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Autologous intramuscular transplantation of engineered satellite cells induces exosome-mediated systemic expression of Fukutin-related protein and rescues disease phenotype in a murine model of limb-girdle muscular dystrophy type 2I
α-Dystroglycanopathies are a group of muscular dystrophies characterized by α-DG hypoglycosylation and reduced extracellular ligand-binding affinity. Among other genes involved in the α-DG glycosylation process, fukutin related protein (FKRP) gene mutations generate a wide range of pathologies from...
Autores principales: | Frattini, Paola, Villa, Chiara, De Santis, Francesca, Meregalli, Mirella, Belicchi, Marzia, Erratico, Silvia, Bella, Pamela, Raimondi, Manuela Teresa, Lu, Qilong, Torrente, Yvan |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5886111/ https://www.ncbi.nlm.nih.gov/pubmed/28666318 http://dx.doi.org/10.1093/hmg/ddx252 |
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