A synonymous codon change alters the drug sensitivity of ΔF508 cystic fibrosis transmembrane conductance regulator

Influenza virus M2 targets cystic fibrosis transmembrane conductance regulator for lysosomal degradation during viral infection

(2015) James David Londino et al.   FASEB JOURNAL

Decoding mechanisms by which silent codon changes influence protein biogenesis and function

(2015) Vedrana Bali et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Synonymous Codon Usage Affects the Expression of Wild Type and F508del CFTR

(2015) Kalpit Shah et al.   JOURNAL OF MOLECULAR BIOLOGY

Alternative molecular formats and therapeutic applications for bispecific antibodies

(2015) Christoph Spiess et al.   MOLECULAR IMMUNOLOGY

Conservation of CFTR codon frequency through primates suggests synonymous mutations could have a functional effect

(2015) Lucilla Pizzo et al.   MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS

Translational tuning optimizes nascent protein folding in cells

(2015) S. J. Kim et al.   SCIENCE

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

(2015) Lianwu Fu et al.   PLoS One

Understanding the Influence of Codon Translation Rates on Cotranslational Protein Folding

(2014) Edward P. O’Brien et al.   ACCOUNTS OF CHEMICAL RESEARCH

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiators Protect G551D but Not ΔF508 CFTR from Thermal Instability

(2014) Xuehong Liu et al.   BIOCHEMISTRY

VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface

(2014) Paul D.W. Eckford et al.   CHEMISTRY & BIOLOGY

Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels

(2014) Z. Kopeikin et al.   Journal of Cystic Fibrosis

Interference with Ubiquitination in CFTR Modifies Stability of Core Glycosylated and Cell Surface Pools

(2014) S. Lee et al.   MOLECULAR AND CELLULAR 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

Exposing synonymous mutations

(2014) Ryan C. Hunt et al.   TRENDS IN GENETICS

Expression of codon optimized genes in microbial systems: current industrial applications and perspectives

(2014) Claudia Elena et al.   Frontiers in Microbiology

MDR1 Synonymous Polymorphisms Alter Transporter Specificity and Protein Stability in a Stable Epithelial Monolayer

(2013) King Leung Fung et al.   CANCER RESEARCH

Sequence and structure-specific elements of HERG mRNA determine channel synthesis and trafficking efficiency

(2013) Jakub Sroubek et al.   FASEB JOURNAL

The silent codon change I507-ATC→ATT contributes to the severity of the ΔF508 CFTR channel dysfunction

(2013) Ahmed Lazrak et al.   FASEB JOURNAL

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

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

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

Human Coding Synonymous Single Nucleotide Polymorphisms at Ramp Regions of mRNA Translation

(2013) Quan Li et al.   PLoS One

Building better drugs: developing and regulating engineered therapeutic proteins

(2013) Chava Kimchi-Sarfaty et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Cystic Fibrosis Transmembrane Regulator Correctors and Potentiators

(2013) S. M. Rowe et al.   Cold Spring Harbor Perspectives in Medicine

Thermal Instability of ΔF508 Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Channel Function: Protection by Single Suppressor Mutations and Inhibiting Channel Activity

(2012) Xuehong Liu et al.   BIOCHEMISTRY

Requirements for Efficient Correction of ΔF508 CFTR Revealed by Analyses of Evolved Sequences

(2012) Juan L. Mendoza et al.   CELL

Correction of Both NBD1 Energetics and Domain Interface Is Required to Restore ΔF508 CFTR Folding and Function

(2012) Wael M. Rabeh et al.   CELL

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

(2012) Lihua He et al.   FASEB JOURNAL

The Delicate Balance Between Secreted Protein Folding and Endoplasmic Reticulum-Associated Degradation in Human Physiology

(2012) Christopher J. Guerriero et al.   PHYSIOLOGICAL REVIEWS

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

Introduction to current and future protein therapeutics: A protein engineering perspective

(2011) Paul J. Carter   EXPERIMENTAL CELL RESEARCH

A Unified View of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gating: Combining the Allosterism of a Ligand-gated Channel with the Enzymatic Activity of an ATP-binding Cassette (ABC) Transporter

(2011) Kevin L. Kirk et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Thermally Unstable Gating of the Most Common Cystic Fibrosis Mutant Channel (ΔF508)

(2011) Wei Wang et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Understanding the contribution of synonymous mutations to human disease

(2011) Zuben E. Sauna et al.   NATURE REVIEWS GENETICS

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

A Synonymous Single Nucleotide Polymorphism in ΔF508CFTRAlters the Secondary Structure of the mRNA and the Expression of the Mutant Protein

(2010) Rafal A. Bartoszewski et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Synonymous but not the same: the causes and consequences of codon bias

(2010) Joshua B. Plotkin et al.   NATURE REVIEWS GENETICS

Thermal unfolding studies show the disease causing F508del mutation in CFTR thermodynamically destabilizes nucleotide-binding domain 1

(2010) Irina Protasevich et al.   PROTEIN SCIENCE

Functional Stability of Rescued ΔF508 Cystic Fibrosis Transmembrane Conductance Regulator in Airway Epithelial Cells

(2009) Asta Jurkuvenaite et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Structure and Dynamics of NBD1 from CFTR Characterized Using Crystallography and Hydrogen/Deuterium Exchange Mass Spectrometry

(2009) H.A. Lewis et al.   JOURNAL OF MOLECULAR BIOLOGY

Mechanisms for Rescue of Correctable Folding Defects in CFTRΔF508

(2009) Diane E. Grove et al.   MOLECULAR BIOLOGY OF THE CELL

Cooperative Assembly and Misfolding of CFTR Domains In Vivo

(2009) Kai Du et al.   MOLECULAR BIOLOGY OF THE CELL

Activation of the Unfolded Protein Response by ΔF508 CFTR

(2008) Rafal Bartoszewski et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

CFTR Function and Prospects for Therapy

(2008) John R. Riordan   Annual Review of Biochemistry

Correctors promote folding of the CFTR in the endoplasmic reticulum

(2008) Tip W. Loo et al.   BIOCHEMICAL JOURNAL

Synonymous Mutations and Ribosome Stalling Can Lead to Altered Folding Pathways and Distinct Minima

(2008) Chung-Jung Tsai et al.   JOURNAL OF MOLECULAR BIOLOGY

One step at a time: endoplasmic reticulum-associated degradation

(2008) Shruthi S. Vembar et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Protein therapeutics: a summary and pharmacological classification

(2007) Benjamin Leader et al.   NATURE REVIEWS DRUG DISCOVERY