Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression-Reference-Cited by-同舟云学术

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression

Published:2014-07-23 Issue:246 Volume:6 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Veit Guido¹,Avramescu Radu G.¹,Perdomo Doranda¹,Phuan Puay-Wah²,Bagdany Miklos¹,Apaja Pirjo M.¹,Borot Florence¹,Szollosi Daniel³⁴,Wu Yu-Sheng¹,Finkbeiner Walter E.⁵,Hegedus Tamas³⁴,Verkman Alan S.²,Lukacs Gergely L.¹⁶⁷

Affiliation:

1. Department of Physiology, McGill University, Montréal, Quebec H3G 1Y6, Canada.

2. Departments of Medicine and Physiology, University of California, San Francisco, San Francisco, CA 94143–0521, USA.

3. MTA-SE Molecular Biophysics Research Group, Hungarian Academy of Sciences, 1444 Budapest, Hungary.

4. Department of Biophysics and Radiation Biology, Semmelweis University, 1444 Budapest P.O. Box 263, Hungary.

5. Department of Pathology, University of California, San Francisco, San Francisco, CA 94143–0511, USA.

6. Department of Biochemistry, McGill University, Montréal, Quebec H3G 1Y6, Canada.

7. Groupe de Recherche Axé sur la Structure des Protéines (GRASP), McGill University, Montréal, Quebec H3G 1Y6, Canada.

Abstract

Ivacaftor, a potentiator of ΔF508-CFTR channel function in cystic fibrosis, reduces the ability of corrector drugs to rescue the ΔF508-CFTR membrane trafficking defect.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference70 articles.

1. Cystic ibrosis: Molecular Biology and Therapeutic Implications

2. CFTR Function and Prospects for Therapy

3. Cystic Fibrosis Transmembrane Conductance Regulator Intracellular Processing, Trafficking, and Opportunities for Mutation-Specific Treatment

4. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis

5. The ΔF508 cystic fibrosis mutation impairs domain-domain interactions and arrests post-translational folding of CFTR

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