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

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 qualit...

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Bibliographic Details
Main Authors: Marcello Carotti, Martina Scano, Irene Fancello, Isabelle Richard, Giovanni Risato, Mona Bensalah, Michela Soardi, Dorianna Sandonà
Format: Article
Language:English
Published: MDPI AG 2020-03-01
Series:International Journal of Molecular Sciences
Subjects:
sarcoglycanopathy
myogenic cells
myotubes
folding-defective proteins
protein folding correctors
cftr correctors
proteostasis regulators
Online Access:https://www.mdpi.com/1422-0067/21/5/1813

Internet

https://www.mdpi.com/1422-0067/21/5/1813

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