Deletion of exons 3−9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy

Dystrophin and Duchenne dystrophy

(2015) Victor Dubowitz et al.   NEUROMUSCULAR DISORDERS

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

(2015) Yusuke Echigoya et al.   Molecular Therapy-Nucleic Acids

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

(2014) Kane L Greer et al.   Molecular Therapy-Nucleic Acids

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

(2012) Y. Aoki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

(2011) K. Anthony et al.   BRAIN

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

A Renaissance for Antisense Oligonucleotide Drugs in Neurology

(2009) Toshifumi Yokota et al.   ARCHIVES OF NEUROLOGY

Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase

(2009) Sylvie Tuffery-Giraud et al.   HUMAN MUTATION

Exon-skipping therapy for Duchenne muscular dystrophy

(2009) Akinori Nakamura et al.   NEUROPATHOLOGY

Follow-up of three patients with a large in-frame deletion of exons 45–55 in the Duchenne muscular dystrophy (DMD) gene

(2008) Akinori Nakamura et al.   JOURNAL OF CLINICAL NEUROSCIENCE