CFTR corrector C17 is effective in muscular dystrophy, in vivo proof of concept in LGMDR3

Limb-girdle muscular dystrophy R3 (LGMDR3) is caused by mutations in the SGCA gene coding for α-sarcoglycan (SG). Together with β- γ- and δ-SG, α-SG forms a tetramer embedded in the dystrophin associated protein complex crucial for protecting the sarcolemma from mechanical stresses elicited by muscl...

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Detalles Bibliográficos
Autores principales: Scano, Martina, Benetollo, Alberto, Nogara, Leonardo, Bondì, Michela, Dalla Barba, Francesco, Soardi, Michela, Furlan, Sandra, Akyurek, Eylem Emek, Caccin, Paola, Carotti, Marcello, Sacchetto, Roberta, Blaauw, Bert, Sandonà, Dorianna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2021
Materias:
General Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8863415/
https://www.ncbi.nlm.nih.gov/pubmed/34505136
http://dx.doi.org/10.1093/hmg/ddab260

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