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...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Frattini, Paola, Villa, Chiara, De Santis, Francesca, Meregalli, Mirella, Belicchi, Marzia, Erratico, Silvia, Bella, Pamela, Raimondi, Manuela Teresa, Lu, Qilong, Torrente, Yvan
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2017
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5886111/
https://ncbi.nlm.nih.gov/pubmed/28666318
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddx252
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