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Novel opportunities for CFTR-targeting drug development using organoids

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 treatmen...

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Detalhes bibliográficos
Main Authors: Dekkers, Johanna F, van der Ent, Cornelis K, Beekman, Jeffrey M
Formato: Artigo
Idioma:Inglês
Publicado em: Landes Bioscience 2013
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3915567/
https://ncbi.nlm.nih.gov/pubmed/25003014
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4161/rdis.27112
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