(Q35482151)

8 August 2017

title

Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54 (English)

1 reference

8 August 2017

Steven M Rowe

1

1 reference

8 August 2017

Peter Sloane

2

1 reference

8 August 2017

Li Ping Tang

3

1 reference

8 August 2017

Kyle Backer

4

1 reference

8 August 2017

Marina Mazur

5

1 reference

8 August 2017

Jessica Buckley-Lanier

6

1 reference

8 August 2017

Igor Nudelman

7

1 reference

8 August 2017

Valery Belakhov

8

1 reference

8 August 2017

Zsuzsa Bebok

9

1 reference

8 August 2017

Erik Schwiebert

10

1 reference

8 August 2017

Timor Baasov

11

1 reference

8 August 2017

David M Bedwell

12

1 reference

8 August 2017

publication date

22 July 2011

1 reference

Journal of Molecular Medicine

8 August 2017

published in

1 reference

8 August 2017

volume

89

1 reference

8 August 2017

issue

11

1 reference

8 August 2017

page(s)

1149-1161

1 reference

DeltaF508 CFTR processing correction and activity in polarized airway and non-airway cell monolayers

8 August 2017

cites work

1 reference

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Activation of the cystic fibrosis transmembrane conductance regulator by the flavonoid quercetin: potential use as a biomarker of ΔF508 cystic fibrosis transmembrane conductance regulator rescue

12 July 2018

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

12 July 2018

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Pharmaceuticals targeting nonsense mutations in genetic diseases: progress in development

12 July 2018

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Activation of intestinal Cl- secretion by lubiprostone requires the cystic fibrosis transmembrane conductance regulator

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

In vitro and ex vivo suppression by aminoglycosides of PCDH15 nonsense mutations underlying type 1 Usher syndrome

12 July 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Restoration of W1282X CFTR activity by enhanced expression

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

No detectable improvements in cystic fibrosis transmembrane conductance regulator by nasal aminoglycosides in patients with cystic fibrosis with stop mutations

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

12 July 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Pharmacologic therapy for stop mutations: how much CFTR activity is enough?

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Efficient intracellular processing of the endogenous cystic fibrosis transmembrane conductance regulator in epithelial cell lines

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Gentamicin-Induced Correction of CFTR Function in Patients with Cystic Fibrosis andCFTRStop Mutations

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Aminoglycoside and its derivatives as ligands to target the ribosome

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Aminoglycoside suppression of a premature stop mutation in a Cftr-/- mouse carrying a human CFTR-G542X transgene.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

A(2) adenosine receptors regulate CFTR through PKA and PLA(2).

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-L-iduronidase activity and reduces lysosomal glycosaminoglycan accumulation

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Development of a novel trans-lentiviral vector that affords predictable safety

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Aminoglycosides: nephrotoxicity

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Cystic fibrosis transmembrane conductance regulator (CFTR) nucleotide-binding domain 1 (NBD-1) and CFTR truncated within NBD-1 target to the epithelial plasma membrane and increase anion permeability

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Suppression of a CFTR premature stop mutation in a bronchial epithelial cell line

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Proposed molecular and cellular mechanism for aminoglycoside ototoxicity

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

The efficiency of translation termination is determined by a synergistic interplay between upstream and downstream sequences in Saccharomyces cerevisiae.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Evidence that epithelial glycoprotein 330/megalin mediates uptake of polybasic drugs

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

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

12 July 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00109-011-0787-6

Mutations in the amino terminus of the cystic fibrosis transmembrane conductance regulator enhance endocytosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00109-011-0787-6

Production of a severe cystic fibrosis mutation in mice by gene targeting

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00109-011-0787-6

Poly-L-aspartic acid enhances and prolongs gentamicin-mediated suppression of the CFTR-G542X mutation in a cystic fibrosis mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00109-011-0787-6

Mutations in the cystic fibrosis transmembrane regulator gene and in vivo transepithelial potentials

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00109-011-0787-6

Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3204584

Normal function of the cystic fibrosis conductance regulator protein can be associated with homozygous (Delta)F508 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=3204584

A pilot study of the effect of gentamicin on nasal potential difference measurements in cystic fibrosis patients carrying stop mutations

26 September 2018

1 reference

12 December 2020

Correlation between genotype and phenotype in patients with cystic fibrosis

1 reference

12 December 2020

Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations

1 reference

12 December 2020

Clinical doses of amikacin provide more effective suppression of the human CFTR-G542X stop mutation than gentamicin in a transgenic CF mouse model

1 reference

12 December 2020

Redesign of aminoglycosides for treatment of human genetic diseases caused by premature stop mutations

1 reference

12 December 2020

10.1007/S00109-011-0787-6

Identifiers

DOI

1 reference

8 August 2017

1 reference

8 August 2017

1 reference