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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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Detalles Bibliográficos
Autores principales: Dekkers, Johanna F, van der Ent, Cornelis K, Beekman, Jeffrey M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Landes Bioscience 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3915567/
https://www.ncbi.nlm.nih.gov/pubmed/25003014
http://dx.doi.org/10.4161/rdis.27112