injected cells (Morgan et al, 1988(Morgan et al, , 1990(Morgan et al, , 1993Karpati et al, 1989;Huard et al, 1991Huard et al, , 1992aHuard et al, , b, 1994aPartridge, 1991;Gussoni et al, 1992Gussoni et al, , 1997Karpati and Worton, 1992;…”

Section: Can Muscle Scs Regenerate Dystrophic Skeletal Muscle More Efmentioning

confidence: 93%

Muscle‐derived stem cells: Potential for muscle regeneration

Huard

Cao

Qu-Petersen

2003

Birth Defects Research Pt C

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Duchenne muscular dystrophy (DMD) is a devastating X-linked muscle disease characterized by progressive muscle weakness caused by the lack of dystrophin expression at the sarcolemma of muscle fibers. Although various approaches to delivering dystrophin in dystrophic muscle have been investigated extensively (e.g., cell and gene therapy), there is still no treatment that alleviates the muscle weakness in this common inherited muscle disease. The transplantation of myoblasts can enable transient delivery of dystrophin and improve the strength of injected dystrophic muscle, but this approach has various limitations, including immune rejection, poor cellular survival rates, and the limited spread of the injected cells. The isolation of muscle cells that can overcome these limitations would enhance the success of myoblast transplantation significantly. The efficiency of cell transplantation might be improved through the use of stem cells, which display unique features, including (1) self-renewal with production of progeny, (2) appearance early in development and persistence throughout life, and (3) long-term proliferation and multipotency. For these reasons, the development of muscle stem cells for use in transplantation or gene transfer (ex vivo approach) as treatment for patients with muscle disorders has become more attractive in the past few years. In this paper, we review the current knowledge regarding the isolation and characterization of stem cells isolated from skeletal muscle by highlighting their biological features and their relationship to satellite cells as well as other populations of stem cells derived from other tissues. We also describe the remarkable ability of stem cells to regenerate skeletal muscle and their potential use to alleviate the muscle weakness associated with DMD.

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Section: Can Muscle Scs Regenerate Dystrophic Skeletal Muscle More Efmentioning

confidence: 93%

Muscle‐derived stem cells: Potential for muscle regeneration

Huard

Cao

Qu-Petersen

2003

Birth Defects Research Pt C

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“…Recent evidence shows, in both man and mouse, that implantation of muscle precursor cells can cause an immune reaction, independent of the degree of matching MHC (Morgan et al 1987;Huard et al 1991; (Law et al 1990(Law et al , 1992 while others failed to find donor dystrophin in HLA-matched and immunosuppressed patients (Gussoni et at. 1992;Karpati et al 1993; see also Hoffman, 1993 (Wernig et al 1991; or after implantation of non-compatible primary MCs from CBA/J mice into non-tolerant A mice (Watt et al 1991 (Goodman, 1991; see also .…”

Section: Discussionmentioning

confidence: 99%

Functional effects of myoblast implantation into histoincompatible mice with or without immunosuppression.

Wernig

Irintchev

Lange

1995

The Journal of Physiology

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1. The goals of this study were to evaluate the immunogenicity of myogenic cells (MCs) (1) immediately after implantation into regenerating muscles, and (2) following their maturation under initial immunosuppression. Implanted mouse soleus muscles were evaluated by isometric tension recordings in vitro followed by histological investigations on frozen sections. 2. Implantation of non‐histocompatible myoblasts into cryodamaged soleus muscles of CBA/J mice induced immune rejection which caused large and permanent deficits in muscle force: 4‐42 weeks postimplantation maximal tetanic tension was 50‐60% that of intact or regenerated cryodamaged control muscles without tendency for recovery or histological signs of muscle regeneration. Specific tension (force per unit muscle weight) was also significantly reduced. 3. On frozen sections, only 62 +/‐ 12% of the total area was desmin‐positive, that is, occupied by muscle fibres, versus 90 +/‐ 4% in regenerated and 92 +/‐ 3% in intact muscles. Also, the total number of muscle fibre profiles was significantly reduced. 4. Under immune suppression with cyclosporin A (CsA), large muscles developed within 4 weeks. Following CsA withdrawal, muscle weight and force, in addition to desmin‐positive areas on cross‐sections, gradually declined over several months despite continual regeneration, indicating retarded immune rejection. 5. Initial application of CsA for 8 weeks after implantation, instead of 4 weeks, did not result in better survival of the implants, nor did a higher initial dose of CsA (100 instead of 50 mg kg‐1 day‐1). Prolonged continuous application of a reduced dose (25 mg kg‐1 day‐1) did not prevent muscle wasting but caused an additional delay. 6. It is concluded that histoincompatible myoblasts are highly immunogenic and that immune rejection causes large and permanent muscle deficits indicating elimination of host muscle tissue. Initial transient immunosuppression protects the incompatible cells, but after withdrawal, prolonged immune rejection and retarded muscle wasting occur.

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“…[11][12][13][14][15][16] Although these studies showed transient restoration of dystrophin and increase of strength in dystrophic muscle, the limited success of myoblast transplantation has been related to immune rejection, poor survival and limited spread of injected myoblasts after transplantation. [17][18][19][20][21][22][23][24][25][26][27][28][29][30][31][32] Indeed, long-term persistence of dystrophin has been fibers) of dystrophin deficient mdx mice. At several timepoints after injection (10, 20 and 30 days), the number of dystrophin-positive fibers was monitored and compared among the different groups.…”

Section: Introductionmentioning

confidence: 99%

Matching host muscle and donor myoblasts for myosin heavy chain improves myoblast transfer therapy

Huard

2000

Gene Ther

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Intensive efforts have been made to develop an effective therapy for Duchenne muscular dystrophy (DMD). Although myoblast transplantation has been found capable of transiently delivering dystrophin and improving the strength of the injected dystrophic muscle, this approach has been hindered by the immune rejection problems as well as the poor survival and limited spread of the injected cells. In the present study, we have investigated whether the careful selection of donor myoblasts and host muscle for the myosin heavy chain expression (MyHCs) plays a role in the success of myoblast transfer. Highly purified normal myoblasts derived from the m. soleus and m. gastrocnemius white of normal mice were transplanted into the m. soleus (containing 70% of type I fibers) and gastrocnemius white (100% of type II

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Correspondence Section: Myoblast transplantation produced dystrophin-positive muscle fibres in a 16-year-old patient with Duchenne muscular dystrophy

Cited by 61 publications

References 4 publications

Muscle‐derived stem cells: Potential for muscle regeneration

Muscle‐derived stem cells: Potential for muscle regeneration

Functional effects of myoblast implantation into histoincompatible mice with or without immunosuppression.

Matching host muscle and donor myoblasts for myosin heavy chain improves myoblast transfer therapy

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