New Pharmacological Approaches to Treat Patients with Cystic Fibrosis with Nonsense Mutations. - National Genomics Data Center (CNCB-NGDC)

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New Pharmacological Approaches to Treat Patients with Cystic Fibrosis with Nonsense Mutations.

Isabelle Sermet-Gaudelus, Olivier Namy

Author Information

Isabelle Sermet-Gaudelus: 1 INSERM U 1151 Paris, France.
Olivier Namy: 3 Institut de Biologie Intégrative de la Cellule CEA, CNRS, Université Paris Sud, and Université Paris Saclay Paris, France.

PMID: 27797609 DOI: 10.1164/rccm.201605-1021ED

No abstract text available.

Codon, Nonsense

Cystic Fibrosis

Cystic Fibrosis Transmembrane Conductance Regulator

Humans

Mutation

Codon, Nonsense

Cystic Fibrosis Transmembrane Conductance Regulator

Journal Article Comment

Pharmacological approaches for targeting cystic fibrosis nonsense mutations.Pharmacological approaches to cystic fibrosis.Two patients with cystic fibrosis, nonsense mutations in each cystic fibrosis gene, and mild pulmonary disease.Novel pharmacological strategies to treat cystic fibrosis.New approaches to cystic fibrosis.New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls.Ataluren/ivacaftor combination therapy: Two N-of-1 trials in cystic fibrosis patients with nonsense mutations.[New therapeutic approaches to Cystic fibrosis].New approaches to optimizing antimicrobial therapy in patients with cystic fibrosis.Suppressing 'nonsense' in cystic fibrosis.

Pharmacological approaches for targeting cystic fibrosis nonsense mutations.Molecular mechanisms of cystic fibrosis - how mutations lead to misfunction and guide therapy.