Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

Casimersen: First Approval

(2021) Matt Shirley   DRUGS

Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis

(2020) Salvatore Crisafulli et al.   Orphanet Journal of Rare Diseases

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

(2020) Philippine Aupy et al.   Molecular Therapy-Methods & Clinical Development

Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model

(2020) Alper Yavas et al.   PLoS One

Genotype–Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease Registry

(2020) Kenji Rowel Q. Lim et al.   Journal of Personalized Medicine

Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the DMD gene

(2019) Yusuke Echigoya et al.   MOLECULAR THERAPY

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

(2019) Maria K. Tsoumpra et al.   EBioMedicine

A dystrophic Duchenne mouse model for testing human antisense oligonucleotides

(2018) Marcel Veltrop et al.   PLoS One

Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates

(2018) Kenji Rowel Q. Lim et al.   MOLECULAR THERAPY

Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy

(2017) Yusuke Echigoya et al.   MOLECULAR THERAPY

Eteplirsen in the treatment of Duchenne muscular dystrophy

(2017) Kenji Rowel Lim et al.   Drug Design Development and Therapy

Peptides for nucleic acid delivery

(2016) Taavi Lehto et al.   ADVANCED DRUG DELIVERY REVIEWS

Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy

(2016) Akinori Nakamura et al.   JOURNAL OF HUMAN GENETICS

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

In vitro stability of therapeutically relevant, internally truncated dystrophins

(2015) Jackie L McCourt et al.   Skeletal Muscle

In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy

(2015) Yusuke Echigoya et al.   PLoS One

DMD transcript imbalance determines dystrophin levels

(2013) Pietro Spitali et al.   FASEB JOURNAL

Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells

(2013) T. Lehto et al.   NUCLEIC ACIDS RESEARCH

Biochemical Characterization of Patients With In-Frame or Out-of-FrameDMDDeletions Pertinent to Exon 44 or 45 Skipping

(2013) Karen Anthony et al.   JAMA Neurology

The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology

(2012) Maaike van Putten et al.   PLoS One

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

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

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

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

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Update on the management of Duchenne muscular dystrophy

(2008) A Y Manzur et al.   ARCHIVES OF DISEASE IN CHILDHOOD