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Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy

Francesco Saverio Tedesco,Mattia F. M. Gerli,Laura Perani,Sara Benedetti,Federica Ungaro,Marco Cassano,Stefania Antonini,Enrico Tagliafico,Valentina Artusi,Emanuela Longa,Rossana Tonlorenzi,Martina Ragazzi, Giorgia Calderazzi,Hidetoshi Hoshiya,Ornella Cappellari,Marina Mora,Benedikt Schoser,Peter Schneiderat,Mitsuo Oshimura,Roberto Bottinelli,Maurilio Sampaolesi,Yvan Torrente,Vania Broccoli,Giulio Cossu

Science Translational Medicine（2012）

Cited 348|Views9
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Abstract
Genetically corrected mesoangioblasts from human iPSCs derived from limb-girdle muscular dystrophy patients produce muscle fibers expressing the therapeutic gene in a mouse model of the disease.
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