Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery

Sarcoglycanopathies are rare limb girdle muscular dystrophies, still incurable, even though symptomatic treatments may slow down the disease progression. Most of the disease-causing defects are missense mutations leading to a folding defective protein, promptly removed by the cell’s quality control,...

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Detalles Bibliográficos
Autores principales: Carotti, Marcello, Scano, Martina, Fancello, Irene, Richard, Isabelle, Risato, Giovanni, Bensalah, Mona, Soardi, Michela, Sandonà, Dorianna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7084537/
https://www.ncbi.nlm.nih.gov/pubmed/32155735
http://dx.doi.org/10.3390/ijms21051813

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7084537/
https://www.ncbi.nlm.nih.gov/pubmed/32155735
http://dx.doi.org/10.3390/ijms21051813

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