(Q35558817)

8 August 2017

title

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

1 reference

8 August 2017

Fredrick Van Goor

1

1 reference

8 August 2017

Sabine Hadida

2

1 reference

8 August 2017

Peter D J Grootenhuis

3

1 reference

8 August 2017

Bill Burton

4

1 reference

8 August 2017

Jeffrey H Stack

5

1 reference

8 August 2017

Kimberly S Straley

6

1 reference

8 August 2017

Caroline J Decker

7

1 reference

8 August 2017

Mark Miller

8

1 reference

8 August 2017

Jason McCartney

9

1 reference

8 August 2017

Eric R Olson

10

1 reference

8 August 2017

Jeffrey J Wine

11

1 reference

8 August 2017

Ray A Frizzell

12

1 reference

8 August 2017

Melissa Ashlock

13

1 reference

8 August 2017

Paul A Negulescu

14

1 reference

8 August 2017

language of work or name

English

0 references

publication date

5 October 2011

1 reference

Proceedings of the National Academy of Sciences of the United States of America

8 August 2017

published in

1 reference

8 August 2017

volume

108

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8 August 2017

issue

46

1 reference

8 August 2017

page(s)

18843-18848

1 reference

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.

8 August 2017

cites work

1 reference

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Peripheral protein quality control removes unfolded CFTR from the plasma membrane

27 July 2018

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Influence of cell background on pharmacological rescue of mutant CFTR.

27 July 2018

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Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

27 July 2018

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27 July 2018

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Chloride channels as drug targets

27 July 2018

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High treatment burden in adults with cystic fibrosis: challenges to disease self-management

27 July 2018

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27 July 2018

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Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice

27 July 2018

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Parallel improvement of sodium and chloride transport defects by miglustat (n-butyldeoxynojyrimicin) in cystic fibrosis epithelial cells.

27 July 2018

1 reference

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Phenylalanine-508 mediates a cytoplasmic-membrane domain contact in the CFTR 3D structure crucial to assembly and channel function

27 July 2018

1 reference

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Detection of cystic fibrosis transmembrane conductance regulator activity in early-phase clinical trials

27 July 2018

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Cystic Fibrosis: Lessons from the Sweat Gland

27 July 2018

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Additive effect of multiple pharmacological chaperones on maturation of CFTR processing mutants

27 July 2018

1 reference

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Cystic fibrosis: a review of epidemiology and pathobiology

27 July 2018

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Domain interdependence in the biosynthetic assembly of CFTR.

27 July 2018

1 reference

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Alpha-1-C-octyl-1-deoxynojirimycin as a pharmacological chaperone for Gaucher disease.

27 July 2018

1 reference

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The role of cystic fibrosis transmembrane conductance regulator phenylalanine 508 side chain in ion channel gating

27 July 2018

1 reference

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Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Rescue of DeltaF508 and other misprocessed CFTR mutants by a novel quinazoline compound

27 July 2018

1 reference

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HERG channel trafficking.

27 July 2018

1 reference

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Side chain and backbone contributions of Phe508 to CFTR folding

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

The DeltaF508 cystic fibrosis mutation impairs domain-domain interactions and arrests post-translational folding of CFTR.

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Probing protein structure by limited proteolysis.

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Cystic fibrosis: a worldwide analysis of CFTR mutations--correlation with incidence data and application to screening

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

The role of a conserved inter-transmembrane domain interface in regulating alpha(2a)-adrenergic receptor conformational stability and cell-surface turnover

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Chemical chaperones mediate increased secretion of mutant alpha 1-antitrypsin (alpha 1-AT) Z: A potential pharmacological strategy for prevention of liver injury and emphysema in alpha 1-AT deficiency

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Conformational maturation of CFTR but not its mutant counterpart (delta F508) occurs in the endoplasmic reticulum and requires ATP

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Altered protein folding may be the molecular basis of most cases of cystic fibrosis

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Abnormal localization of cystic fibrosis transmembrane conductance regulator in primary cultures of cystic fibrosis airway epithelia

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Cystic fibrosis: a disease of vulnerability to airway surface dehydration.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Destabilizing mutations promote membrane protein misfolding

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Protein folding stability can determine the efficiency of escape from endoplasmic reticulum quality control

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

Altered chloride ion channel kinetics associated with the ΔF508 cystic fibrosis mutation

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3219147

The delta F508 mutation decreases the stability of cystic fibrosis transmembrane conductance regulator in the plasma membrane. Determination of functional half-lives on transfected cells

7 December 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21976485

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21976485

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Limited proteolysis as a probe for arrested conformational maturation of delta F508 CFTR

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21976485

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1073/PNAS.1105787108

1 reference

8 August 2017

0 references

1 reference

8 August 2017

1 reference