Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy

Synthesis and evaluation of compounds that induce readthrough of premature termination codons

(2011) Michael E. Jung et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

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

Development and validation of an HPLC method for quantitation of BA-TPQ, a novel iminoquinone anticancer agent, and an initial pharmacokinetic study in mice

(2010) Haibo Li et al.   BIOMEDICAL CHROMATOGRAPHY

Illuminating Insights into Firefly Luciferase and Other Bioluminescent Reporters Used in Chemical Biology

(2010) Natasha Thorne et al.   CHEMISTRY & BIOLOGY

Site-directed gene repair of the dystrophin gene mediated by PNA–ssODNs

(2010) Refik Kayali et al.   HUMAN MOLECULAR GENETICS

A 3 months mild functional test regime does not affect disease parameters in young mdx mice

(2010) Maaike van Putten et al.   NEUROMUSCULAR DISORDERS

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

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

Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-β

(2009) Sylvia A. Vetrone et al.   JOURNAL OF CLINICAL INVESTIGATION

Nonaminoglycoside compounds induce readthrough of nonsense mutations

(2009) Liutao Du et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Preclinical drug trials in themdxmouse: Assessment of reliable and sensitive outcome measures

(2009) Christopher F. Spurney et al.   MUSCLE & NERVE

Nonsense suppression activity of PTC124 (ataluren)

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

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

Laminin-111 protein therapy prevents muscle disease in the mdx mouse model for Duchenne muscular dystrophy

(2009) J. E. Rooney et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A simple and reliable method for determining plasma concentration of dehydroxymethylepoxyquinomicin by high performance liquid chromatography with mass spectrometry

(2008) Etsuko Watanabe et al.   JOURNAL OF CHROMATOGRAPHY B-ANALYTICAL TECHNOLOGIES IN THE BIOMEDICAL AND LIFE SCIENCES

Effective Exon Skipping and Restoration of Dystrophin Expression by Peptide Nucleic Acid Antisense Oligonucleotides in mdx Mice

(2007) Haifang Yin et al.   MOLECULAR THERAPY