相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy A Nonrandomized Controlled Trial
Jerry R. Mendell et al.
JAMA NEUROLOGY (2020)
Development of Novel Micro-dystrophins with Enhanced Functionality
Julian N. Ramos et al.
MOLECULAR THERAPY (2019)
AAV-based gene therapies for the muscular dystrophies
Julie M. Crudele et al.
HUMAN MOLECULAR GENETICS (2019)
Cardiac Pathophysiology and the Future of Cardiac Therapies in Duchenne Muscular Dystrophy
Tatyana A. Meyers et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)
Mineralocorticoid Receptor Signaling Contributes to Normal Muscle Repair After Acute Injury
J. Spencer Hauck et al.
FRONTIERS IN PHYSIOLOGY (2019)
Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate
Stephen C. Kolwicz et al.
JACC-BASIC TO TRANSLATIONAL SCIENCE (2019)
Simvastatin provides long-term improvement of left ventricular function and prevents cardiac fibrosis in muscular dystrophy
Min J. Kim et al.
PHYSIOLOGICAL REPORTS (2019)
Cardiac-Specific Expression of H2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy
Nalinda B. Wasala et al.
HUMAN GENE THERAPY (2018)
Cardiac MRI biomarkers for Duchenne muscular dystrophy
Patrick Magrath et al.
BIOMARKERS IN MEDICINE (2018)
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Caroline Le Guiner et al.
NATURE COMMUNICATIONS (2017)
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy
Chady H. Hakim et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
The Pathogenesis and Therapy of Muscular Dystrophies
Simon Guiraud et al.
ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS, VOL 16 (2015)
Claudin-5 levels are reduced from multiple cell types in human failing hearts and are associated with mislocalization of ephrin-B1
Sarah A. Swager et al.
CARDIOVASCULAR PATHOLOGY (2015)
Contemporary Cardiac Issues in Duchenne Muscular Dystrophy
Elizabeth M. McNally et al.
CIRCULATION (2015)
Dystrophin-glycoprotein complex regulates muscle nitric oxide production through mechanoregulation of AMPK signaling
Joanne F. Garbincius et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2015)
Regional Circumferential Strain is a Biomarker for Disease Severity in Duchenne Muscular Dystrophy Heart Disease: A Cross-Sectional Study
Kan N. Hor et al.
PEDIATRIC CARDIOLOGY (2015)
Alterations of dystrophin-associated glycoproteins in the heart lacking dystrophin or dystrophin and utrophin
Katharine M. Sharpe et al.
JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY (2013)
Prevalence and distribution of late gadolinium enhancement in a large population of patients with Duchenne muscular dystrophy: effect of age and left ventricular systolic function
Kan N. Hor et al.
JOURNAL OF CARDIOVASCULAR MAGNETIC RESONANCE (2013)
Patterns of left ventricular remodeling in patients with Duchenne Muscular Dystrophy: a cardiac MRI study of ventricular geometry, global function, and strain
Wojciech Mazur et al.
INTERNATIONAL JOURNAL OF CARDIOVASCULAR IMAGING (2012)
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
Brian Bostick et al.
JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY (2012)
Sustaining Cardiac Claudin-5 Levels Prevents Functional Hallmarks of Cardiomyopathy in a Muscular Dystrophy Mouse Model
Dawn A. Delfin et al.
MOLECULAR THERAPY (2012)
Cardiomyopathy in the dystrophin/utrophin-deficient mouse model of severe muscular dystrophy is characterized by dysregulation of matrix metalloproteinases
Dawn A. Delfin et al.
NEUROMUSCULAR DISORDERS (2012)
Proteomic Analysis Reveals New Cardiac-Specific Dystrophin-Associated Proteins
Eric K. Johnson et al.
PLOS ONE (2012)
Early Treatment With Lisinopril and Spironolactone Preserves Cardiac and Skeletal Muscle in Duchenne Muscular Dystrophy Mice
Jill A. Rafael-Fortney et al.
CIRCULATION (2011)
Cardiac Involvement in Patients With Muscular Dystrophies Magnetic Resonance Imaging Phenotype and Genotypic Considerations
David Verhaert et al.
CIRCULATION-CARDIOVASCULAR IMAGING (2011)
Distinct pathophysiological mechanisms of cardiomyopathy in hearts lacking dystrophin or the sarcoglycan complex
DeWayne Townsend et al.
FASEB JOURNAL (2011)
Age-matched comparison reveals early electrocardiography and echocardiography changes in dystrophin-deficient dogs
Deborah M. Fine et al.
NEUROMUSCULAR DISORDERS (2011)
GENDER INFLUENCES CARDIAC FUNCTION IN THE mdx MODEL OF DUCHENNE CARDIOMYOPATHY
Brian Bostick et al.
MUSCLE & NERVE (2010)
The Polyproline Site in Hinge 2 Influences the Functional Capacity of Truncated Dystrophins
Glen B. Banks et al.
PLOS GENETICS (2010)
Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy
Yi Lai et al.
JOURNAL OF CLINICAL INVESTIGATION (2009)
Cardiac Expression of a Mini-dystrophin That Normalizes Skeletal Muscle Force Only Partially Restores Heart Function in Aged Mdx Mice
Brian Bostick et al.
MOLECULAR THERAPY (2009)
Claudin-5 levels are reduced in human end-stage cardiomyopalhy
Tessily A. Mays et al.
JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY (2008)
Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle
DeWayne Townsend et al.
MOLECULAR THERAPY (2008)
Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure
DeWayne Townsend et al.
MOLECULAR THERAPY (2007)
Abnormalities of diastolic function precede dilated cardiomyopathy associated with Duchenne muscular dystrophy
Larry W. Markham et al.
JOURNAL OF THE AMERICAN SOCIETY OF ECHOCARDIOGRAPHY (2006)
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
Paul Gregorevic et al.
NATURE MEDICINE (2006)
Utrophin deficiency worsens cardiac contractile dysfunction present in dystrophin-deficient mdx mice
PML Janssen et al.
AMERICAN JOURNAL OF PHYSIOLOGY-HEART AND CIRCULATORY PHYSIOLOGY (2005)
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
P Gregorevic et al.
NATURE MEDICINE (2004)
Cardiomyopathic features associated with muscular dystrophy are independent of dystrophin absence in cardiovasculature
TA Hainsey et al.
NEUROMUSCULAR DISORDERS (2003)