Dystrophin Cardiomyopathies: Clinical Management, Molecular Pathogenesis and Evolution towards Precision Medicine

Mechanotransduction of human pluripotent stem cells cultivated on tunable cell-derived extracellular matrix

(2018) In Gul Kim et al.   BIOMATERIALS

Effect of geometrical constraints on human pluripotent stem cell nuclei in pluripotency and differentiation

(2018) Eleonora Grespan et al.   Integrative Biology

At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy—Are We Closer to Effective Treatment for Patients?

(2018) Kinga Gawlik   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Detection and management of cardiomyopathy in female dystrophinopathy carriers

(2018) Katsuhito Adachi et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis

(2018) Yuko Shimizu-Motohashi et al.   Orphanet Journal of Rare Diseases

Investigation of human iPSC-derived cardiac myocyte functional maturation by single cell traction force microscopy

(2018) Matthew Wheelwright et al.   PLoS One

Contractile force measurement of human induced pluripotent stem cell-derived cardiac cell sheet-tissue

(2018) Daisuke Sasaki et al.   PLoS One

Generation of induced pluripotent stem cells from a Becker muscular dystrophy patient carrying a deletion of exons 45-55 of the dystrophin gene ( CCMi002BMD-A-9 ∆45-55 )

(2018) Aoife Gowran et al.   Stem Cell Research

Generation of Duchenne muscular dystrophy patient-specific induced pluripotent stem cell line lacking exons 45–50 of the dystrophin gene (IITi001-A)

(2018) Binyamin Eisen et al.   Stem Cell Research

Engineering human pluripotent stem cells into a functional skeletal muscle tissue

(2018) Lingjun Rao et al.   Nature Communications

Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy

(2018) Mark A. Aminzadeh et al.   Stem Cell Reports

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

(2018) Chengzu Long et al.   Science Advances

Activation of human aortic valve interstitial cells by local stiffness involves YAP-dependent transcriptional signaling

(2018) Rosaria Santoro et al.   BIOMATERIALS

A current approach to heart failure in Duchenne muscular dystrophy

(2017) Domenico D’Amario et al.   HEART

Left ventricular assist device as destination therapy in cardiac end-stage dystrophinopathies: Midterm results

(2017) Gianluigi Perri et al.   JOURNAL OF THORACIC AND CARDIOVASCULAR SURGERY

A cardiac patch from aligned microvessel and cardiomyocyte patches

(2017) Jeremy A. Schaefer et al.   Journal of Tissue Engineering and Regenerative Medicine

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

(2017) Craig M McDonald et al.   LANCET

Derivation of the Duchenne muscular dystrophy patient-derived induced pluripotent stem cell line lacking DMD exons 49 and 50 (CCMi001DMD-A-3, ∆ 49, ∆ 50)

(2017) Gabriella Spaltro et al.   Stem Cell Research

Epigenetic Erasing and Pancreatic Differentiation of Dermal Fibroblasts into Insulin-Producing Cells are Boosted by the Use of Low-Stiffness Substrate

(2017) Georgia Pennarossa et al.   Stem Cell Reviews and Reports

Concise Review: Induced Pluripotent Stem Cell Research in the Era of Precision Medicine

(2017) Takashi Hamazaki et al.   STEM CELLS

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

(2017) Caroline Le Guiner et al.   Nature Communications

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Epigenetic Erasing and Pancreatic Differentiation of Dermal Fibroblasts into Insulin-Producing Cells are Boosted by the Use of Low-Stiffness Substrate

(2017) Georgia Pennarossa et al.   Stem Cell Reviews and Reports

Dystrophin Dp116: A yet to Be Investigated Product of the Duchenne Muscular Dystrophy Gene

(2017) Masafumi Matsuo et al.   Genes

MUSCLEMOTION

(2017) Luca Sala et al.   CIRCULATION RESEARCH

Timing and localization of human dystrophin isoform expression provide insights into the cognitive phenotype of Duchenne muscular dystrophy

