Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

(2012) Y. Aoki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle

(2011) Eric P. Hoffman et al.   AMERICAN JOURNAL OF PATHOLOGY

Scavenger receptor-mediated uptake of cell-penetrating peptide nanocomplexes with oligonucleotides

(2011) Kariem Ezzat et al.   FASEB JOURNAL

Exon-skipping therapy for Duchenne muscular dystrophy

(2011) Akinori Nakamura et al.   LANCET

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

(2011) Sebahattin Cirak et al.   LANCET

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Skeletal muscle laminin and MDC1A: pathogenesis and treatment strategies

(2011) Kinga I Gawlik et al.   Skeletal Muscle

Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

(2010) Hong M. Moulton et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

RNA-targeted splice-correction therapy for neuromuscular disease

(2010) M. J. A. Wood et al.   BRAIN

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

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

(2010) Yoshitsugu Aoki et al.   MOLECULAR THERAPY

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

(2010) HaiFang Yin et al.   MOLECULAR THERAPY

Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-Deleted DMD Patient

(2010) Takashi Saito et al.   PLoS One

Our trails and trials in the subsarcolemmal cytoskeleton network and muscular dystrophy researches in the dystrophin era

(2010) Eijiro OZAWA   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

Toward an Oligonucleotide Therapy for Duchenne Muscular Dystrophy: A Complex Development Challenge

(2010) M. J. A. Wood   Science Translational Medicine

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

A Renaissance for Antisense Oligonucleotide Drugs in Neurology

(2009) Toshifumi Yokota et al.   ARCHIVES OF NEUROLOGY

Cellular Uptake of Neutral Phosphorodiamidate Morpholino Oligomers

(2009) Patrick Iversen et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY