Réhabilitation de la dystrophine humaine dans le modèle scid/mdx après greffe de cellules souches de patients DMD corrigées par saut d’exon

Rachid Benchaouir; Mirella Meregalli; Andrea Farini; Giuseppe D’Antona; Marzia Belicchi; Aurélie Goyenvalle; Maurizio Battistelli; Nereo Bresolin; Roberto Bottinelli; Luis Garcia; Yvan Torrente

doi:10.1051/medsci/200824199

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Volume 24 / No 1 (Janvier 2008)

Med Sci (Paris), 24 1 (2008) 99-101

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Issue		Med Sci (Paris) Volume 24, Number 1, Janvier 2008


Page(s)		99 - 101
Section		Dernière Heure
DOI		https://doi.org/10.1051/medsci/200824199
Published online		15 January 2008

Benchaouir R, Meregalli M, Farini A, D’Antona G, Belicchi M, Goyenvalle A, Battistelli M, Bresolin N, Bottinelli R, Garcia L, Torrente Y. Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. Cell Stem Cell 2007; 1 : 646–57. [Google Scholar]
Torrente Y, Belicchi M, Sampaolesi M, et al. Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle. J Clin Invest 2004; 114 : 182–95. [Google Scholar]
Torrente Y, Belicchi M, Marchesi C, et al. Autologous transplantation of muscle-derived CD133⁺ stem cells in Duchenne muscle patients. Cell Transplant 2007; 16 : 563–77. [Google Scholar]
Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 2004; 306 : 1796–9. [Google Scholar]
Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O. Le saut d’exon thérapeutique : un espoir pour les dystrophinopathies. Med Sci (Paris) 2004; 20 : 1163–5. [Google Scholar]

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[1] Benchaouir R, Meregalli M, Farini A, D’Antona G, Belicchi M, Goyenvalle A, Battistelli M, Bresolin N, Bottinelli R, Garcia L, Torrente Y. Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. Cell Stem Cell 2007; 1 : 646–57. [Google Scholar]

[2] Torrente Y, Belicchi M, Sampaolesi M, et al. Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle. J Clin Invest 2004; 114 : 182–95. [Google Scholar]

[3] Torrente Y, Belicchi M, Marchesi C, et al. Autologous transplantation of muscle-derived CD133⁺ stem cells in Duchenne muscle patients. Cell Transplant 2007; 16 : 563–77. [Google Scholar]

[4] Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 2004; 306 : 1796–9. [Google Scholar]

[5] Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O. Le saut d’exon thérapeutique : un espoir pour les dystrophinopathies. Med Sci (Paris) 2004; 20 : 1163–5. [Google Scholar]