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

Prectinical 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, corre...

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Bibliographic Details
Published in:Science (American Association for the Advancement of Science) 2003-07, Vol.301 (5632), p.487-492
Main Authors: Sampaolesi, Maurilio, Torrente, Yvan, Innocenzi, Anna, Tonlorenzi, Rossana
Format: Article
Language:English
Subjects:
Animals
Clinical trials
Cytology
Donors
Females
Genetics
Individualized Instruction
Males
Success
Therapy
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Summary:Prectinical 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]-SC 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. [PUBLICATION ABSTRACT]
ISSN:0036-8075
1095-9203