(2017) Nathalie Doorenweerd et al.   Scientific Reports

The nuclear pore protein Nup153 associates with chromatin and regulates cardiac gene expression in dystrophicmdxhearts

(2016) Simona Nanni et al.   CARDIOVASCULAR RESEARCH

Mechanical Forces Reshape Differentiation Cues That Guide Cardiomyogenesis

(2016) Cassandra L. Happe et al.   CIRCULATION RESEARCH

Exon skipping: a first in class strategy for Duchenne muscular dystrophy

(2016) Erik H. Niks et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Generation of Induced Pluripotent Stem Cells From Patients With Duchenne Muscular Dystrophy and Their Induction to Cardiomyocytes

(2016) Akihito Hashimoto et al.   International Heart Journal

Approving a Problematic Muscular Dystrophy Drug

(2016) Aaron S. Kesselheim et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

The importance of genetic diagnosis for Duchenne muscular dystrophy

(2016) Annemieke Aartsma-Rus et al.   JOURNAL OF MEDICAL GENETICS

Beating heart on a chip: a novel microfluidic platform to generate functional 3D cardiac microtissues

(2016) Anna Marsano et al.   LAB ON A CHIP

Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy

(2016) David Gloss et al.   NEUROLOGY

Mechanical Circulatory Support: Current Status and Future Directions

(2016) Jorge Silva Enciso   PROGRESS IN CARDIOVASCULAR DISEASES

Young at Heart: Pioneering Approaches to Model Nonischaemic Cardiomyopathy with Induced Pluripotent Stem Cells

(2016) Aoife Gowran et al.   Stem Cells International

Reactivation of Endogenous Genes and Epigenetic Remodeling Are Barriers for Generating Transgene-Free Induced Pluripotent Stem Cells in Pig

(2016) Kwang-Hwan Choi et al.   PLoS One

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

(2016) Valeria Ricotti et al.   PLoS One

Nanopatterned Human iPSC-Based Model of a Dystrophin-Null Cardiomyopathic Phenotype

(2015) Jesse Macadangdang et al.   Cellular and Molecular Bioengineering

Contemporary Cardiac Issues in Duchenne Muscular Dystrophy: Table.

(2015) Elizabeth M. McNally et al.   CIRCULATION

Modeling and study of the mechanism of dilated cardiomyopathy using induced pluripotent stem cells derived from individuals with Duchenne muscular dystrophy

(2015) B. Lin et al.   Disease Models & Mechanisms

Ocular and neurodevelopmental features of Duchenne muscular dystrophy: a signature of dystrophin function in the central nervous system

(2015) Valeria Ricotti et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus

(2015) Yongping Yue et al.   HUMAN MOLECULAR GENETICS

Hemodynamics of Mechanical Circulatory Support

(2015) Daniel Burkhoff et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Current Understanding of Molecular Pathology and Treatment of Cardiomyopathy in Duchenne Muscular Dystrophy

(2015) Tirsa van Westering et al.   MOLECULES

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

(2015) Aurélie Goyenvalle et al.   NATURE MEDICINE

Implantation of a left ventricular assist device as a destination therapy in Duchenne muscular dystrophy patients with end stage cardiac failure: Management and lessons learned

(2015) Francesca Iodice et al.   NEUROMUSCULAR DISORDERS

Substrate Stiffness and Composition Specifically Direct Differentiation of Induced Pluripotent Stem Cells

(2015) Laura Macrí-Pellizzeri et al.   TISSUE ENGINEERING PART A

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

(2015) Corinne A. Betts et al.   Scientific Reports

Gene therapy for Duchenne muscular dystrophy

(2015) Julian Ramos et al.   Expert Opinion on Orphan Drugs

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

(2015) Hongmei Lisa Li et al.   Stem Cell Reports

Controlled electromechanical cell stimulation on-a-chip

(2015) Andrea Pavesi et al.   Scientific Reports

Reduced cerebral gray matter and altered white matter in boys with Duchenne muscular dystrophy

