A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies

(2021) Omar Sheikh et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides

(2021) Yuichiro Tone et al.   Nucleic Acid Therapeutics

Duchenne muscular dystrophy

(2021) Dongsheng Duan et al.   Nature Reviews Disease Primers

Casimersen: First Approval

(2021) Matt Shirley   DRUGS

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

(2020) A. Moretti et al.   NATURE MEDICINE

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

(2020) Diane E. Frank et al.   NEUROLOGY

CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy

(2020) Kenji Rowel Q. Lim et al.   Genes

Chaetocin Improves Pig Cloning Efficiency by Enhancing Epigenetic Reprogramming and Autophagic Activity

(2020) Pil-Soo Jeong et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Application of Genetically Engineered Pigs in Biomedical Research

(2020) Magdalena Hryhorowicz et al.   Genes

A novel mouse model of Duchenne muscular dystrophy carrying a multi-exonic Dmd deletion exhibits progressive muscular dystrophy and early-onset cardiomyopathy

(2020) Tatianna Wai Ying Wong et al.   Disease Models & Mechanisms

Pathological evaluation of rats carrying in-frame mutations in the dystrophin gene: a new model of Becker muscular dystrophy

(2020) Naomi Teramoto et al.   Disease Models & Mechanisms

Gene therapies in canine models for Duchenne muscular dystrophy

(2019) Peter P. Nghiem et al.   HUMAN GENETICS

Therapeutic developments for Duchenne muscular dystrophy

(2019) Ingrid E. C. Verhaart et al.   Nature Reviews Neurology

Production of non-mosaic genome edited porcine embryos by injection of CRISPR/Cas9 into germinal vesicle oocytes

(2019) Xiaohu Su et al.   Journal of Genetics and Genomics

Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

(2019) Lindsay N. Alfano et al.   MEDICINE

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

(2019) Yusuke Echigoya et al.   MOLECULAR THERAPY

Efficient base editing for multiple genes and loci in pigs using base editors

(2019) Jingke Xie et al.   Nature Communications

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

(2019) Maria K. Tsoumpra et al.   EBioMedicine

Cardiac Pathophysiology and the Future of Cardiac Therapies in Duchenne Muscular Dystrophy

(2019) Tatyana A. Meyers et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Tracking progress: an update on animal models for Duchenne muscular dystrophy

(2018) Dominic J. Wells   Disease Models & Mechanisms

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

(2018) Nathalie Goemans et al.   NEUROMUSCULAR DISORDERS

Modeling lethal X-linked genetic disorders in pigs with ensured fertility

(2018) Hitomi Matsunari et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

(2018) Hirofumi Komaki et al.   Science Translational Medicine

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

(2018) Michael J. Gait et al.   Nucleic Acid Therapeutics

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

(2017) Yusuke Echigoya et al.   MOLECULAR THERAPY

Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

(2017) Yusuke Echigoya et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Eteplirsen in the treatment of Duchenne muscular dystrophy

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

Predictors of Death in Adults With Duchenne Muscular Dystrophy–Associated Cardiomyopathy

(2017) Daniel Cheeran et al.   Journal of the American Heart Association

Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies

(2016) Narinder Janghra et al.   PLoS One

Derivation of Porcine Embryonic Stem-Like Cells from In Vitro-Produced Blastocyst-Stage Embryos

(2016) Dao-Rong Hou et al.   Scientific Reports

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

(2015) J. W. McGreevy et al.   Disease Models & Mechanisms

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

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

Barriers for Deriving Transgene-Free Pig iPS Cells with Episomal Vectors

(2015) Xuguang Du et al.   STEM CELLS

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

(2015) Yusuke Echigoya et al.   Molecular Therapy-Nucleic Acids

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

Development and translational imaging of a TP53 porcine tumorigenesis model

(2014) Jessica C. Sieren et al.   JOURNAL OF CLINICAL INVESTIGATION

Genetically engineered SCN5A mutant pig hearts exhibit conduction defects and arrhythmias

(2014) David S. Park et al.   JOURNAL OF CLINICAL INVESTIGATION

A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy

(2014) Jean K. Mah et al.   NEUROMUSCULAR DISORDERS

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

(2013) Nikolai Klymiuk et al.   HUMAN MOLECULAR GENETICS

Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic Mdx and Mdx52 Mice

(2013) Yusuke Echigoya et al.   PLoS One

Initial Pulmonary Respiration Causes Massive Diaphragm Damage and Hyper-CKemia in Duchenne Muscular Dystrophy Dog

(2013) Akinori Nakamura et al.   Scientific Reports

Evidence-based path to newborn screening for duchenne muscular dystrophy

(2012) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Age-matched comparison reveals early electrocardiography and echocardiography changes in dystrophin-deficient dogs

(2011) Deborah M. Fine et al.   NEUROMUSCULAR DISORDERS

Effect of epigenetic regulation during swine embryogenesis and on cloning by nuclear transfer

(2010) Jianguo Zhao et al.   CELL AND TISSUE RESEARCH

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

Current status and applications of somatic cell nuclear transfer in dogs

(2010) Goo Jang et al.   THERIOGENOLOGY

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Ringo: Discordance between the molecular and clinical manifestation in a Golden Retriever Muscular Dystrophy dog

(2009) Eder Zucconi et al.   NEUROMUSCULAR DISORDERS

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Production of CFTR-null and CFTR-ΔF508 heterozygous pigs by adeno-associated virus–mediated gene targeting and somatic cell nuclear transfer

(2008) Christopher S. Rogers et al.   JOURNAL OF CLINICAL INVESTIGATION

Disruption of the CFTR Gene Produces a Model of Cystic Fibrosis in Newborn Pigs

(2008) C. S. Rogers et al.   SCIENCE