Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

Maurilio Sampaolesi, Stephane Blot, Giuseppe D’Antona, Nicolas Granger, Rossana Tonlorenzi, Anna Innocenzi, Paolo Mognol, Jean-Lauren Thibaud, Beatriz G. Galvez, Ines Barthélémy, Laura Perani, Sara Mantero, Maria Guttinger, Orietta Pansarasa, Chiara Rinaldi, M. Gabriella Cusella De Angelis, Yvan Torrente, Claudio Bordignon, Roberto Bottinelli () and Giulio Cossu ()
Additional contact information
Maurilio Sampaolesi: Università Vita e Salute, Stem Cell Research Institute
Stephane Blot: Neurobiology Laboratory, École Vétérinaire d’Alfort
Giuseppe D’Antona: University of Pavia
Nicolas Granger: Neurobiology Laboratory, École Vétérinaire d’Alfort
Rossana Tonlorenzi: Università Vita e Salute, Stem Cell Research Institute
Anna Innocenzi: Università Vita e Salute, Stem Cell Research Institute
Paolo Mognol: Politecnico di Milano, Piazza Leonardo Da Vinci
Jean-Lauren Thibaud: Neurobiology Laboratory, École Vétérinaire d’Alfort
Beatriz G. Galvez: Università Vita e Salute, Stem Cell Research Institute
Ines Barthélémy: Neurobiology Laboratory, École Vétérinaire d’Alfort
Laura Perani: Università Vita e Salute, Stem Cell Research Institute
Sara Mantero: Politecnico di Milano, Piazza Leonardo Da Vinci
Maria Guttinger: Institute of Cell Biology and Tissue Engineering, San Raffaele Biomedical Science Park of Rome
Orietta Pansarasa: University of Pavia
Chiara Rinaldi: University of Pavia
M. Gabriella Cusella De Angelis: University of Pavia
Yvan Torrente: University of Milan
Claudio Bordignon: Università Vita e Salute, Stem Cell Research Institute
Roberto Bottinelli: University of Pavia
Giulio Cossu: Università Vita e Salute, Stem Cell Research Institute

Nature, 2006, vol. 444, issue 7119, 574-579

Abstract: Abstract Duchenne muscular dystrophy remains an untreatable genetic disease that severely limits motility and life expectancy in affected children. The only animal model specifically reproducing the alterations in the dystrophin gene and the full spectrum of human pathology is the golden retriever dog model. Affected animals present a single mutation in intron 6, resulting in complete absence of the dystrophin protein, and early and severe muscle degeneration with nearly complete loss of motility and walking ability. Death usually occurs at about 1 year of age as a result of failure of respiratory muscles. Here we report that intra-arterial delivery of wild-type canine mesoangioblasts (vessel-associated stem cells) results in an extensive recovery of dystrophin expression, normal muscle morphology and function (confirmed by measurement of contraction force on single fibres). The outcome is a remarkable clinical amelioration and preservation of active motility. These data qualify mesoangioblasts as candidates for future stem cell therapy for Duchenne patients.

Date: 2006
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DOI: 10.1038/nature05282

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