Gene Therapy · 2006 · 203 citations · 20 references
MedicineCell BiologyMolecular MedicineGene TherapyMuscular DystrophyNeuromuscular JunctionAntisense Oligonucleotide DesignAntisense Oligonucleotide SilencingDystrophin DeficiencyExon SkippingExon Skipping TherapyMuscular Dystrophy GeneticsMuscular Dystrophy Pathophysiology
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Dystrophin: The protein product of the duchenne muscular dystrophy locus
Eric P. Hoffman, Robert H. Brown, Louis M. Kunkel · Cell · 1987 · 4.7K citations
ESEfinder: a web resource to identify exonic splicing enhancers
Luca Cartegni · Nucleic Acids Research · 2003 · 1.6K citations · Full text
The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs
Barry J. Cooper, Nena J. Winand, Hansell H. Stedman et al. · Nature · 1988 · 429 citations