Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons
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Title
Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons
Authors
Keywords
Translational readthrough, Stop codon suppression, Nonsense suppression, Premature termination codon, Genetic diseases
Journal
MOLECULAR MEDICINE
Volume 24, Issue 1, Pages -
Publisher
Springer Nature
Online
2018-05-29
DOI
10.1186/s10020-018-0024-7
References
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Note: Only part of the references are listed.- Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences
- (2017) Xiaojiao Xue et al. HUMAN MOLECULAR GENETICS
- Gentamicin induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients
- (2017) David T. Woodley et al. JOURNAL OF CLINICAL INVESTIGATION
- Amlexanox Enhances Premature Termination Codon Read-Through in COL7A1 and Expression of Full Length Type VII Collagen: Potential Therapy for Recessive Dystrophic Epidermolysis Bullosa
- (2017) Velina S. Atanasova et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
- (2017) Craig M McDonald et al. LANCET
- In vitro translational readthrough by gentamicin and geneticin improves GLA activity in Fabry disease
- (2017) Halil Dündar et al. MOLECULAR GENETICS AND METABOLISM
- Characterization of new-generation aminoglycoside promoting premature termination codon readthrough in cancer cells
- (2017) Laure Bidou et al. RNA Biology
- The nucleoside analog clitocine is a potent and efficacious readthrough agent
- (2017) Westley J. Friesen et al. RNA
- Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations
- (2016) Venkateshwar Mutyam et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- Targeting Nonsense Mutations in Diseases with Translational Read-Through-Inducing Drugs (TRIDs)
- (2016) Kerstin Nagel-Wolfrum et al. BIODRUGS
- Novel small molecules potentiate premature termination codon readthrough by aminoglycosides
- (2016) Alireza Baradaran-Heravi et al. NUCLEIC ACIDS RESEARCH
- Synthesis and activity of a novel inhibitor of nonsense-mediated mRNA decay
- (2016) Victoria J. B. Gotham et al. ORGANIC & BIOMOLECULAR CHEMISTRY
- Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression
- (2016) Bijoyita Roy et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Aminoglycoside-stimulated readthrough of premature termination codons in selected genes involved in primary ciliary dyskinesia
- (2016) Zuzanna Bukowy-Bieryllo et al. RNA Biology
- Rules of UGA-N decoding by near-cognate tRNAs and analysis of readthrough on short uORFs in yeast
- (2016) Petra Beznosková et al. RNA
- NMD: At the crossroads between translation termination and ribosome recycling
- (2015) Alper Celik et al. BIOCHIMIE
- A flow cytometry-based reporter assay identifies macrolide antibiotics as nonsense mutation read-through agents
- (2015) Michal Caspi et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- Nonsense suppression by near-cognate tRNAs employs alternative base pairing at codon positions 1 and 3
- (2015) Bijoyita Roy et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Translational readthrough potential of natural termination codons in eucaryotes – The impact of RNA sequence
- (2015) Maciej Dabrowski et al. RNA Biology
- Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases
- (2015) Marta Gómez-Grau et al. PLoS One
- Therapeutics Based on Stop Codon Readthrough
- (2014) Kim M. Keeling et al. Annual Review of Genomics and Human Genetics
- Rescue of wild-type E-cadherin expression from nonsense-mutated cancer cells by a suppressor-tRNA
- (2014) Renata Bordeira-Carriço et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Toward a Rationale for the PTC124 (Ataluren) Promoted Readthrough of Premature Stop Codons: A Computational Approach and GFP-Reporter Cell-Based Assay
- (2014) Laura Lentini et al. MOLECULAR PHARMACEUTICS
- Aminoglycosides Restore Full-length Type VII Collagen by Overcoming Premature Termination Codons: Therapeutic Implications for Dystrophic Epidermolysis Bullosa
- (2014) Jon Cogan et al. MOLECULAR THERAPY
- Ataluren treatment of patients with nonsense mutation dystrophinopathy
- (2014) Katharine Bushby et al. MUSCLE & NERVE
- Nonsense-mediated decay in genetic disease: Friend or foe?
