A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect. (Q38178772)

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5 March 2020

title

A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect (English)

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5 March 2020

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1

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5 March 2020

author name string

Kris De Boeck

series ordinal

2

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5 March 2020

publication date

30 January 2013

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The Lancet. Respiratory medicine

5 March 2020

published in

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5 March 2020

volume

1

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5 March 2020

page(s)

158-163

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5 March 2020

issue

2

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

5 March 2020

cites work

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

The cystic fibrosis transmembrane conductance regulator impedes proteolytic stimulation of the epithelial Na+ channel

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

A novel host defense system of airways is defective in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Airway epithelial cell inflammatory signalling in cystic fibrosis.

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Evidence for airway surface dehydration as the initiating event in CF airway disease

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Identification of the cystic fibrosis gene: genetic analysis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Molecular targeting of CFTR as a therapeutic approach to cystic fibrosis.

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Front-runners for pharmacotherapeutic correction of the airway ion transport defect in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

A CFTR potentiator in patients with cystic fibrosis and the G551D mutation

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Promising new era dawns for cystic fibrosis treatment

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

PTC124 targets genetic disorders caused by nonsense mutations

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Ivacaftor potentiation of multiple CFTR channels with gating mutations

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Newborn screening for cystic fibrosis in Alberta: Two years of experience

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

The very low penetrance of cystic fibrosis for the R117H mutation: a reappraisal for genetic counselling and newborn screening

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Therapeutics development for cystic fibrosis: a successful model for a multisystem genetic disease

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Guideline on the design and conduct of cystic fibrosis clinical trials: the European Cystic Fibrosis Society-Clinical Trials Network (ECFS-CTN).

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Lung disease associated with the IVS8 5T allele of the CFTR gene

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Early pulmonary manifestation of cystic fibrosis in children with the DeltaF508/R117H-7T genotype

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

Targeting F508del-CFTR to develop rational new therapies for cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2812%2970057-7

7 January 2021

10.1016/S2213-2600(12)70057-7

Identifiers

DOI

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24429096%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

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