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Ataluren as an agent for therapeutic nonsense suppression.

Ataluren as an agent for therapeutic nonsense suppression.

http://www.wikidata.org/entity/Q37670599

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New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicals Therapeutic suppression of premature termination codons: mechanisms and clinical considerations (review)Blasticidin S inhibits translation by trapping deformed tRNA on the ribosome Nonsense-Mediated mRNA Decay: Degradation of Defective Transcripts Is Only Part of the Story Organizing principles of mammalian nonsense-mediated mRNA decay Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases Recent advances using zebrafish animal models for muscle disease drug discovery Aminoglycosides, 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 trials Toward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assay Protein 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 expression Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy Expanding rare disease drug trials based on shared molecular etiology Disease-proportional proteasomal degradation of missense dystrophins Selective 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.

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Ataluren as an agent for therapeutic nonsense suppression.

http://www.wikidata.org/entity/Q37670599

description

2012 nî lūn-bûn

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2012年の論文

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2012年学术文章

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2012年学术文章

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2012年学术文章

@zh-hans

2012年学术文章

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2012年学术文章

@zh-sg

2012年學術文章

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2012年學術文章

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2012年學術文章

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name

Ataluren as an agent for therapeutic nonsense suppression.

@en

Ataluren as an agent for therapeutic nonsense suppression.

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type

label

Ataluren as an agent for therapeutic nonsense suppression.

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Ataluren as an agent for therapeutic nonsense suppression.

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prefLabel

Ataluren as an agent for therapeutic nonsense suppression.

@en

Ataluren as an agent for therapeutic nonsense suppression.

@nl

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Annual Review of Medicine

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Ataluren as an agent for therapeutic nonsense suppression.

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P2093

Allan Jacobson

http://www.w3.org/2001/XMLSchema#string

Ellen M Welch

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Manal Morsy

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Stuart W Peltz

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P2860

Molecular mechanisms for the RNA-dependent ATPase activity of Upf1 and its regulation by Upf2

Interactions between UPF1, eRFs, PABP and the exon junction complex suggest an integrated model for mammalian NMD pathways

Upf1 ATPase-dependent mRNP disassembly is required for completion of nonsense- mediated mRNA decay

SMG-8 and SMG-9, two novel subunits of the SMG-1 complex, regulate remodeling of the mRNA surveillance complex during nonsense-mediated mRNA decay

The surveillance complex interacts with the translation release factors to enhance termination and degrade aberrant mRNAs

A competition between stimulators and antagonists of Upf complex recruitment governs human nonsense-mediated mRNA decay

RNA codewords and protein synthesis, VII. On the general nature of the RNA code

Guidelines for diagnosis of cystic fibrosis in newborns through older adults: Cystic Fibrosis Foundation consensus report

Recognition of cognate transfer RNA by the 30S ribosomal subunit

Structural basis of highly conserved ribosome recycling in eukaryotes and archaea

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