ArticleExperimental neurology2009
Engraftment of embryonic stem cell-derived myogenic progenitors in a dominant model of muscular dystrophy.
Article in Experimental neurology, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
16 citing papers in PubMed, 45 citations in OpenAlex.
- Cell therapy for Duchenne muscular dystrophy: promises, challenges, and controversies.Cellular and molecular life sciences : CMLS · 2025Review
- Transplantation of PSC-derived myogenic progenitors counteracts disease phenotypes in FSHD mice.NPJ Regenerative medicine · 2022Article
- Article
- Myogenic progenitor specification from pluripotent stem cells.Seminars in cell & developmental biology · 2017Review
- Coaxing stem cells for skeletal muscle repair.Advanced drug delivery reviews · 2015Review
- WNT3A promotes myogenesis of human embryonic stem cells and enhances in vivo engraftment.Scientific reports · 2014Article
- Stem cells for skeletal muscle regeneration: therapeutic potential and roadblocks.Translational research : the journal of laboratory and clinical medicine · 2014Review
- Advancements in stem cells treatment of skeletal muscle wasting.Frontiers in physiology · 2014Review
- Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells.Frontiers in physiology · 2014Review
- Gene and cell-mediated therapies for muscular dystrophy.Muscle & nerve · 2013Review
- The emerging biology of muscle stem cells: implications for cell-based therapies.BioEssays : news and reviews in molecular, cellular and developmental biology · 2013Review
- Directed in vitro myogenesis of human embryonic stem cells and their in vivo engraftment.PloS one · 2013Article
- Concise review: stem cell therapy for muscular dystrophies.Stem cells translational medicine · 2012Review
- Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- A case of cellular alchemy: lineage reprogramming and its potential in regenerative medicine.Journal of molecular cell biology · 2012Review
- Stem cells for skeletal muscle repair.Philosophical transactions of the Royal Society of London. Series B, Biological sciences · 2011Review
Corrections and comments
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Authors and funding
6 authors at 4 institutions in 2 countries.
Funding
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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.