title: Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs.
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.
authors.affiliation: San Raffaele Scientific Institute, Università Vita e Salute, Stem Cell Research Institute, Via Olgettina 58, 20132 Milan, Italy.
authors.forename: Maurilio
authors.initials: M
authors.lastname: Sampaolesi
authors.forename: Stephane
authors.initials: S
authors.lastname: Blot
authors.forename: Giuseppe
authors.initials: G
authors.lastname: D'Antona
authors.forename: Nicolas
authors.initials: N
authors.lastname: Granger
authors.forename: Rossana
authors.initials: R
authors.lastname: Tonlorenzi
authors.forename: Anna
authors.initials: A
authors.lastname: Innocenzi
authors.forename: Paolo
authors.initials: P
authors.lastname: Mognol
authors.forename: Jean-Lauren
authors.initials: JL
authors.lastname: Thibaud
authors.forename: Beatriz G
authors.initials: BG
authors.lastname: Galvez
authors.forename: Ines
authors.initials: I
authors.lastname: Barthélémy
authors.forename: Laura
authors.initials: L
authors.lastname: Perani
authors.forename: Sara
authors.initials: S
authors.lastname: Mantero
authors.forename: Maria
authors.initials: M
authors.lastname: Guttinger
authors.forename: Orietta
authors.initials: O
authors.lastname: Pansarasa
authors.forename: Chiara
authors.initials: C
authors.lastname: Rinaldi
authors.forename: M Gabriella
authors.initials: MG
authors.lastname: Cusella De Angelis
authors.forename: Yvan
authors.initials: Y
authors.lastname: Torrente
authors.forename: Claudio
authors.initials: C
authors.lastname: Bordignon
authors.forename: Roberto
authors.initials: R
authors.lastname: Bottinelli
authors.forename: Giulio
authors.initials: G
authors.lastname: Cossu
mesh_terms: D053687:Adult Stem Cells / Q000276:immunology / Q000637:transplantation*; D000818:Animals; D003131:Combined Modality Therapy; D003402:Creatine Kinase / Q000097:blood; D004285:Dogs; D016189:Dystrophin / Q000096:biosynthesis / Q000235:genetics / Q000276:immunology; D015316:Genetic Therapy; D006801:Humans; D008297:Male; D032342:Muscle Cells; D009137:Muscular Dystrophy, Animal / Q000628:therapy*; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*; D011993:Recombinant Fusion Proteins / Q000096:biosynthesis / Q000235:genetics; D033581:Stem Cell Transplantation; D014182:Transplantation, Autologous; D014183:Transplantation, Heterologous
chemical_list: D016189:Dystrophin; D011993:Recombinant Fusion Proteins; D003402:Creatine Kinase