- (2014) Jake N. Miller et al. MUTATION RESEARCH-REVIEWS IN MUTATION RESEARCH
- Evidence of efficient stop codon readthrough in four mammalian genes
- (2014) Gary Loughran et al. NUCLEIC ACIDS RESEARCH
- Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial
- (2014) Eitan Kerem et al. Lancet Respiratory Medicine
- Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor
- (2013) Xiaojiao Xue et al. AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY
- Designer Aminoglycosides That Selectively Inhibit Cytoplasmic Rather than Mitochondrial Ribosomes Show Decreased Ototoxicity
- (2013) Eli Shulman et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Long-term nonsense suppression therapy moderates MPS I-H disease progression
- (2013) Gwen Gunn et al. MOLECULAR GENETICS AND METABOLISM
- A New Series of Small Molecular Weight Compounds Induce Read Through of All Three Types of Nonsense Mutations in the ATM Gene
- (2013) Liutao Du et al. MOLECULAR THERAPY
- Attenuation of Nonsense-Mediated mRNA Decay Enhances In Vivo Nonsense Suppression
- (2013) Kim M. Keeling et al. PLoS One
- Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy
- (2013) Richard S. Finkel et al. PLoS One
- SMRT compounds correct nonsense mutations in primary immunodeficiency and other genetic models
- (2012) Richard A. Gatti Annals of the New York Academy of Sciences
- Structure-Activity Relationships among the Kanamycin Aminoglycosides: Role of Ring I Hydroxyl and Amino Groups
- (2012) Sumantha Salian et al. ANTIMICROBIAL AGENTS AND CHEMOTHERAPY
- Bumetanide Hyperpolarizes Madin–Darby Canine Kidney Cells and Enhances Cellular Gentamicin Uptake by Elevating Cytosolic Ca2+ Thus Facilitating Intermediate Conductance Ca2+-Activated Potassium Channels
- (2012) Tian Wang et al. CELL BIOCHEMISTRY AND BIOPHYSICS
- Suppression of premature termination codons as a therapeutic approach
- (2012) Kim M. Keeling et al. CRITICAL REVIEWS IN BIOCHEMISTRY AND MOLECULAR BIOLOGY
- A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of anUSH1Cnonsense mutation
- (2012) Tobias Goldmann et al. EMBO Molecular Medicine
- Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy
- (2012) Refik Kayali et al. HUMAN MOLECULAR GENETICS
- Analysis of a read-through promoting compound in a severe mouse model of spinal muscular atrophy
- (2012) Virginia B. Mattis et al. NEUROSCIENCE LETTERS
- Rescue of nonsense mutations by amlexanox in human cells
- (2012) Sara Gonzalez-Hilarion et al. Orphanet Journal of Rare Diseases
- Pharmaceutical therapies to recode nonsense mutations in inherited diseases
- (2012) Hui-Ling Rose Lee et al. PHARMACOLOGY & THERAPEUTICS
- Endocytic Receptors in the Renal Proximal Tubule
- (2012) Erik I. Christensen et al. PHYSIOLOGY
- Statistical Analysis of Readthrough Levels for Nonsense Mutations in Mammalian Cells Reveals a Major Determinant of Response to Gentamicin
- (2012) Célia Floquet et al. PLoS Genetics
- Effective Drug Delivery System for Duchenne Muscular Dystrophy Using Hybrid Liposomes Including Gentamicin along with Reduced Toxicity
- (2011) Mamiko Yukihara et al. BIOLOGICAL & PHARMACEUTICAL BULLETIN
- Synthesis and evaluation of compounds that induce readthrough of premature termination codons
- (2011) Michael E. Jung et al. BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
- Evidence of abundant stop codon readthrough in Drosophila and other metazoa
- (2011) I. Jungreis et al. GENOME RESEARCH
- New developments in aminoglycoside therapy and ototoxicity
- (2011) Jing Xie et al. HEARING RESEARCH
- Functional characterization and targeted correction of ATM mutations identified in Japanese patients with ataxia-telangiectasia
- (2011) Kotoka Nakamura et al. HUMAN MUTATION
- TRPA1-Mediated Accumulation of Aminoglycosides in Mouse Cochlear Outer Hair Cells
- (2011) Ruben S. Stepanyan et al. JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY
- Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54
- (2011) Steven M. Rowe et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients
- (2011) Chinmoy Sarkar et al. MOLECULAR GENETICS AND METABOLISM
- The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse
