Title: Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs
Authors: Sampaolesi, Maurilio ×
Blot, Stephane
D'Antona, Giuseppe
Granger, Nicolas
Tonlorenzi, Rossana
Innocenzi, Anna
Mognol, Paolo
Thibaud, Jean-Laurent
Galvez, Beatriz G
Barthelemy, Ines
Perani, Laura
Mantero, Sara
Guttinger, Maria
Pansarasa, Orietta
Rinaldi, Chiara
De Angelis, M. Gabriella Cusella
Torrente, Yvan
Bordignon, Claudio
Bottinelli, Roberto
Cossu, Giulio #
Issue Date: Nov-2006
Series Title: Nature vol:444 issue:7119 pages:574-579
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.
ISSN: 0028-0836
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Interdepartemental Stem Cell Institute (-)
× corresponding author
# (joint) last author

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