Personalized CFTR Modulator Therapy for

CFTR CFTR modulator G85E N1303K cystic fibrosis human nasal epithelial cells intestinal current measurement nasal potential difference

Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
02 Aug 2023
Historique:
received: 21 06 2023
revised: 28 07 2023
accepted: 31 07 2023
medline: 14 8 2023
pubmed: 12 8 2023
entrez: 12 8 2023
Statut: epublish

Résumé

CFTR modulator therapy with elexacaftor/tezacaftor/ivacaftor (ETI) has been approved for people with CF and at least one F508del allele in Europe. In the US, the ETI label has been expanded to 177 rare

Identifiants

pubmed: 37569738
pii: ijms241512365
doi: 10.3390/ijms241512365
pmc: PMC10418744
pii:
doi:

Substances chimiques

Cystic Fibrosis Transmembrane Conductance Regulator 126880-72-6
Chlorides 0
Benzodioxoles 0
CFTR protein, human 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Christiane Herzog Stiftung, Stuttgart, Germany and the Mukoviszidose Institut gGmbH, Bonn
ID : 2101 C-H-P
Organisme : Federal Ministry of Education and Research
ID : 82DZL009B1
Organisme : Deutsche Forschungsgemeinschaft
ID : CRC 1449 - project 431232613; sub-project Z02; and project 450557679

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Auteurs

Simon Y Graeber (SY)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.
Berlin Institute of Health (BIH) at Charité-Universitätsmedizin Berlin, 10117 Berlin, Germany.

Anita Balázs (A)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Niklas Ziegahn (N)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.

Tihomir Rubil (T)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Constanze Vitzthum (C)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Linus Piehler (L)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Marika Drescher (M)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Kathrin Seidel (K)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Alexander Rohrbach (A)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Jobst Röhmel (J)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.
Berlin Institute of Health (BIH) at Charité-Universitätsmedizin Berlin, 10117 Berlin, Germany.

Stephanie Thee (S)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.
Berlin Institute of Health (BIH) at Charité-Universitätsmedizin Berlin, 10117 Berlin, Germany.

Julia Duerr (J)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.

Marcus A Mall (MA)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.
Berlin Institute of Health (BIH) at Charité-Universitätsmedizin Berlin, 10117 Berlin, Germany.

Mirjam Stahl (M)

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 13353 Berlin, Germany.
German Centre for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany.
Berlin Institute of Health (BIH) at Charité-Universitätsmedizin Berlin, 10117 Berlin, Germany.

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