(2014) Nathalie Doorenweerd et al.   ANNALS OF NEUROLOGY

Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy

(2014) Doris G. Leung et al.   ANNALS OF NEUROLOGY

Implantation of the HeartMate II and HeartWare Left Ventricular Assist Devices in Patients With Duchenne Muscular Dystrophy

(2014) Thomas D. Ryan et al.   ASAIO JOURNAL

Long-term treatment with naproxcinod significantly improves skeletal and cardiac disease phenotype in the mdx mouse model of dystrophy

(2014) K. Uaesoontrachoon et al.   HUMAN MOLECULAR GENETICS

Maintenance of undifferentiated state of human induced pluripotent stem cells through cytoskeleton-driven force acting to secreted fibronectin on a dendrimer-immobilized surface

(2014) Mee-Hae Kim et al.   JOURNAL OF BIOSCIENCE AND BIOENGINEERING

Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystrophy

(2014) Christopher Spurney et al.   MUSCLE & NERVE

Treatment of dystrophin cardiomyopathies

(2014) Josef Finsterer et al.   Nature Reviews Cardiology

Substratum-induced differentiation of human pluripotent stem cells reveals the coactivator YAP is a potent regulator of neuronal specification

(2014) S. Musah et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Complete restoration of multiple dystrophin isoforms in genetically corrected Duchenne muscular dystrophy patient–derived cardiomyocytes

(2014) Susi Zatti et al.   Molecular Therapy-Methods & Clinical Development

Dystrophin complex functions as a scaffold for signalling proteins

(2013) Bruno Constantin   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Fifth INTERMACS annual report: Risk factor analysis from more than 6,000 mechanical circulatory support patients

(2013) James K. Kirklin et al.   JOURNAL OF HEART AND LUNG TRANSPLANTATION

Oral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy

(2013) Brent J. Barber et al.   JOURNAL OF PEDIATRICS

Patient Selection for Ventricular Assist Devices

(2013) Leslie W. Miller et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Splicing therapy for neuromuscular disease

(2013) Andrew G.L. Douglas et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

Biowire: a platform for maturation of human pluripotent stem cell–derived cardiomyocytes

(2013) Sara S Nunes et al.   NATURE METHODS

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

Rigid microenvironments promote cardiac differentiation of mouse and human embryonic stem cells

(2013) Armin Arshi et al.   SCIENCE AND TECHNOLOGY OF ADVANCED MATERIALS

Dystrophin-deficient cardiomyocytes derived from human urine: New biologic reagents for drug discovery

(2013) Xuan Guan et al.   Stem Cell Research

Exon Skipping and Gene Transfer Restore Dystrophin Expression in Human Induced Pluripotent Stem Cells-Cardiomyocytes Harboring DMD Mutations

(2013) Emily Dick et al.   STEM CELLS AND DEVELOPMENT

Fabrication of 3D cell-laden hydrogel microstructures through photo-mold patterning

(2013) P Occhetta et al.   Biofabrication

Peptide Conjugation of 2′-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice

(2013) Silvana M.G. Jirka et al.   Nucleic Acid Therapeutics

Effects of Angiotensin-Converting Enzyme Inhibitors and/or Beta Blockers on the Cardiomyopathy in Duchenne Muscular Dystrophy

(2012) Laurence Viollet et al.   AMERICAN JOURNAL OF CARDIOLOGY

Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice

(2012) Radbod Darabi et al.   Cell Stem Cell

Interference with Myostatin/ActRIIB Signaling as a Therapeutic Strategy for Duchenne Muscular Dystrophy

(2012) Helge Amthor et al.   CURRENT GENE THERAPY

Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy

(2012) Helen Foster et al.   HUMAN GENE THERAPY

Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer in mdx Mice

(2012) Stefanie Schinkel et al.   HUMAN GENE THERAPY

Control of stem cell fate and function by engineering physical microenvironments

(2012) Kshitiz et al.   Integrative Biology

Left ventricular assist device in Duchenne Cardiomyopathy: Can we change the natural history of cardiac disease?

