Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells

Bachrach, Estanislao; Li, Sheng; Pérez, Antonio L.; Schienda, Jaclyn; Liadaki, Kalliopi; Volinski, Jay; Flint, Alan; Chamberlain, Jeffrey S.; Kunkel, Louis M.

doi:10.1073/pnas.0400373101

Cited by 127 publications

(106 citation statements)

References 42 publications

Supporting

Mentioning

103

Contrasting

Unclassified

Order By: Relevance

“…No effective treatment exists although novel therapeutic strategies, ranging from new drugs to gene and cell therapy, hold promise for significant advance in the future 2 . In particular, different types of stem cell have been shown to induce dystrophin synthesis and partial rescue of the pathology in dystrophic mice [3][4][5][6][7][8] . However, dystrophic mice do not display clinical signs of the disease, and to proceed to a clinical trial it is imperative to show efficacy in a large, non-syngeneic animal model of muscular dystrophy.…”

mentioning

confidence: 99%

Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

Sampaolesi

Blot

D’Antona

et al. 2006

Nature

677

569

View full text Add to dashboard Cite

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.

show abstract

mentioning

confidence: 99%

Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

Sampaolesi

Blot

D’Antona

et al. 2006

Nature

677

569

View full text Add to dashboard Cite

show abstract

“…Among non-resident precursors, a variety of different cells with intrinsic myogenic properties have been isolated. These include adipose-tissue derived stem cells [47], mesoangioblasts [48], pericytes [49], muscle-derived stem cells [50], side-population cells [51][52][53], Ac133 + cells [54], stem and/or precursor cells from muscle endothelium [55], and synovium [56]. Mesoangioblasts, multipotent progenitors of mesodermal tissues, particularly attracted scientific attention for their possible use in stem cell therapy since they ameliorated some myopathies in animal models.…”

Section: Adiponectin As a Tissue-regenerating Hormonementioning

confidence: 99%

Adiponectin as a tissue regenerating hormone: more than a metabolic function

Fiaschi

Magherini

Gamberi

et al. 2013

Cell. Mol. Life Sci.

View full text Add to dashboard Cite

“…In response to muscle injuries, quiescent satellite cells undergo activation, proliferate, and fuse with each other or with damaged fibers (26). Other sources of myogenic precursors, mostly of mesodermic origin, have been identified (27)(28)(29)(30)(31)(32)(33)(34)(35)(36) including mesoangioblasts from the embryonic dorsal aorta and their counterparts associated with microvascular walls in the adult skeletal muscle, pericytes (37), and have been shown to contribute to muscle regeneration (36,(38)(39)(40)(41)(42). Upon transplantation in regenerating injured skeletal muscle, mesoangioblasts participate in tissue growth and regeneration and fuse with resident satellite cells (43).…”

mentioning

confidence: 99%

Transplanted Mesoangioblasts Require Macrophage IL-10 for Survival in a Mouse Model of Muscle Injury

Bosurgi

Corna

Vezzoli

et al. 2012

The Journal of Immunology

View full text Add to dashboard Cite

The aim of this study was to verify whether macrophages influence the fate of transplanted mesoangioblasts—vessel-associated myogenic precursors—in a model of sterile toxin-induced skeletal muscle injury. We have observed that in the absence of macrophages, transplanted mesoangioblasts do not yield novel fibers. Macrophages retrieved from skeletal muscles at various times after injury display features that resemble those of immunoregulatory macrophages. Indeed, they secrete IL-10 and express CD206 and CD163 membrane receptors and high amounts of arginase I. We have reconstituted the muscle-associated macrophage population by injecting polarized macrophages before mesoangioblast injection: alternatively activated, immunoregulatory macrophages only support mesoangioblast survival and function. This action depends on the secretion of IL-10 in the tissue. Our results reveal an unanticipated role for tissue macrophages in mesoangioblast function. Consequently, the treatment of muscle disorders with mesoangioblasts should take into consideration coexisting inflammatory pathways, whose activation may prove crucial for its success.

show abstract

Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells

Cited by 127 publications

References 42 publications

Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

Adiponectin as a tissue regenerating hormone: more than a metabolic function

Transplanted Mesoangioblasts Require Macrophage IL-10 for Survival in a Mouse Model of Muscle Injury

Contact Info

Product

Resources

About