Article
Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids.
The European respiratory journal - 1 Aug 2016
Dekkers Johanna F, Gogorza Gondra Ricardo A, Kruisselbrink Evelien, Vonk Annelotte M, Janssens Hettie M, de Winter-de Groot Karin M, van der Ent Cornelis K, Beekman Jeffrey M
Abstract excerpt
Small-molecule therapies that restore defects in cystic fibrosis transmembrane conductance regulator (CFTR) gating (potentiators) or trafficking (correctors) are being developed for cystic fibrosis (CF) in a mutation-specific fashion. Options for pharmacological correction of CFTR-p.Phe508del (F508del) are being extensively studied but correction of other trafficking mutants that may also benefit from corrector...
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