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Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs.
Am J Pathol. 2011 Nov; 179(5):2501-18.AJ

Abstract

Duchenne muscular dystrophy (DMD) is a genetic progressive muscle disease resulting from the lack of dystrophin and without effective treatment. Adult stem cell populations have given new impetus to cell-based therapy of neuromuscular diseases. One of them, muscle-derived stem cells, isolated based on delayed adhesion properties, contributes to injured muscle repair. However, these data were collected in dystrophic mice that exhibit a relatively mild tissue phenotype and clinical features of DMD patients. Here, we characterized canine delayed adherent stem cells and investigated the efficacy of their systemic delivery in the clinically relevant DMD animal model to assess potential therapeutic application in humans. Delayed adherent stem cells, named MuStem cells (muscle stem cells), were isolated from healthy dog muscle using a preplating technique. In vitro, MuStem cells displayed a large expansion capacity, an ability to proliferate in suspension, and a multilineage differentiation potential. Phenotypically, they corresponded to early myogenic progenitors and uncommitted cells. When injected in immunosuppressed dystrophic dogs, they contributed to myofiber regeneration, satellite cell replenishment, and dystrophin expression. Importantly, their systemic delivery resulted in long-term dystrophin expression, muscle damage course limitation with an increased regeneration activity and an interstitial expansion restriction, and persisting stabilization of the dog's clinical status. These results demonstrate that MuStem cells could provide an attractive therapeutic avenue for DMD patients.

Authors+Show Affiliations

INRA, UMR 703 Développement et Pathologie du Tissu Musculaire, Nantes, France. karl.rouger@nantes.inra.frNo 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 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

21924229

Citation

Rouger, Karl, et al. "Systemic Delivery of Allogenic Muscle Stem Cells Induces Long-term Muscle Repair and Clinical Efficacy in Duchenne Muscular Dystrophy Dogs." The American Journal of Pathology, vol. 179, no. 5, 2011, pp. 2501-18.
Rouger K, Larcher T, Dubreil L, et al. Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs. Am J Pathol. 2011;179(5):2501-18.
Rouger, K., Larcher, T., Dubreil, L., Deschamps, J. Y., Le Guiner, C., Jouvion, G., Delorme, B., Lieubeau, B., Carlus, M., Fornasari, B., Theret, M., Orlando, P., Ledevin, M., Zuber, C., Leroux, I., Deleau, S., Guigand, L., Testault, I., Le Rumeur, E., ... Chérel, Y. (2011). Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs. The American Journal of Pathology, 179(5), 2501-18. https://doi.org/10.1016/j.ajpath.2011.07.022
Rouger K, et al. Systemic Delivery of Allogenic Muscle Stem Cells Induces Long-term Muscle Repair and Clinical Efficacy in Duchenne Muscular Dystrophy Dogs. Am J Pathol. 2011;179(5):2501-18. PubMed PMID: 21924229.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs. AU - Rouger,Karl, AU - Larcher,Thibaut, AU - Dubreil,Laurence, AU - Deschamps,Jack-Yves, AU - Le Guiner,Caroline, AU - Jouvion,Gregory, AU - Delorme,Bruno, AU - Lieubeau,Blandine, AU - Carlus,Marine, AU - Fornasari,Benoît, AU - Theret,Marine, AU - Orlando,Priscilla, AU - Ledevin,Mireille, AU - Zuber,Céline, AU - Leroux,Isabelle, AU - Deleau,Stéphane, AU - Guigand,Lydie, AU - Testault,Isabelle, AU - Le Rumeur,Elisabeth, AU - Fiszman,Marc, AU - Chérel,Yan, Y1 - 2011/09/13/ PY - 2011/03/04/received PY - 2011/07/13/revised PY - 2011/07/19/accepted PY - 2011/9/20/entrez PY - 2011/9/20/pubmed PY - 2012/1/25/medline SP - 2501 EP - 18 JF - The American journal of pathology JO - Am. J. Pathol. VL - 179 IS - 5 N2 - Duchenne muscular dystrophy (DMD) is a genetic progressive muscle disease resulting from the lack of dystrophin and without effective treatment. Adult stem cell populations have given new impetus to cell-based therapy of neuromuscular diseases. One of them, muscle-derived stem cells, isolated based on delayed adhesion properties, contributes to injured muscle repair. However, these data were collected in dystrophic mice that exhibit a relatively mild tissue phenotype and clinical features of DMD patients. Here, we characterized canine delayed adherent stem cells and investigated the efficacy of their systemic delivery in the clinically relevant DMD animal model to assess potential therapeutic application in humans. Delayed adherent stem cells, named MuStem cells (muscle stem cells), were isolated from healthy dog muscle using a preplating technique. In vitro, MuStem cells displayed a large expansion capacity, an ability to proliferate in suspension, and a multilineage differentiation potential. Phenotypically, they corresponded to early myogenic progenitors and uncommitted cells. When injected in immunosuppressed dystrophic dogs, they contributed to myofiber regeneration, satellite cell replenishment, and dystrophin expression. Importantly, their systemic delivery resulted in long-term dystrophin expression, muscle damage course limitation with an increased regeneration activity and an interstitial expansion restriction, and persisting stabilization of the dog's clinical status. These results demonstrate that MuStem cells could provide an attractive therapeutic avenue for DMD patients. SN - 1525-2191 UR - https://www.unboundmedicine.com/medline/citation/21924229/Systemic_delivery_of_allogenic_muscle_stem_cells_induces_long_term_muscle_repair_and_clinical_efficacy_in_duchenne_muscular_dystrophy_dogs_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0002-9440(11)00741-3 DB - PRIME DP - Unbound Medicine ER -