Muscle Gene Therapy

Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac Muscle.- Codon Optimization of the Micro-Dystrophin Gene for Duchene Muscular Dystrophy Gene Therapy.- Monitoring Duchenne Muscular Dystrophy Gene Therapy with Epitope-Specific Monoclonal Antibodies.- Methods for Non-Invasive Monitoring of Muscle Fiber Survival with an AAV Vector Encoding the mSEAP Reporter Gene.- Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy.- Phenotyping Cardiac Gene Therapy in Mice.- Golden Retriever Muscular Dystrophy (GRMD): Developing and Maintaining a Colony and Physiological Functional Measurements.- Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene Therapy.- Systemic Gene Transfer to Skeletal Muscle Using Reengineered AAV Vectors.- Bioinformatic and Functional Optimization of Antisense Phosphorodiamidate Morpholino Oligomers (PMOs) for Therapeutic Modulation of RNA Splicing in Muscle.- Engineering Exon-Skipping Vectors Expressing U7snRNA Constructs for Duchenne Muscular Dystrophy Gene Therapy.- Application of MicroRNA in Cardiac and Skeletal Muscle Disease Gene Therapy.- Molecular Imaging of RNA Interference Therapy Targeting PHD2 for Treatment of Myocardial Ischemia.- Lentiviral Vector Delivery of shRNA into Cultured Primary Myogenic Cells: A Tool for Therapeutic Target Validation.- Fetal Muscle Gene Therapy/Gene Delivery in Large Animals.- Electroporation of Plasmid DNA to Swine Muscle.- Local Gene Delivery and Methods to Control Immune Responses in Muscles of Normal and Dystrophic Dogs.- Gene Transfer to Muscle from the Isolated Regional Circulation.- AAV-Mediated Gene Therapy to the Isolated Limb in Rhesus Macaques.- Antisense Oligo-Mediated Multiple Exon-Skipping in a Dog Model of Duchenne Muscular Dystrophy.- Whole Body Skeletal Muscle Transduction in Neonatal Dogs with AAV-9.- A Translatable, Closed Recirculation System for AAV6 Vector-Mediated Myocardial Gene Delivery in the Large Animal.- Method of Gene Delivery in Large Animal Models of Cardiovascular Diseases.- Percutaneous Transendocardial Delivery of Self-Complementary Adeno-Associated Virus 6 in the Canine.