Aminoglycoside suppression of a premature stop mutation in a Cftr-/- mouse carrying a human CFTR-G542X transgene.
about
Aminoglycosides and other nonsense suppression therapies for the treatment of dystrophinopathyTranslational readthrough potential of natural termination codons in eucaryotes--The impact of RNA sequenceCystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectivesCFTR Modulators: Shedding Light on Precision Medicine for Cystic FibrosisEx vivo treatment with a novel synthetic aminoglycoside NB54 in primary fibroblasts from Rett syndrome patients suppresses MECP2 nonsense mutationsNovel small molecules potentiate premature termination codon readthrough by aminoglycosidesIn vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot studyDevelopment of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutationsSynthetic aminoglycosides efficiently suppress cystic fibrosis transmembrane conductance regulator nonsense mutations and are enhanced by ivacaftorNew trends in aminoglycosides use.Safety, 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 regulatorClass 1 CF MutationsAminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophyDevelopment of transgenic mice containing an introduced stop codon on the human methylmalonyl-CoA mutase locus.Gene therapy for muscular dystrophy: moving the field forward.Nonsense-mediated decay in genetic disease: friend or foe?PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse modelHigh 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.Rett syndrome like phenotypes in the R255X Mecp2 mutant mouse are rescued by MECP2 transgeneSuppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54No detectable improvements in cystic fibrosis transmembrane conductance regulator by nasal aminoglycosides in patients with cystic fibrosis with stop mutations.A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin.Chimeric constructs endow the human CFTR Cl- channel with the gating behavior of murine CFTRSuppression of premature termination codons as a therapeutic approach.Understanding drug ototoxicity: molecular insights for prevention and clinical management.Development of generic immunoassay for the detection of a series of aminoglycosides with 6'-OH group for the treatment of genetic diseases in biological samples.Production of beta-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous beta(0)39 thalassemia patients.Targeted therapy for cystic fibrosis: cystic fibrosis transmembrane conductance regulator mutation-specific pharmacologic strategies.Pharmacological read-through of nonsense ARSB mutations as a potential therapeutic approach for mucopolysaccharidosis VIRead-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophyWhat have we learned from mouse models for cystic fibrosis?On being a pathologist.Mice with missense and nonsense NF1 mutations display divergent phenotypes compared with human neurofibromatosis type I.Readthrough of nonsense mutation W822X in the SCN5A gene can effectively restore expression of cardiac Na+ channels.Animal models of chronic lung infection with Pseudomonas aeruginosa: useful tools for cystic fibrosis studies.Nonaminoglycoside compounds induce readthrough of nonsense mutations.Correction of ATM gene function by aminoglycoside-induced read-through of premature termination codons.
P2860
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P2860
Aminoglycoside suppression of a premature stop mutation in a Cftr-/- mouse carrying a human CFTR-G542X transgene.
description
2002 nî lūn-bûn
@nan
2002年の論文
@ja
2002年論文
@yue
2002年論文
@zh-hant
2002年論文
@zh-hk
2002年論文
@zh-mo
2002年論文
@zh-tw
2002年论文
@wuu
2002年论文
@zh
2002年论文
@zh-cn
name
Aminoglycoside suppression of ...... a human CFTR-G542X transgene.
@en
type
label
Aminoglycoside suppression of ...... a human CFTR-G542X transgene.
@en
prefLabel
Aminoglycoside suppression of ...... a human CFTR-G542X transgene.
@en
P2093
P1476
Aminoglycoside suppression of ...... g a human CFTR-G542X transgene
@en
P2093
Albert Tousson
Chitta R Dey
David M Bedwell
Eric J Sorscher
J Russell Lindsey
Jeffrey A Whitsett
Jessica Lanier
Julie R Jones
Kim M Keeling
Martin J Evans
P2888
P304
P356
10.1007/S00109-002-0363-1
P577
2002-07-03T00:00:00Z