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Engraftment of embryonic stem cell-derived myogenic progenitors in a dominant model of muscular dystrophy.
Exp Neurol. 2009 Nov; 220(1):212-6.EN

Abstract

Muscular dystrophies (MDs) consist of a genetically heterogeneous group of disorders, recessive or dominant, characterized by progressive skeletal muscle weakening. To date, no effective treatment is available. Experimental strategies pursuing muscle regeneration through the transplantation of stem cell preparations have brought hope to patients affected by this disorder. Efficacy has been demonstrated in recessive MD models through contribution of wild-type nuclei to the muscle fiber heterokaryon; however, to date, there has been no study investigating the efficacy of a cell therapy in a dominant model of MD. We have recently demonstrated that Pax3-induced embryonic stem (ES) cell-derived myogenic progenitors are able to engraft and improve muscle function in mdx mice, a recessive mouse model for Duchenne MD. To assess whether this therapeutic effect can be extended to a dominant type of muscle disorder, here we transplanted these cells into FRG1 transgenic mice, a dominant model that has been associated with facioscapulohumeral muscular dystrophy. Our results show that Pax3-induced ES-derived myogenic progenitors are capable of significant engraftment after intramuscular or systemic transplantation into Frg1 mice. Analyses of contractile parameters revealed functional improvement in treated muscles of male mice, but not females, which are less severely affected. This study is the first to use Frg1 transgenic mice to assess muscle regeneration as well as to support the use of a cell-based therapy for autosomal dominant types of MD.

Authors+Show Affiliations

Department of Developmental Biology, University of Texas Southwestern Medical Center, Dallas, TX, USA.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

19682990

Citation

Darabi, Radbod, et al. "Engraftment of Embryonic Stem Cell-derived Myogenic Progenitors in a Dominant Model of Muscular Dystrophy." Experimental Neurology, vol. 220, no. 1, 2009, pp. 212-6.
Darabi R, Baik J, Clee M, et al. Engraftment of embryonic stem cell-derived myogenic progenitors in a dominant model of muscular dystrophy. Exp Neurol. 2009;220(1):212-6.
Darabi, R., Baik, J., Clee, M., Kyba, M., Tupler, R., & Perlingeiro, R. C. (2009). Engraftment of embryonic stem cell-derived myogenic progenitors in a dominant model of muscular dystrophy. Experimental Neurology, 220(1), 212-6. https://doi.org/10.1016/j.expneurol.2009.08.002
Darabi R, et al. Engraftment of Embryonic Stem Cell-derived Myogenic Progenitors in a Dominant Model of Muscular Dystrophy. Exp Neurol. 2009;220(1):212-6. PubMed PMID: 19682990.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Engraftment of embryonic stem cell-derived myogenic progenitors in a dominant model of muscular dystrophy. AU - Darabi,Radbod, AU - Baik,June, AU - Clee,Mark, AU - Kyba,Michael, AU - Tupler,Rossella, AU - Perlingeiro,Rita C R, Y1 - 2009/08/13/ PY - 2009/03/27/received PY - 2009/07/24/revised PY - 2009/08/06/accepted PY - 2009/8/18/entrez PY - 2009/8/18/pubmed PY - 2009/11/17/medline SP - 212 EP - 6 JF - Experimental neurology JO - Exp. Neurol. VL - 220 IS - 1 N2 - Muscular dystrophies (MDs) consist of a genetically heterogeneous group of disorders, recessive or dominant, characterized by progressive skeletal muscle weakening. To date, no effective treatment is available. Experimental strategies pursuing muscle regeneration through the transplantation of stem cell preparations have brought hope to patients affected by this disorder. Efficacy has been demonstrated in recessive MD models through contribution of wild-type nuclei to the muscle fiber heterokaryon; however, to date, there has been no study investigating the efficacy of a cell therapy in a dominant model of MD. We have recently demonstrated that Pax3-induced embryonic stem (ES) cell-derived myogenic progenitors are able to engraft and improve muscle function in mdx mice, a recessive mouse model for Duchenne MD. To assess whether this therapeutic effect can be extended to a dominant type of muscle disorder, here we transplanted these cells into FRG1 transgenic mice, a dominant model that has been associated with facioscapulohumeral muscular dystrophy. Our results show that Pax3-induced ES-derived myogenic progenitors are capable of significant engraftment after intramuscular or systemic transplantation into Frg1 mice. Analyses of contractile parameters revealed functional improvement in treated muscles of male mice, but not females, which are less severely affected. This study is the first to use Frg1 transgenic mice to assess muscle regeneration as well as to support the use of a cell-based therapy for autosomal dominant types of MD. SN - 1090-2430 UR - https://www.unboundmedicine.com/medline/citation/19682990/Engraftment_of_embryonic_stem_cell_derived_myogenic_progenitors_in_a_dominant_model_of_muscular_dystrophy_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0014-4886(09)00328-8 DB - PRIME DP - Unbound Medicine ER -