Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy

Mendell, Jerry R.; Kissel, John T.; Amato, Anthony A.; King, Wendy; Signore, L.; Prior, Thomas W.; Sahenk, Zarife; Benson, Sandra; McAndrew, Patricia; Rice, Robert L.; Nagaraja, Haikady N.; Stephens, R. E.; Lantry, Laura E.; Morris, G. E.; Burghes, Arthur H.M.

doi:10.1056/nejm199509283331303

Cited by 481 publications

(287 citation statements)

References 29 publications

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“…14,15 Despite positive results in these small rodents, [16][17][18][19][20] clinical trials have presented limited success. [21][22][23][24][25][26][27][28][29] Improvements of injection and immunosuppression parameters have recently been achieved in primates 9,10,30 and translated to the human situation with encouraging, although localized success. 31 Nevertheless, one of the most important problems of heterologous MT remains the immune response raised by the recipient against donor cells.…”

Section: Introductionmentioning

confidence: 99%

Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Vilquin

Sacconi

et al. 2005

Gene Ther

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Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant disease characterized by a typical regional distribution, featuring composed patterns of clinically affected and unaffected muscles. No treatment is available for this condition, in which the pathophysiological mechanism is still unknown. Autologous transfer of myoblasts from unaffected to affected territories could be considered as a potential strategy to delay or stop muscle degeneration. To evaluate the feasibility of this concept, we explored and compared the growth and differentiation characteristics of myoblasts prepared from phenotypically unaffected muscles of five FSHD patients and 10 control donors. According to a clinically approved procedure, 10 9 cells of a high degree of purity were obtained within 16-23 days. More than 80% of these cells were myoblasts, as demonstrated by labeling of the muscle markers CD56 and desmin. FSHD myoblasts presented a doubling time equivalent to that of control cells; they kept high proliferation ability and did not show early telomere shortening. In vitro, these cells were able to differentiate and to express muscle-specific antigens. In vivo, they participated to muscle structures when injected into immunodeficient mice. These data suggest that myoblasts expanded from unaffected FSHD muscles may be suitable tools in view of autologous cell transplantation clinical trials.

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Section: Introductionmentioning

confidence: 99%

Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Vilquin

Sacconi

et al. 2005

Gene Ther

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“…[7][8][9][10] However, the transplantation of donor muscle cells into host muscle has been hindered by various limitations, such as immune rejection that consequently leads to poor spreading and survival of injected cells. [11][12][13][14][15][16][17][18][19][20] The success of cell transplantation may be dramatically improved by using a specific population of cells that has the capacity to overcome these hurdles.…”

Section: Introductionmentioning

confidence: 99%

Muscle-derived cell-mediated ex vivo gene therapy for urological dysfunction

et al. 2002

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“…[8][9][10] In the case of DMD, the only clinical trials performed to date involved healthy myoblast graft. [11][12][13][14][15][16][17][18] In these trials, which involved a total of 60 patients, the heterologous cells were obtained from healthy donors, either HLA compatible or incompatible; 16,18 one trial involved homozygous siblings. 19 An additional 150 patients were involved in further studies performed, 20 which however were controversial.…”

Section: Introductionmentioning

confidence: 99%

Immune rejection of human dystrophin following intramuscular injections of naked DNA in mdx mice

et al. 2000

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Intramuscular administration of plasmid expressing fulllength human dystrophin in dystrophin-deficient adult mdx mice resulted in humoral and weak specific T cell responses against the human dystrophin protein. Following plasmid injection, human dystrophin was detected in the injected muscles at 7 days, but decreased thereafter. Anti-dystrophin antibodies were found 21 days following plasmid injection, which coincided with transient myositis. This immune rejec-

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Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy

Cited by 481 publications

References 29 publications

Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Muscle-derived cell-mediated ex vivo gene therapy for urological dysfunction

Immune rejection of human dystrophin following intramuscular injections of naked DNA in mdx mice

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