Steve D. Wilton

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 · 852 citations · Full text

Exon Skipping, Urology, Duchenne Muscular Dystrophy +4

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Maria Kinali, Virginia Arechavala‐Gomeza, Lucy Feng et al. · The Lancet Neurology · 2009 · 635 citations · Full text

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

J. Alter, Fang Lou, Adam Rabinowitz et al. · Nature Medicine · 2006 · 491 citations

Regenerative Medicine, Morpholino Oligonucleotide, Pathology +5

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

Qi Long Lu, Christopher J. Mann, Fang Lou et al. · Nature Medicine · 2003 · 388 citations

Knockout Mouse, Developmental Biology, Mdx Dystrophic Mouse +4

Antisense-induced exon skipping and synthesis of dystrophin in the <i>mdx</i> mouse

Christopher J. Mann, Kaite Honeyman, Andy Cheng et al. · Proceedings of the National Academy of Sciences · 2000 · 380 citations