Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies

The emperor's new dystrophin: finding sense in the noise

(2015) S.D. Wilton et al.   TRENDS IN MOLECULAR MEDICINE

Pseudoexon activation increases phenotype severity in a Becker muscular dystrophy patient

(2015) Kane Greer et al.   Molecular Genetics & Genomic Medicine

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

(2014) Nicolas Wein et al.   NATURE MEDICINE

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

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

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene

(2012) Sue Fletcher et al.   Molecular Therapy-Nucleic Acids

Clinical and molecular characterization of a cohort of patients with novel nucleotide alterations of the Dystrophin gene detected by direct sequencing

(2011) Francesca Magri et al.   BMC Medical Genetics

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching

(2011) Clayton T Fragall et al.   BMC Medical Genetics

Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene

(2010) Kevin M. Flanigan et al.   HUMAN MUTATION

Mutation spectrum of the dystrophin gene in 442 Duchenne/Becker muscular dystrophy cases from one Japanese referral center

(2010) Yasuhiro Takeshima et al.   JOURNAL OF HUMAN GENETICS

DMDexon 1 truncating point mutations: Amelioration of phenotype by alternative translation initiation in exon 6

(2009) Olga L. Gurvich et al.   HUMAN MUTATION

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

Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY