Systemic bis-phosphinic acid derivative restores chloride transport in Cystic Fibrosis mice.


Journal

Scientific reports
ISSN: 2045-2322
Titre abrégé: Sci Rep
Pays: England
ID NLM: 101563288

Informations de publication

Date de publication:
12 04 2022
Historique:
received: 01 08 2021
accepted: 28 02 2022
entrez: 13 4 2022
pubmed: 14 4 2022
medline: 15 4 2022
Statut: epublish

Résumé

Mutations in the Cystic Fibrosis Transmembrane Conductance Regulator gene (CFTR) are responsible for Cystic Fibrosis (CF). The most common CF-causing mutation is the deletion of the 508th amino-acid of CFTR (F508del), leading to dysregulation of the epithelial fluid transport in the airway's epithelium and the production of a thickened mucus favoring chronic bacterial colonization, sustained inflammation and ultimately respiratory failure. c407 is a bis-phosphinic acid derivative which corrects CFTR dysfunction in epithelial cells carrying the F508del mutation. This study aimed to investigate c407 in vivo activity in the F508del Cftr

Identifiants

pubmed: 35413967
doi: 10.1038/s41598-022-09678-9
pii: 10.1038/s41598-022-09678-9
pmc: PMC9005718
doi:

Substances chimiques

Chlorides 0
Phosphinic Acids 0
Cystic Fibrosis Transmembrane Conductance Regulator 126880-72-6

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

6132

Informations de copyright

© 2022. The Author(s).

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Auteurs

Mélanie Faria da Cunha (MF)

INSERM U1151, équipe 11, Paris, France.
Université de Paris, Paris, France.

Iwona Pranke (I)

INSERM U1151, équipe 11, Paris, France.
Université de Paris, Paris, France.

Ali Sassi (A)

INSERM U1151, équipe 11, Paris, France.
Université de Paris, Paris, France.

Christiane Schreiweis (C)

INSERM U1151, équipe 8, Paris, France.

Stéphanie Moriceau (S)

INSERM U1151, équipe 8, Paris, France.

Dragana Vidovic (D)

Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.

Aurélie Hatton (A)

INSERM U1151, équipe 11, Paris, France.
Université de Paris, Paris, France.

Mariane Sylvia Carlon (MS)

Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.

Geordie Creste (G)

Université de Paris, Paris, France.
UMR 8601, CNRS, LCBPT, Paris, France.

Farouk Berhal (F)

Université de Paris, Paris, France.
UMR 8601, CNRS, LCBPT, Paris, France.

Guillaume Prestat (G)

Université de Paris, Paris, France.
UMR 8601, CNRS, LCBPT, Paris, France.

Romain Freund (R)

Université de Paris, Paris, France.
Unité de Biostatistiques, Assistance Publique Hôpitaux de Paris, Paris, France.

Norbert Odolczyk (N)

Institute of Biochemistry and Biophysics, Polish Academy of Sciences, Warsaw, Poland.
Laboratory of Systems Biology, Institute of Experimental Plant Biology and Biotechnology, Warsaw, Poland.

Jean Philippe Jais (JP)

Unité de Biostatistiques, Assistance Publique Hôpitaux de Paris, Paris, France.

Christine Gravier-Pelletier (C)

Université de Paris, Paris, France.
UMR 8601, CNRS, LCBPT, Paris, France.

Piotr Zielenkiewicz (P)

Institute of Biochemistry and Biophysics, Polish Academy of Sciences, Warsaw, Poland.
Laboratory of Systems Biology, Institute of Experimental Plant Biology and Biotechnology, Warsaw, Poland.

Vincent Jullien (V)

Laboratoire de Bactériologie-Virologie-Hygiène, Hôpital Avicenne, Bobigny, France.

Alexandre Hinzpeter (A)

INSERM U1151, équipe 11, Paris, France.
Université de Paris, Paris, France.

Franck Oury (F)

Université de Paris, Paris, France.
INSERM U1151, équipe 8, Paris, France.

Aleksander Edelman (A)

INSERM U1151, équipe 11, Paris, France.
Université de Paris, Paris, France.

Isabelle Sermet-Gaudelus (I)

INSERM U1151, équipe 11, Paris, France. isabelle.sermet@aphp.fr.
Université de Paris, Paris, France. isabelle.sermet@aphp.fr.
Centre de Référence et de Compétence Maladies Rares, Mucoviscidose et maladies apparentées, Hôpital Necker Enfants malades, Paris, France. isabelle.sermet@aphp.fr.
European Reference Network for Rare Respiratory Diseases (ERN-LUNG), Brussels, Belgium. isabelle.sermet@aphp.fr.
Institut Necker Enfants Malades, 160 rue de Vaugirard, 75015, Paris, France. isabelle.sermet@aphp.fr.

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