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

Long-term nonsense suppression therapy with NB84 moderates MPS IH disease progression

(2014) Gwendolyn G. Gunn et al.   MOLECULAR GENETICS AND METABOLISM

4′-O-substitutions determine selectivity of aminoglycoside antibiotics

(2014) Déborah Perez-Fernandez et al.   Nature Communications

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

The mitochondrion: A perpetrator of acquired hearing loss

(2013) Erik C. Böttger et al.   HEARING RESEARCH

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

A Novel Role of Cytosolic Protein Synthesis Inhibition in Aminoglycoside Ototoxicity

(2013) S. P. Francis et al.   JOURNAL OF NEUROSCIENCE

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

The initiation of mammalian protein synthesis and mRNA scanning mechanism

(2013) Ivan B. Lomakin et al.   NATURE

Identification of the molecular attributes required for aminoglycoside activity against Leishmania

(2013) M. Shalev et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

A new understanding of the decoding principle on the ribosome

(2012) Natalia Demeshkina et al.   NATURE

The GABRG2 nonsense mutation, Q40X, associated with Dravet syndrome activated NMD and generated a truncated subunit that was partially rescued by aminoglycoside-induced stop codon read-through

(2012) Xuan Huang et al.   NEUROBIOLOGY OF DISEASE

Rescue of nonsense mutations by amlexanox in human cells

(2012) Sara Gonzalez-Hilarion et al.   Orphanet Journal of Rare Diseases

Dissociation of antibacterial activity and aminoglycoside ototoxicity in the 4-monosubstituted 2-deoxystreptamine apramycin

(2012) T. Matt et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

N-Acetylcysteine Use for Amelioration of Aminoglycoside-Induced Ototoxicity in Dialysis Patients

(2012) Leonid Feldman et al.   SEMINARS IN DIALYSIS

Sense from nonsense: therapies for premature stop codon diseases

(2012) Laure Bidou et al.   TRENDS IN MOLECULAR MEDICINE

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

A Homo sapiens cytoplasmic ribosomal decoding A-site affinity screen evaluating aminoglycoside and analogue binding

(2011) Georgia Kythreoti et al.   ANALYTICAL BIOCHEMISTRY

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

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

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

Readthrough of Premature Termination Codons in the Adenomatous Polyposis Coli Gene Restores Its Biological Activity in Human Cancer Cells

(2011) Célia Floquet et al.   PLoS One

Crystal Structure of the Eukaryotic 60S Ribosomal Subunit in Complex with Initiation Factor 6

(2011) S. Klinge et al.   SCIENCE

The Structure of the Eukaryotic Ribosome at 3.0 A Resolution

(2011) A. Ben-Shem et al.   SCIENCE

Repairing faulty genes by aminoglycosides: Identification of new pharmacophore with enhanced suppression of disease-causing nonsense mutations

(2011) Jeyakumar Kandasamy et al.   MedChemComm

Suppression of nonsense mutations as a therapeutic approach to treat genetic diseases

(2011) Kim M. Keeling et al.   Wiley Interdisciplinary Reviews-RNA

Cover Picture: Light-Driven Transport of a Molecular Walker in Either Direction along a Molecular Track (Angew. Chem. Int. Ed. 1/2011)

(2010) Michael J. Barrell et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

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

The Future of Aminoglycosides: The End or Renaissance?

(2010) Jacob L. Houghton  et al.   CHEMBIOCHEM

Recognition of the amber UAG stop codon by release factor RF1

(2010) Andrei Korostelev et al.   EMBO JOURNAL

Dual-acting hybrid antibiotics: a promising strategy to combat bacterial resistance

(2010) Varvara Pokrovskaya et al.   Expert Opinion on Drug Discovery

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

Rescue of non-sense mutated p53 tumor suppressor gene by aminoglycosides

(2010) Célia Floquet et al.   NUCLEIC ACIDS RESEARCH

Structure of the 70S ribosome bound to release factor 2 and a substrate analog provides insights into catalysis of peptide release

(2010) H. Jin et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124

(2010) D. S. Auld et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Crystal Structure of the Eukaryotic Ribosome

(2010) A. Ben-Shem et al.   SCIENCE

Crystal Structure of the Eukaryotic 40S Ribosomal Subunit in Complex with Initiation Factor 1

(2010) J. Rabl et al.   SCIENCE

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

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

(2009) Virginia B Mattis et al.   BMC NEUROSCIENCE

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

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

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

Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression

(2009) D. S. Auld et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Structural Insights into Ribosome Recycling Factor Interactions with the 70S Ribosome

(2008) Raj D. Pai et al.   JOURNAL OF MOLECULAR BIOLOGY

Structural basis for translation termination on the 70S ribosome

(2008) Martin Laurberg et al.   NATURE

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

Insights into Translational Termination from the Structure of RF2 Bound to the Ribosome

(2008) A. Weixlbaumer et al.   SCIENCE