- (2011) Dan Wang et al. MOLECULAR GENETICS AND METABOLISM
- Rescue of Melanocortin 4 Receptor (MC4R) Nonsense Mutations by Aminoglycoside-Mediated Read-Through
- (2011) Harald Brumm et al. Obesity
- Ex Vivo Treatment with a Novel Synthetic Aminoglycoside NB54 in Primary Fibroblasts from Rett Syndrome Patients Suppresses MECP2 Nonsense Mutations
- (2011) Manuela Vecsler et al. PLoS One
- Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis
- (2010) Isabelle Sermet-Gaudelus et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy
- (2010) Vinod Malik et al. ANNALS OF NEUROLOGY
- Repairing faulty genes by aminoglycosides: Development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations
- (2010) Igor Nudelman et al. BIOORGANIC & MEDICINAL CHEMISTRY
- Upf1 Senses 3′UTR Length to Potentiate mRNA Decay
- (2010) J. Robert Hogg et al. CELL
- Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression
- (2010) Bingjing Wang et al. JOURNAL OF APPLIED PHYSIOLOGY
- Readthrough of nonsense mutations in Rett syndrome: evaluation of novel aminoglycosides and generation of a new mouse model
- (2010) Cornelia Brendel et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- New insights into the mechanism of aminoglycoside nephrotoxicity: an integrative point of view
- (2010) Jose M. Lopez-Novoa et al. KIDNEY INTERNATIONAL
- Rescue of non-sense mutated p53 tumor suppressor gene by aminoglycosides
- (2010) Célia Floquet et al. NUCLEIC ACIDS RESEARCH
- The Effect of Gentamicin-Induced Readthrough on a Novel Premature Termination Codon of CD18 Leukocyte Adhesion Deficiency Patients
- (2010) Amos J. Simon et al. PLoS One
- Production of β-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous β039 thalassemia patients
- (2009) Francesca Salvatori et al. AMERICAN JOURNAL OF HEMATOLOGY
- Cubilin and Megalin Co-Localize in the Neonatal Inner Ear
- (2009) Jacob Tauris et al. AUDIOLOGY AND NEURO-OTOLOGY
- In vitro readthrough of termination codons by gentamycin in the Stüve–Wiedemann Syndrome
- (2009) Samuel Bellais et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Restoration of APC gene function in colorectal cancer cells by aminoglycoside- and macrolide-induced read-through of premature termination codons
- (2009) A. Zilberberg et al. GUT
- Aminoglycoside antibiotics restore functional expression of truncated HERG channels produced by nonsense mutations
- (2009) Yan Yao et al. HEART RHYTHM
- Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model
- (2009) Virginia B. Mattis et al. HUMAN MOLECULAR GENETICS
- Poly-l-aspartic Acid Enhances and Prolongs Gentamicin-mediated Suppression of theCFTR-G542X Mutation in a Cystic Fibrosis Mouse Model
- (2009) Ming Du et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Nonaminoglycoside compounds induce readthrough of nonsense mutations
- (2009) Liutao Du et al. JOURNAL OF EXPERIMENTAL MEDICINE
- Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations
- (2009) Igor Nudelman et al. JOURNAL OF MEDICINAL CHEMISTRY
- Recognition of nonsense mRNA: towards a unified model: Figure 1
- (2008) Oliver Mühlemann BIOCHEMICAL SOCIETY TRANSACTIONS
- Interactions between UPF1, eRFs, PABP and the exon junction complex suggest an integrated model for mammalian NMD pathways
- (2008) Pavel V Ivanov et al. EMBO JOURNAL
- Systemic aminoglycoside treatment in rodent models of retinitis pigmentosa
- (2008) K. Guerin et al. EXPERIMENTAL EYE RESEARCH
- A meta-analysis of nonsense mutations causing human genetic disease
- (2008) Matthew Mort et al. HUMAN MUTATION
- Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial
- (2008) Eitan Kerem et al. LANCET
- PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model
- (2008) M. Du et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Genetic analysis of interactions with eukaryotic rRNA identify the mitoribosome as target in aminoglycoside ototoxicity
- (2008) S. N. Hobbie et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Drug-induced readthrough of premature stop codons leads to the stabilization of laminin α2 chain mRNA in CMD myotubes
- (2007) Valérie Allamand et al. JOURNAL OF GENE MEDICINE
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