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• W2029151587 endingPage "e27112" @default.
• W2029151587 startingPage "e27112" @default.
• W2029151587 abstract "Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR mutations lead to production of non-functional CFTR, reduced amounts of normal functioning CFTR or misfolded CFTR with defects in trafficking or function. For decades, CF treatment has been focused on the symptoms of CF, but pharmacotherapy using small molecules that target the basic defect of CF, the mutant CFTR protein, is now possible for a limited amount of subjects with CF. This raises the exciting possibility that the majority of people with CF may receive effective treatment targeting the different CFTR mutants in the future. We recently described a functional CFTR assay using rectal biopsies from subjects with CF that were cultured in vitro into self-organizing mini-guts or organoids. We here describe how this model may assist in the discovery of new CFTR-targeting drugs, the subjects that may benefit from these drugs, and the mechanisms underlying variability in CFTR genotype-phenotype relations." @default.
• W2029151587 created "2016-06-24" @default.
• W2029151587 creator A5015719837 @default.
• W2029151587 creator A5027077813 @default.
• W2029151587 creator A5052340428 @default.
• W2029151587 date "2013-01-01" @default.
• W2029151587 modified "2023-10-16" @default.
• W2029151587 title "Novel opportunities for CFTR-targeting drug development using organoids" @default.
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• W2029151587 doi "https://doi.org/10.4161/rdis.27112" @default.
• W2029151587 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/3915567" @default.
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• W2029151587 hasPublicationYear "2013" @default.
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