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Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts.

Sampaolesi, M; Torrente, Y; Innocenzi, A; Tonlorenzi, R; D'Antona, G; Pellegrino, MA; Barresi, R; ... Cossu, G; + view all (2003) Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science , 301 (5632) pp. 487-492. 10.1126/science.1082254.

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Abstract

Preclinical or clinical trials for muscular dystrophies have met with modest success, mainly because of inefficient delivery of viral vectors or donor cells to dystrophic muscles. We report here that intra-arterial delivery of wild-type mesoangioblasts, a class of vessel-associated stem cells, corrects morphologically and functionally the dystrophic phenotype of virtually all downstream muscles in adult immunocompetent alpha-sarcoglycan (alpha-SG) null mice, a model organism for limb-girdle muscular dystrophy. When mesoangioblasts isolated from juvenile dystrophic mice and transduced with a lentiviral vector expressing alpha-SG were injected into the femoral artery of dystrophic mice, they reconstituted skeletal muscle in a manner similar to that seen in wild-type cells. The success of this protocol was mainly due to widespread distribution of donor stem cells through the capillary network, a distinct advantage of this strategy over previous approaches.

Type: Article
Title: Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts.
Location: United States
DOI: 10.1126/science.1082254
Keywords: Animals, Blood Vessels, Cell Differentiation, Cell Line, Cell Movement, Cytoskeletal Proteins, Dystrophin, Endothelium, Vascular, Female, Femoral Artery, Genetic Vectors, Lentivirus, Locomotion, Male, Membrane Glycoproteins, Mesoderm, Mice, Mice, Knockout, Mice, Transgenic, Muscle Contraction, Muscle Fibers, Skeletal, Muscle, Skeletal, Muscular Dystrophy, Animal, Regeneration, Sarcoglycans, Stem Cell Transplantation, Stem Cells, Transfection
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences > Cell and Developmental Biology
URI: http://discovery.ucl.ac.uk/id/eprint/1342547
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