Ataluren as an agent for therapeutic nonsense suppression.
about
New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicalsTherapeutic suppression of premature termination codons: mechanisms and clinical considerations (review)Blasticidin S inhibits translation by trapping deformed tRNA on the ribosomeNonsense-Mediated mRNA Decay: Degradation of Defective Transcripts Is Only Part of the StoryOrganizing principles of mammalian nonsense-mediated mRNA decayEvaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage DiseasesRecent advances using zebrafish animal models for muscle disease drug discoveryAminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes.NBCe1 as a model carrier for understanding the structure-function properties of Na⁺ -coupled SLC4 transporters in health and disease.Translational read-through as an alternative approach for ocular gene therapy of retinal dystrophies caused by in-frame nonsense mutations.Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trialsToward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assayProtein Mis-Termination Initiates Genetic Diseases, Cancers, and Restricts Bacterial Genome Expansion.Lumacaftor and ivacaftor in the management of patients with cystic fibrosis: current evidence and future prospects.Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.Mice homozygous for c.451C>T mutation in Cln1 gene recapitulate INCL phenotype.Identification of SMG6 cleavage sites and a preferred RNA cleavage motif by global analysis of endogenous NMD targets in human cells.Nonsense suppression by near-cognate tRNAs employs alternative base pairing at codon positions 1 and 3.NMD: At the crossroads between translation termination and ribosome recycling.Nonsense-mediated RNA decay--a switch and dial for regulating gene expressionLarge-scale serum protein biomarker discovery in Duchenne muscular dystrophyExpanding rare disease drug trials based on shared molecular etiologyDisease-proportional proteasomal degradation of missense dystrophinsSelective stalling of human translation through small-molecule engagement of the ribosome nascent chain.Premature termination codon read-through in the ABCC6 gene: potential treatment for pseudoxanthoma elasticum.The intimate relationships of mRNA decay and translation.Cystic fibrosis - a multiorgan protein misfolding disease.Expectations and experiences of investigators and parents involved in a clinical trial for Duchenne/Becker muscular dystrophy.Therapeutics based on stop codon readthrough.Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases.New innovations: therapeutic opportunities for intellectual disabilities.Gene-based therapies of neuromuscular disorders: an update and the pivotal role of patient organizations in their discovery and implementation.The functional consequences of intron retention: alternative splicing coupled to NMD as a regulator of gene expression.Nonsense suppression therapies in ocular genetic diseases.The nucleoside analog clitocine is a potent and efficacious readthrough agent.Mechanism, factors, and physiological role of nonsense-mediated mRNA decay.Targeting Nonsense Mutations in Diseases with Translational Read-Through-Inducing Drugs (TRIDs).Precision medicine, genomics and drug discovery.Rescue of the MERTK phagocytic defect in a human iPSC disease model using translational read-through inducing drugs.
P2860
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P2860
Ataluren as an agent for therapeutic nonsense suppression.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年学术文章
@wuu
2012年学术文章
@zh-cn
2012年学术文章
@zh-hans
2012年学术文章
@zh-my
2012年学术文章
@zh-sg
2012年學術文章
@yue
2012年學術文章
@zh
2012年學術文章
@zh-hant
name
Ataluren as an agent for therapeutic nonsense suppression.
@en
Ataluren as an agent for therapeutic nonsense suppression.
@nl
type
label
Ataluren as an agent for therapeutic nonsense suppression.
@en
Ataluren as an agent for therapeutic nonsense suppression.
@nl
prefLabel
Ataluren as an agent for therapeutic nonsense suppression.
@en
Ataluren as an agent for therapeutic nonsense suppression.
@nl
P2093
P2860
P1476
Ataluren as an agent for therapeutic nonsense suppression.
@en
P2093
Allan Jacobson
Ellen M Welch
Manal Morsy
Stuart W Peltz
P2860
P304
P356
10.1146/ANNUREV-MED-120611-144851
P577
2012-11-28T00:00:00Z