Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice;Réhabilitation de la dystrophine humaine dans le modèle scid/mdx après greffe de cellules souches de patients DMD corrigées par saut d'exon

Medecine/Sciences

Volumn 24, Issue 1, 2008, Pages 99-101

  Benchaouir, Rachid ^a   Meregalli, Mirella ^a   Farini, Andrea ^a   D'Antona, Giuseppe ^b   Belicchi, Marzia ^a   Goyenvalle, Aurélie ^d   Battistelli, Maurizio ^a   Bresolin, Nereo ^a   Bottinelli, Roberto ^b   Garcia, Luis ^c   Torrente, Yvan ^a  

^a UNIVERSITY OF MILAN   (Italy)

^b UNIVERSITY OF PAVIA   (Italy)

^c SORBONNE UNIVERSITÉ   (France)

^d NONE

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN;

GENE DELETION; GENE MUTATION; GENE SEQUENCE; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; MUSCULAR DYSTROPHY; NONHUMAN; NOTE; STEM CELL; TRANSPLANTATION;

MUS;

EID: 38849124255     PISSN: 07670974     EISSN: None     Source Type: Journal    
DOI: 10.1051/medsci/200824199     Document Type: Note

References (5)

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.
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.
3. + stem cells in Duchenne muscle patients. Cell Transplant 2007 ; 16 : 563-77.
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.
5. Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O. Le saut d'exon thérapeutique : un espoir pour les dystrophinapathies. Med Sci (Paris) 2004 ; 20 : 1163-5.