Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

Eric P. Hoffman, Corlee J. Bertelson, Anthony P. Monaco et al. · Cell · 1987 · 2.4K citations

Life Sciences, Genetics, Molecular Genetics +11

The Molecular Basis of Muscular Dystrophy in the <i>mdx</i> Mouse: a Point Mutation

Piotr Siciński, Yan Geng, Allan S. Ryder-Cook et al. · Science · 1989 · 1.2K citations

Medicine, Genetics, Molecular Genetics +13

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Sebahattin Çirak, Virginia Arechavala‐Gomeza, Michela Guglieri et al. · The Lancet · 2011 · 862 citations · Full text

Medicine, Neurology, Pharmacotherapy +13

Eteplirsen for the treatment of Duchenne muscular dystrophy

Jerry R. Mendell, Louise R. Rodino‐Klapac, Zarife Sahenk et al. · Annals of Neurology · 2013 · 795 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +14

Utrophin-Dystrophin-Deficient Mice as a Model for Duchenne Muscular Dystrophy

Anne E. Deconinck, Jill A. Rafael, Judith A. Skinner et al. · Cell · 1997 · 730 citations · Full text

Medicine, Genetics, Molecular Genetics +9