Repairing folding-defective α-sarcoglycan mutants by CFTR correctors, a potential therapy for limb-girdle muscular dystrophy 2D

Written on . Posted in Drug therapy.

Hum Mol Genet . 2018 Mar 15;27(6):969-984. doi: 10.1093/hmg/ddy013.

Marcello Carotti 1, Justine Marsolier 2 3, Michela Soardi 1, Elisa Bianchini 1 4, Chiara Gomiero 5, Chiara Fecchio 1, Sara F Henriques 2 3, Romeo Betto 6, Roberta Sacchetto 5, Isabelle Richard 2 3, Dorianna Sandonà 1


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PMID: 29351619 PMCID: PMC5886177 DOI: 10.1093/hmg/ddy013
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https://pubmed.ncbi.nlm.nih.gov/29351619/