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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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Publicado no: | BMJ |
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Main Authors: | , |
Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
BMJ Publishing Group Ltd.
2016
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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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