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Additive Potentiation of R334W-CFTR Function by Novel Small Molecules

The R334W (c.1000C>T, p.Arg334Trp) is a rare cystic fibrosis (CF)-causing mutation for which no causal therapy is currently approved. This mutation leads to a significant reduction of CF transmembrane conductance regulator (CFTR) channel conductance that still allows for residual function. Potent...

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Detalles Bibliográficos
Autores principales: Bacalhau, Mafalda, Ferreira, Filipa C., Silva, Iris A. L., Buarque, Camilla D., Amaral, Margarida D., Lopes-Pacheco, Miquéias
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9862739/
https://www.ncbi.nlm.nih.gov/pubmed/36675763
http://dx.doi.org/10.3390/jpm13010102