Functional rescue of an ABCB11 mutant by ivacaftor: A new targeted pharmacotherapy approach in bile salt export pump deficiency.

Mareux, Elodie; Lapalus, Martine; Amzal, Rachida; Almes, Marion; Aït-Slimane, Tounsia; Delaunay, Jean-Louis; Adnot, Pauline; Collado-Hilly, Mauricette; Davit-Spraul, Anne; Falguières, Thomas; Callebaut, Isabelle; Gonzales, Emmanuel; Jacquemin, Emmanuel

Mareux, Elodie; Lapalus, Martine; Amzal, Rachida; Almes, Marion; Aït-Slimane, Tounsia; Delaunay, Jean-Louis; Adnot, Pauline; Collado-Hilly, Mauricette; Davit-Spraul, Anne; Falguières, Thomas; Callebaut, Isabelle; Gonzales, Emmanuel; Jacquemin, Emmanuel.

Afiliação

Mareux E; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Lapalus M; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Amzal R; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Almes M; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Aït-Slimane T; Paediatric Hepatology & Paediatric Liver Transplant Department, Reference Center for Rare Paediatric Liver Diseases, FILFOIE, ERN RARE LIVER, Assistance Publique-Hôpitaux de Paris, Faculty of Medicine Paris-Saclay, CHU Bicêtre, Le Kremlin-Bicêtre, France.
Delaunay JL; Inserm, Centre de Recherche Saint-Antoine (CRSA), UMR_S 938, Institute of Cardiometabolism and Nutrition (ICAN), Sorbonne Université, Paris, France.
Adnot P; Inserm, Centre de Recherche Saint-Antoine (CRSA), UMR_S 938, Institute of Cardiometabolism and Nutrition (ICAN), Sorbonne Université, Paris, France.
Collado-Hilly M; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Davit-Spraul A; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Falguières T; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Callebaut I; CHU Bicêtre, Biochemistry Unit, Assistance Publique-Hôpitaux de Paris, Le Kremlin-Bicêtre, France.
Gonzales E; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.
Jacquemin E; Muséum National d'Histoire Naturelle, UMR CNRS 7590, Institut de Minéralogie de Physique des Matériaux et de Cosmochimie, IMPMC, Sorbonne Université, Paris, France.

Liver Int ; 40(8): 1917-1925, 2020 08.

Article em En | MEDLINE | ID: mdl-32433800

Assuntos

Colestase Intra-Hepática; Quinolonas; Membro 11 da Subfamília B de Transportadores de Cassetes de Ligação de ATP/genética; Aminofenóis; Animais; Ácidos e Sais Biliares; Colestase Intra-Hepática/tratamento farmacológico; Colestase Intra-Hepática/genética; Cães; Humanos; Ratos

Palavras-chave

ABC transporters superfamily; PFIC2; VX-770; cholestatic liver diseases; paediatrics; potentiator

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Colestase Intra-Hepática / Quinolonas Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Colestase Intra-Hepática / Quinolonas Idioma: En Ano de publicação: 2020 Tipo de documento: Article