Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis.
about
Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosisAminoglycosides and other nonsense suppression therapies for the treatment of dystrophinopathyNew developments in aminoglycoside therapy and ototoxicityNew and emerging targeted therapies for cystic fibrosisTranslational readthrough potential of natural termination codons in eucaryotes--The impact of RNA sequenceCystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectivesNovel small molecules potentiate premature termination codon readthrough by aminoglycosidesPharmacological induction of CFTR function in patients with cystic fibrosis: mutation-specific therapy.Intracellular mechanisms of aminoglycoside-induced cytotoxicity.In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot studyAminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes.Synthetic aminoglycosides efficiently suppress cystic fibrosis transmembrane conductance regulator nonsense mutations and are enhanced by ivacaftorSafety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosisTargets for cystic fibrosis therapy: proteomic analysis and correction of mutant cystic fibrosis transmembrane conductance regulatorAtaluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.Class 1 CF MutationsPharmacokinetics and pharmacodynamics of aerosolized antibacterial agents in chronically infected cystic fibrosis patients.Translational read-through of the RP2 Arg120stop mutation in patient iPSC-derived retinal pigment epithelium cells.Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis.Pediatrics, surfactant, and cystic fibrosis in AJRCCM 2001.Towards the pharmacogenomics of cystic fibrosis.Nonsense-mediated decay in genetic disease: friend or foe?Readthrough of nonsense mutations in Rett syndrome: evaluation of novel aminoglycosides and generation of a new mouse modelPTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse modelA new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.Making sense of nonsense GABA(A) receptor mutations associated with genetic epilepsies.Gentamicin as gene therapy.Optimizing nasal potential difference analysis for CFTR modulator development: assessment of ivacaftor in CF subjects with the G551D-CFTR mutation.Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutationsThe phenotypic consequences of CFTR mutations.Suppression of nonsense mutations as a therapeutic approach to treat genetic diseases.Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54Emerging drug treatments for cystic fibrosis.Pharmacological approaches to correcting the ion transport defect in cystic fibrosis.Home management of haemophilia.No detectable improvements in cystic fibrosis transmembrane conductance regulator by nasal aminoglycosides in patients with cystic fibrosis with stop mutations.Cystic fibrosis lung disease: genetic influences, microbial interactions, and radiological assessment.Suppression of premature termination codons as a therapeutic approach.Understanding drug ototoxicity: molecular insights for prevention and clinical management.
P2860
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P2860
Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis.
description
2001 nî lūn-bûn
@nan
2001年の論文
@ja
2001年学术文章
@wuu
2001年学术文章
@zh
2001年学术文章
@zh-cn
2001年学术文章
@zh-hans
2001年学术文章
@zh-my
2001年学术文章
@zh-sg
2001年學術文章
@yue
2001年學術文章
@zh-hant
name
Evidence that systemic gentami ...... patients with cystic fibrosis.
@en
Evidence that systemic gentami ...... patients with cystic fibrosis.
@nl
type
label
Evidence that systemic gentami ...... patients with cystic fibrosis.
@en
Evidence that systemic gentami ...... patients with cystic fibrosis.
@nl
prefLabel
Evidence that systemic gentami ...... patients with cystic fibrosis.
@en
Evidence that systemic gentami ...... patients with cystic fibrosis.
@nl
P2093
P2860
P1476
Evidence that systemic gentami ...... patients with cystic fibrosis.
@en
P2093
Bedwell DM
Macaluso M
P2860
P304
P356
10.1164/AJRCCM.163.7.2004001
P407
P577
2001-06-01T00:00:00Z