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Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis

The most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene leads to deletion of the phenylalanine at position 508 (ΔF508) in the CFTR protein and causes multiple folding and functional defects. Contrary to large-scale efforts by industry and academia, no signific...

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Detalles Bibliográficos
Autores principales: Bali, Vedrana, Lazrak, Ahmed, Guroji, Purushotham, Matalon, Sadis, Bebok, Zsuzsanna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4877091/
https://www.ncbi.nlm.nih.gov/pubmed/27214033
http://dx.doi.org/10.1371/journal.pone.0155882