Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle |
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Authors: | Torrente Yvan Belicchi Marzia Sampaolesi Maurilio Pisati Federica Meregalli Mirella D'Antona Giuseppe Tonlorenzi Rossana Porretti Laura Gavina Manuela Mamchaoui Kamel Pellegrino Maria Antonietta Furling Denis Mouly Vincent Butler-Browne Gillian S Bottinelli Roberto Cossu Giulio Bresolin Nereo |
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Institution: | Stem Cell Laboratory, Department of Neurological Science, Instituto di Ricovero e Cura a Carattere Scientifico Ospedale Maggiore Policlinico, Centro Dino Ferrari, University of Milan, Italy. torrenteyvan@hotmail.com |
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Abstract: | Duchenne muscular dystrophy (DMD) is a common X-linked disease characterized by widespread muscle damage that invariably leads to paralysis and death. There is currently no therapy for this disease. Here we report that a subpopulation of circulating cells expressing AC133, a well-characterized marker of hematopoietic stem cells, also expresses early myogenic markers. Freshly isolated, circulating AC133(+) cells were induced to undergo myogenesis when cocultured with myogenic cells or exposed to Wnt-producing cells in vitro and when delivered in vivo through the arterial circulation or directly into the muscles of transgenic scid/mdx mice (which allow survival of human cells). Injected cells also localized under the basal lamina of host muscle fibers and expressed satellite cell markers such as M-cadherin and MYF5. Furthermore, functional tests of injected muscles revealed a substantial recovery of force after treatment. As these cells can be isolated from the blood, manipulated in vitro, and delivered through the circulation, they represent a possible tool for future cell therapy applications in DMD disease or other muscular dystrophies. |
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