The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

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

Gentamicin B1 is a minor gentamicin component with major nonsense mutation suppression activity

(2017) Alireza Baradaran-Heravi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Aminoglycoside interactions and impacts on the eukaryotic ribosome

(2017) Irina Prokhorova et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Functional rescue of REP1 following treatment with PTC124 and novel derivative PTC-414 in human choroideremia fibroblasts and the nonsense-mediated zebrafish model

(2016) Mariya Moosajee et al.   HUMAN MOLECULAR GENETICS

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

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

(2015) Manuel Haas et al.   NEUROMUSCULAR DISORDERS

The novel Cln1R151X mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy

(2014) Jake N. Miller et al.   HUMAN MOLECULAR GENETICS

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

Structural basis for the inhibition of the eukaryotic ribosome

(2014) Nicolas Garreau de Loubresse et al.   NATURE

When proteins start to make sense: fine-tuning of aminoglycosides for PTC suppression therapy

(2014) Moran Shalev et al.   MedChemComm

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

Postnatal manipulation of Pax6 dosage reverses congenital tissue malformation defects

(2013) Cheryl Y. Gregory-Evans et al.   JOURNAL OF CLINICAL INVESTIGATION

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

(2013) Richard S. Finkel et al.   PLoS One

Ataluren as an Agent for Therapeutic Nonsense Suppression

(2012) Stuart W. Peltz et al.   Annual Review of Medicine

Increased Selectivity toward Cytoplasmic versus Mitochondrial Ribosome Confers Improved Efficiency of Synthetic Aminoglycosides in Fixing Damaged Genes: A Strategy for Treatment of Genetic Diseases Caused by Nonsense Mutations

(2012) Jeyakumar Kandasamy et al.   JOURNAL OF MEDICINAL CHEMISTRY

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

(2011) M. Wilschanski et al.   EUROPEAN RESPIRATORY JOURNAL

Test Characteristics of Urinary Biomarkers Depend on Quantitation Method in Acute Kidney Injury

(2011) A. Md Ralib et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Determination of Gentamicin Sulphate Composition and Related Substances in Pharmaceutical Preparations by LC with Charged Aerosol Detection

(2010) Karolina Stypulkowska et al.   CHROMATOGRAPHIA

Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy

(2010) Vinod Malik et al.   Therapeutic Advances in Neurological Disorders

Development of K562 cell clones expressing β-globin mRNA carrying the β039 thalassaemia mutation for the screening of correctors of stop-codon mutations

(2009) Francesca Salvatori et al.   BIOTECHNOLOGY AND APPLIED BIOCHEMISTRY

A meta-analysis of nonsense mutations causing human genetic disease

(2008) Matthew Mort et al.   HUMAN MUTATION

Using the Zebrafish Lateral Line to Screen for Ototoxicity

(2008) Lynn L. Chiu et al.   JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY

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

Aminoglycoside induced ototoxicity

(2008) O’neil W. Guthrie   TOXICOLOGY

Introducing sense into nonsense in treatments of human genetic diseases

(2008) Liat Linde et al.   TRENDS IN GENETICS

Designer aminoglycosides: the race to develop improved antibiotics and compounds for the treatment of human genetic diseases

(2007) Mariana Hainrichson et al.   ORGANIC & BIOMOLECULAR CHEMISTRY