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New and emerging targeted therapies for cystic fibrosis

Cystic fibrosis (CF) is a monogenic autosomal recessive disorder that affects about 70 000 people worldwide. The clinical manifestations of the disease are caused by defects in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The discovery of the CFTR gene in 1989 has led to a...

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Detalhes bibliográficos
Publicado no:BMJ
Main Authors: Quon, Bradley S, Rowe, Steven M
Formato: Artigo
Idioma:Inglês
Publicado em: BMJ Publishing Group Ltd. 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4817245/
https://ncbi.nlm.nih.gov/pubmed/27030675
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/bmj.i859
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