(2012) Antonio Amodeo et al.   INTERNATIONAL JOURNAL OF CARDIOLOGY

AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy

(2012) Brian Bostick et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Cardiac phenotype of Duchenne Muscular Dystrophy: Insights from cellular studies

(2012) Natalia Shirokova et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Sildenafil reduces respiratory muscle weakness and fibrosis in the mdx mouse model of Duchenne muscular dystrophy

(2012) Justin M Percival et al.   JOURNAL OF PATHOLOGY

A single ascending-dose study of muscle regulator ace-031 in healthy volunteers

(2012) Kenneth M. Attie et al.   MUSCLE & NERVE

Gene and cell-mediated therapies for muscular dystrophy

(2012) Patryk Konieczny et al.   MUSCLE & NERVE

Directed cardiomyocyte differentiation from human pluripotent stem cells by modulating Wnt/β-catenin signaling under fully defined conditions

(2012) Xiaojun Lian et al.   Nature Protocols

Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure

(2012) Meilan M. Rutter et al.   NEUROMUSCULAR DISORDERS

The medical genetics of dystrophinopathies: Molecular genetic diagnosis and its impact on clinical practice

(2012) Alessandra Ferlini et al.   NEUROMUSCULAR DISORDERS

Gene therapy for muscular dystrophy: Lessons learned and path forward

(2012) Jerry R. Mendell et al.   NEUROSCIENCE LETTERS

Effects of glucocorticoids and idebenone on respiratory function in patients with duchenne muscular dystrophy

(2012) Gunnar M. Buyse et al.   PEDIATRIC PULMONOLOGY

Micro-Arrayed Human Embryonic Stem Cells-Derived Cardiomyocytes for In Vitro Functional Assay

(2012) Elena Serena et al.   PLoS One

Recombinant MG53 Protein Modulates Therapeutic Cell Membrane Repair in Treatment of Muscular Dystrophy

(2012) N. Weisleder et al.   Science Translational Medicine

The Muscular Dystrophies

(2012) Kevin Flanigan   SEMINARS IN NEUROLOGY

Ventricular Assist Devices as a Bridge to Heart Transplantation or as Destination Therapy in Pediatric Patients

(2012) G. Brancaccio et al.   TRANSPLANTATION PROCEEDINGS

Pathophysiology and Therapy of Cardiac Dysfunction in Duchenne Muscular Dystrophy

(2011) Daniel P. Judge et al.   American Journal of Cardiovascular Drugs

Urocortins Improve Dystrophic Skeletal Muscle Structure and Function through Both PKA- and Epac-Dependent Pathways

(2011) Julie Reutenauer-Patte et al.   AMERICAN JOURNAL OF PATHOLOGY

Human myostatin negatively regulates human myoblast growth and differentiation

(2011) Craig McFarlane et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Early Treatment With Lisinopril and Spironolactone Preserves Cardiac and Skeletal Muscle in Duchenne Muscular Dystrophy Mice

(2011) Jill A. Rafael-Fortney et al.   CIRCULATION

Cardiac Involvement in Patients With Muscular Dystrophies

(2011) David Verhaert et al.   Circulation-Cardiovascular Imaging

Losartan Decreases Cardiac Muscle Fibrosis and Improves Cardiac Function in Dystrophin-Deficient Mdx Mice

(2011) Christopher F. Spurney et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY AND THERAPEUTICS

The Carboxy-Terminal Third of Dystrophin Enhances Actin Binding Activity

(2011) Davin M. Henderson et al.   JOURNAL OF MOLECULAR BIOLOGY

Dystrophin Dp71: The Smallest but Multifunctional Product of the Duchenne Muscular Dystrophy Gene

