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Therapeutic approaches for nonsense mutations in CFTR

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Year: 2026
Type: review
Source: Journal of Cystic Fibrosis
DOI: 10.1016/j.jcf.2026.02.014
Authors: Mairead Kelly-Aubert, Kari Thrasher, Andrei A. Korostelev, John D. Lueck, Isabelle Sermet-Gaudelus
Institutions: Centre National de la Recherche Scientifique, Inserm, Université Paris Cité, Institut Necker Enfants Malades, University of Rochester Medicine, University of Rochester, University of Massachusetts Chan Medical School, Hôpital Necker-Enfants Malades
Language: English

Topic: Cystic Fibrosis Research Advances
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
SDG: Good health and well-being

Open Access status: green

Funders: Cystic Fibrosis Foundation Therapeutics, Agence Nationale de la Recherche, National Institutes of Health, National Heart, Lung, and Blood Institute, National Institute of General Medical Sciences
Awards: G357962950, Multidisciplinary Training in Pulmonary Research, Disrupting abnormal protein-protein interactions to correct the functional defect of F508del-CFTR, In vivo delivery of engineered tRNAs for suppression of nonsense mutations, Translational Control: Discovery and Mechanisms
OpenAlex ID: W7133333035
FWCI: 1.287
Cites: 58
Cited by: 1
Related to: 10
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