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

Maurilio Sampaolesi, Yvan Torrente, Anna Innocenzi, Rossana Tonlorenzi, Giuseppe D'Antona, M. Antonietta Pellegrino, Rita Barresi, Nereo Bresolin, M. Gabriella Cusella De Angelis, Kevin P. Campbell, Roberto Bottinelli, Giulio Cossu

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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 wildtype mesoangioblasts, a class of vessel-associated stem cells, corrects morphologically and functionally the dystrophic phenotype of virtually all downstream muscles in adult immunocompetent α-sarcoglycan (α-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 α-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.

Original languageEnglish
Pages (from-to)487-492
Number of pages6
JournalScience
Volume301
Issue number5632
DOIs
Publication statusPublished - Jul 25 2003

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    Sampaolesi, M., Torrente, Y., Innocenzi, A., Tonlorenzi, R., D'Antona, G., Pellegrino, M. A., Barresi, R., Bresolin, N., De Angelis, M. G. C., Campbell, K. P., Bottinelli, R., & Cossu, G. (2003). Cell therapy of α-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science, 301(5632), 487-492. https://doi.org/10.1126/science.1082254