The Autophagy Inhibitor Spautin-1 Antagonizes Rescue of Mutant CFTR Through an Autophagy-Independent and USP13-Mediated Mechanism

Specific autophagy and ESCRT components participate in the unconventional secretion of CFTR

(2018) Shin Hye Noh et al.   Autophagy

Noncanonical autophagy at ER exit sites regulates procollagen turnover

(2018) Shakib Omari et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

High-throughput screening identifies FAU protein as a regulator of mutant cystic fibrosis transmembrane conductance regulator channel

(2017) Valeria Tomati et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Direct Binding of the Corrector VX-809 to Human CFTR NBD1: Evidence of an Allosteric Coupling between the Binding Site and the NBD1:CL4 Interface

(2017) Rhea P. Hudson et al.   MOLECULAR PHARMACOLOGY

USP13 regulates the RAP80-BRCA1 complex dependent DNA damage response

(2017) Yunhui Li et al.   Nature Communications

Cystic fibrosis: a clinical view

(2016) Carlo Castellani et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

RNF5, DAB2 and Friends: Novel Drug Targets for Cystic Fibrosis

(2016) Elvira Sondo et al.   CURRENT PHARMACEUTICAL DESIGN

New and emerging targeted therapies for cystic fibrosis

(2016) Bradley S Quon et al.   BMJ-British Medical Journal

New and emerging targeted therapies for cystic fibrosis

(2016) Bradley S Quon et al.   BMJ-British Medical Journal

Origins of Cystic Fibrosis Lung Disease

(2015) David A. Stoltz et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genetic Inhibition Of The Ubiquitin Ligase Rnf5 Attenuates Phenotypes Associated To F508del Cystic Fibrosis Mutation

(2015) Valeria Tomati et al.   Scientific Reports

ΔF508 CFTR Surface Stability Is Regulated by DAB2 and CHIP-Mediated Ubiquitination in Post-Endocytic Compartments

(2015) Lianwu Fu et al.   PLoS One

A highly potent and selective Vps34 inhibitor alters vesicle trafficking and autophagy

(2014) Baptiste Ronan et al.   Nature Chemical Biology

Some gating potentiators, including VX-770, diminish  F508-CFTR functional expression

(2014) G. Veit et al.   Science Translational Medicine

Potentiator ivacaftor abrogates pharmacological correction of  F508 CFTR in cystic fibrosis

(2014) D. M. Cholon et al.   Science Translational Medicine

Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor

(2013) Xiaojiao Xue et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Revertants, Low Temperature, and Correctors Reveal the Mechanism of F508del-CFTR Rescue by VX-809 and Suggest Multiple Agents for Full Correction

(2013) Carlos M. Farinha et al.   CHEMISTRY & BIOLOGY

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1

(2013) Hong Yu Ren et al.   MOLECULAR BIOLOGY OF THE CELL

Deubiquitylation and stabilization of PTEN by USP13

(2013) Jinsong Zhang et al.   NATURE CELL BIOLOGY

Mechanism-based corrector combination restores ΔF508-CFTR folding and function

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

Managing the Underlying Cause of Cystic Fibrosis: A Future Role for Potentiators and Correctors

(2013) Luis J. V. Galietta   PEDIATRIC DRUGS

Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein

(2012) Lihua He et al.   FASEB JOURNAL

Association of TMEM16A chloride channel overexpression with airway goblet cell metaplasia

(2012) Paolo Scudieri et al.   JOURNAL OF PHYSIOLOGY-LONDON

Rescue of the mutant CFTR chloride channel by pharmacological correctors and low temperature analyzed by gene expression profiling

(2011) Elvira Sondo et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Rescue of ΔF508-CFTR Trafficking via a GRASP-Dependent Unconventional Secretion Pathway

(2011) Heon Yung Gee et al.   CELL

Beclin1 Controls the Levels of p53 by Regulating the Deubiquitination Activity of USP10 and USP13

(2011) Junli Liu et al.   CELL

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

(2011) Gergely L. Lukacs et al.   TRENDS IN MOLECULAR MEDICINE

Influence of cell background on pharmacological rescue of mutant CFTR

(2010) Nicoletta Pedemonte et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

c-Cbl Facilitates Endocytosis and Lysosomal Degradation of Cystic Fibrosis Transmembrane Conductance Regulator in Human Airway Epithelial Cells

(2010) Siying Ye et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Defective CFTR induces aggresome formation and lung inflammation in cystic fibrosis through ROS-mediated autophagy inhibition

(2010) Alessandro Luciani et al.   NATURE CELL BIOLOGY

Peripheral Protein Quality Control Removes Unfolded CFTR from the Plasma Membrane

(2010) T. Okiyoneda et al.   SCIENCE

The Deubiquitinating Enzyme USP10 Regulates the Post-endocytic Sorting of Cystic Fibrosis Transmembrane Conductance Regulator in Airway Epithelial Cells

(2009) Jennifer M. Bomberger et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

An ATP-competitive Mammalian Target of Rapamycin Inhibitor Reveals Rapamycin-resistant Functions of mTORC1

(2009) Carson C. Thoreen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Two Distinct Pathways for Cyclooxygenase-2 Protein Degradation

(2008) Uri R. Mbonye et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Gp78 Cooperates with RMA1 in Endoplasmic Reticulum-associated Degradation of CFTRΔF508

(2008) Daisuke Morito et al.   MOLECULAR BIOLOGY OF THE CELL