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Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle.
J Clin Invest. 2004 Jul; 114(2):182-95.JCI

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.

Authors+Show Affiliations

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.comNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

15254585

Citation

Torrente, Yvan, et al. "Human Circulating AC133(+) Stem Cells Restore Dystrophin Expression and Ameliorate Function in Dystrophic Skeletal Muscle." The Journal of Clinical Investigation, vol. 114, no. 2, 2004, pp. 182-95.
Torrente Y, Belicchi M, Sampaolesi M, et al. Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle. J Clin Invest. 2004;114(2):182-95.
Torrente, Y., Belicchi, M., Sampaolesi, M., Pisati, F., Meregalli, M., D'Antona, G., Tonlorenzi, R., Porretti, L., Gavina, M., Mamchaoui, K., Pellegrino, M. A., Furling, D., Mouly, V., Butler-Browne, G. S., Bottinelli, R., Cossu, G., & Bresolin, N. (2004). Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle. The Journal of Clinical Investigation, 114(2), 182-95.
Torrente Y, et al. Human Circulating AC133(+) Stem Cells Restore Dystrophin Expression and Ameliorate Function in Dystrophic Skeletal Muscle. J Clin Invest. 2004;114(2):182-95. PubMed PMID: 15254585.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle. AU - Torrente,Yvan, AU - Belicchi,Marzia, AU - Sampaolesi,Maurilio, AU - Pisati,Federica, AU - Meregalli,Mirella, AU - D'Antona,Giuseppe, AU - Tonlorenzi,Rossana, AU - Porretti,Laura, AU - Gavina,Manuela, AU - Mamchaoui,Kamel, AU - Pellegrino,Maria Antonietta, AU - Furling,Denis, AU - Mouly,Vincent, AU - Butler-Browne,Gillian S, AU - Bottinelli,Roberto, AU - Cossu,Giulio, AU - Bresolin,Nereo, PY - 2003/10/17/received PY - 2004/04/27/accepted PY - 2004/7/16/pubmed PY - 2004/9/1/medline PY - 2004/7/16/entrez SP - 182 EP - 95 JF - The Journal of clinical investigation JO - J. Clin. Invest. VL - 114 IS - 2 N2 - 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. SN - 0021-9738 UR - https://www.unboundmedicine.com/medline/citation/15254585/Human_circulating_AC133_+__stem_cells_restore_dystrophin_expression_and_ameliorate_function_in_dystrophic_skeletal_muscle_ L2 - https://doi.org/10.1172/JCI20325 DB - PRIME DP - Unbound Medicine ER -