Increasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule Therapeutics

W. Joon Chung; Jennifer L. Goeckeler-Fried; Viktoria Havasi; Annette Chiang; Steven M. Rowe; Zackery E. Plyler; Jeong S. Hong; Marina Mazur; Gary A. Piazza; Adam B. Keeton; E. Lucile White; Lynn Rasmussen; Allan M. Weissman; R. Aldrin Denny; Jeffrey L. Brodsky; Eric J. Sorscher

doi:10.1371/journal.pone.0163615

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Research Article

Increasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule Therapeutics

W. Joon Chung,

Affiliations Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States of America, Department of Neurobiology, University of Alabama at Birmingham, Birmingham, Alabama, United States of America
⨯
Jennifer L. Goeckeler-Fried,

Affiliation Department of Biological Sciences, University of Pittsburgh, Pittsburgh, Pennsylvania, United States of America
⨯
Viktoria Havasi,

Affiliation Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States of America
⨯
Annette Chiang,

Affiliation Department of Biological Sciences, University of Pittsburgh, Pittsburgh, Pennsylvania, United States of America
⨯
Steven M. Rowe,

Affiliations Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States of America, Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama, United States of America, Department of Cellular, Developmental and Integrative Biology, University of Alabama at Birmingham, Birmingham, Alabama, United States of America
⨯
Zackery E. Plyler,

Affiliation Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States of America
⨯
Jeong S. Hong,

Affiliations Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States of America, Department of Cellular, Developmental and Integrative Biology, University of Alabama at Birmingham, Birmingham, Alabama, United States of America
⨯
Marina Mazur,

Affiliation Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States of America
⨯
Gary A. Piazza,

Affiliation Oncologic Sciences, USA Mitchell Cancer Institute, University of South Alabama, Mobile, Alabama, United States of America
⨯
Adam B. Keeton,

Affiliation Oncologic Sciences, USA Mitchell Cancer Institute, University of South Alabama, Mobile, Alabama, United States of America
⨯
E. Lucile White,

Affiliation Drug Discovery Division, Southern Research Institute, Birmingham, Alabama, United States of America
⨯
Lynn Rasmussen,

Affiliation Drug Discovery Division, Southern Research Institute, Birmingham, Alabama, United States of America
⨯
Allan M. Weissman,

Affiliation Center for Cancer Research, National Institutes of Health, Frederick, Maryland, United States of America
⨯
R. Aldrin Denny,

Affiliation Worldwide Medicinal Chemistry, Pfizer, Cambridge, Massachusetts, United States of America
⨯
Jeffrey L. Brodsky,

Affiliation Department of Biological Sciences, University of Pittsburgh, Pittsburgh, Pennsylvania, United States of America
⨯
[ ... ],
Eric J. Sorscher

* E-mail: esorscher@emory.edu

Affiliation Department of Pediatrics, Emory University, Atlanta, Georgia, United States of America
⨯
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Increasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule Therapeutics

W. Joon Chung,
Jennifer L. Goeckeler-Fried,
Viktoria Havasi,
Annette Chiang,
Steven M. Rowe,
Zackery E. Plyler,
Jeong S. Hong,
Marina Mazur,
Gary A. Piazza,
Adam B. Keeton

Published: October 12, 2016
https://doi.org/10.1371/journal.pone.0163615

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