(2011) Ramin Tadayoni et al.   MOLECULAR NEUROBIOLOGY

AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice

(2011) Brian Bostick et al.   MOLECULAR THERAPY

Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

(2011) HaiFang Yin et al.   MOLECULAR THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

P1.14 Cardiac transplantation in Duchenne muscular dystrophy: A case report

(2011) L. Cripe et al.   NEUROMUSCULAR DISORDERS

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse

(2011) Jonathon M. Tinsley et al.   PLoS One

Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy

(2011) F. S. Tedesco et al.   Science Translational Medicine

The Muscular Dystrophies: Distinct Pathogenic Mechanisms Invite Novel Therapeutic Approaches

(2011) Zarife Sahenk et al.   Current Rheumatology Reports

Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy

(2010) Vinod Malik et al.   ANNALS OF NEUROLOGY

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

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

Soft substrates drive optimal differentiation of human healthy and dystrophic myotubes

(2010) Elena Serena et al.   Integrative Biology

Surface-patterned electrode bioreactor for electrical stimulation

(2010) Nina Tandon et al.   LAB ON A CHIP

Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs

(2010) Joe N Kornegay et al.   MOLECULAR THERAPY

Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice

(2010) Alberto Malerba et al.   MOLECULAR THERAPY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Myostatin reduces Akt/TORC1/p70S6K signaling, inhibiting myoblast differentiation and myotube size

(2009) Anne Ulrike Trendelenburg et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice

(2009) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

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

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

Mapping of the Lipid-Binding and Stability Properties of the Central Rod Domain of Human Dystrophin

(2009) Sébastien Legardinier et al.   JOURNAL OF MOLECULAR BIOLOGY

Cardiac magnetic resonance imaging and management of dilated cardiomyopathy in a Duchenne muscular dystrophy manifesting carrier

(2009) A. Barison et al.   JOURNAL OF NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression

(2009) En Kimura et al.   MOLECULAR THERAPY

Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: A comparative study from the Pediatric Cardiomyopathy Registry

(2008) David M. Connuck et al.   AMERICAN HEART JOURNAL

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

(2008) Kathryn R. Wagner et al.   ANNALS OF NEUROLOGY

Production of arrays of cardiac and skeletal muscle myofibers by micropatterning techniques on a soft substrate

(2008) Elisa Cimetta et al.   BIOMEDICAL MICRODEVICES

Highly Efficient, Functional Engraftment of Skeletal Muscle Stem Cells in Dystrophic Muscles

(2008) Massimiliano Cerletti et al.   CELL

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

(2008) Brian Bostick et al.   HUMAN GENE THERAPY

Beneficial effects of beta-blockers and angiotensin-converting enzyme inhibitors in Duchenne muscular dystrophy

(2008) Hitoko Ogata et al.   Journal of Cardiology

Embryonic cardiomyocytes beat best on a matrix with heart-like elasticity: scar-like rigidity inhibits beating

(2008) A. J. Engler et al.   JOURNAL OF CELL SCIENCE

By-passing the nonsense mutation in the 4CVmouse model of muscular dystrophy by induced exon skipping

(2008) Chalermchai Mitrpant et al.   JOURNAL OF GENE MEDICINE

Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles

(2008) Stéphanie Lorain et al.   MOLECULAR THERAPY

Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines

(2008) Lawrence T Bish et al.   MOLECULAR THERAPY

Recombinant Adeno-associated Virus Transduction and Integration

(2008) Brian R Schultz et al.   MOLECULAR THERAPY

Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer

(2008) Helen Foster et al.   MOLECULAR THERAPY

Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice

(2008) Paul Gregorevic et al.   MOLECULAR THERAPY

Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy

(2008) Larry W. Markham et al.   NEUROMUSCULAR DISORDERS

Sildenafil and cardiomyocyte-specific cGMP signaling prevent cardiomyopathic changes associated with dystrophin deficiency

(2008) M. Khairallah et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA