Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2006-11

AUTHORS

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, Giulio Cossu

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. More... »

PAGES

574

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/nature05282

DOI

http://dx.doi.org/10.1038/nature05282

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1011740155

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/17108972


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