Functional rescue of an ABCB11 mutant by ivacaftor: A new targeted pharmacotherapy approach in bile salt export pump deficiency.
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| Title: | Functional rescue of an ABCB11 mutant by ivacaftor: A new targeted pharmacotherapy approach in bile salt export pump deficiency. |
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| Authors: | 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.; 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., Aït-Slimane T; Inserm, Centre de Recherche Saint-Antoine (CRSA), UMR_S 938, Institute of Cardiometabolism and Nutrition (ICAN), Sorbonne Université, Paris, 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, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, 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.; CHU Bicêtre, Biochemistry Unit, Assistance Publique-Hôpitaux de Paris, Le Kremlin-Bicêtre, France., Falguières T; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France., Callebaut I; Muséum National d'Histoire Naturelle, UMR CNRS 7590, Institut de Minéralogie de Physique des Matériaux et de Cosmochimie, IMPMC, Sorbonne Université, Paris, France., Gonzales E; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.; 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., Jacquemin E; Inserm, Physiopathogénèse et traitement des maladies du foie, UMR_S 1193, Hepatinov, Université Paris-Saclay, Orsay, France.; 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. |
| Source: | Liver international : official journal of the International Association for the Study of the Liver [Liver Int] 2020 Aug; Vol. 40 (8), pp. 1917-1925. Date of Electronic Publication: 2020 Jun 08. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Wiley-Blackwell Country of Publication: United States NLM ID: 101160857 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1478-3231 (Electronic) Linking ISSN: 14783223 NLM ISO Abbreviation: Liver Int Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1478-3231 |
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| DOI: | 10.1111/liv.14518 |
