Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
about
Potentiators (specific therapies for class III and IV mutations) for cystic fibrosisNew and emerging targeted therapies for cystic fibrosisTargeted therapies to improve CFTR function in cystic fibrosisTranslational readthrough potential of natural termination codons in eucaryotes--The impact of RNA sequenceNew approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicalsCystic fibrosis transmembrane conductance regulator dysfunction and its treatmentTherapeutic suppression of premature termination codons: mechanisms and clinical considerations (review)Correction of chloride transport and mislocalization of CFTR protein by vardenafil in the gastrointestinal tract of cystic fibrosis miceA synthetic chloride channel restores chloride conductance in human cystic fibrosis epithelial cellsUnderstanding protein kinase CK2 mis-regulation upon F508del CFTR expressionCystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectivesCFTR Modulators: Shedding Light on Precision Medicine for Cystic FibrosisCurrent and Emerging Therapies for the Treatment of Cystic Fibrosis or Mitigation of Its SymptomsA functional CFTR assay using primary cystic fibrosis intestinal organoidsAttenuation of nonsense-mediated mRNA decay enhances in vivo nonsense suppressionA lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assaysAminoglycoside-stimulated readthrough of premature termination codons in selected genes involved in primary ciliary dyskinesia.The unfolded protein response affects readthrough of premature termination codonsSynthetic aminoglycosides efficiently suppress cystic fibrosis transmembrane conductance regulator nonsense mutations and are enhanced by ivacaftorLumacaftor/ivacaftor combination for cystic fibrosis patients homozygous for Phe508del-CFTR.CFTR Modulators for the Treatment of Cystic Fibrosis.Aminoglycoside-Induced Premature Stop Codon Read-Through of Mucopolysaccharidosis Type I Patient Q70X and W402X Mutations in Cultured Cells.Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosisAtaluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.Class 1 CF MutationsState of progress in treating cystic fibrosis respiratory disease.Nanotechnology approaches for inhalation treatment of fibrosis.Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis.Ataluren treatment of patients with nonsense mutation dystrophinopathy.Apical CFTR expression in human nasal epithelium correlates with lung disease in cystic fibrosis.Characterization of nasal potential difference in cftr knockout and F508del-CFTR mice.Nonsense-mediated decay in genetic disease: friend or foe?High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomicsMulticenter intestinal current measurements in rectal biopsies from CF and non-CF subjects to monitor CFTR function.Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutationsAtaluren: first global approval.Outcome measures for clinical trials assessing treatment of cystic fibrosis lung disease.Suppression of premature termination codons as a therapeutic approach.A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation
P2860
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P2860
Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
description
2010 nî lūn-bûn
@nan
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
2010年论文
@zh
2010年论文
@zh-cn
name
Ataluren (PTC124) induces cyst ...... ense mutation cystic fibrosis.
@en
Ataluren
@nl
type
label
Ataluren (PTC124) induces cyst ...... ense mutation cystic fibrosis.
@en
Ataluren
@nl
prefLabel
Ataluren (PTC124) induces cyst ...... ense mutation cystic fibrosis.
@en
Ataluren
@nl
P2093
P2860
P1476
Ataluren (PTC124) induces cyst ...... ense mutation cystic fibrosis.
@en
P2093
Agnès Mogenet
Allen Reha
Delphine Roussel
François Vermeulen
Gary L Elfring
Georges J Casimir
Isabelle Sermet-Gaudelus
Janine Fritsch
Kris De Boeck
Langdon L Miller
P2860
P304
P356
10.1164/RCCM.201001-0137OC
P407
P577
2010-07-09T00:00:00Z