Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense Drug Development

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Accurate quantification of dystrophin mRNA and exon skipping levels in Duchenne Muscular Dystrophy

(2010) Pietro Spitali et al.   LABORATORY INVESTIGATION

Guanine Analogues Enhance Antisense Oligonucleotide-induced Exon Skipping in Dystrophin Gene In Vitro and In Vivo

(2010) Yihong Hu et al.   MOLECULAR THERAPY

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

Exonic Sequences Provide Better Targets for Antisense Oligonucleotides Than Splice Site Sequences in the Modulation of Duchenne Muscular Dystrophy Splicing

(2010) Annemieke Aartsma-Rus et al.   OLIGONUCLEOTIDES

Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy

(2010) Annemieke Aartsma-Rus   RNA Biology

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy

(2009) Hans Heemskerk et al.   Annals of the New York Academy of Sciences

Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

(2009) B Wu et al.   GENE THERAPY

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

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice

(2009) Bo Wu et al.   MOLECULAR THERAPY

Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms

(2008) Annemieke Aartsma-Rus et al.   MOLECULAR THERAPY

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

(2008) B. Wu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA