Muscular Dystrophy Therapeutics

Current Strategies of Muscular Dystrophy Therapeutics: An Overview.- The Story of Viltolarsen: From Preclinical Studies to FDA Approval.- Rapid Freezing of Skeletal and Cardiac Muscles Using Isopentane Cooled with Liquid Nitrogen and Tragacanth Gum for Histological, Genetic, and Protein Expression Studies.- Cardiac and Skeletal Muscle Pathology in the D2/mdx Mouse Model and Caveats Associated with the Quantification of Utrophin.- Physiological Assessment of Muscle, Heart, and Whole Body Function in the Canine Model of Duchenne Muscular Dystrophy.- Restoring Dystrophin Expression by Skipping Exon 6 and 8 in Neonatal Dystrophic Dogs.- Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes.- Restoring Dystrophin Expression with Duchenne Muscular Dystrophy Exon 45 Skipping in Induced-Pluripotent Stem Cell-Derived Cardiomyocytes.- Quantitative Evaluation of Exon Skipping in Urine-Derived Cells for Duchenne Muscular Dystrophy.- Use of Glycine to Augment Exon Skipping and Cell Therapies for Duchenne Muscular Dystrophy.- Morpholino-Mediated Exons 28–29 Skipping in Dysferlin.- Knocking Down DUX4 in Immortalized Facioscapulohumeral Muscular Dystrophy Patient-Derived Muscle Cells.- Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.- Developing Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G Mutation.- Molecular and Biochemical Assessment of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy.- Histological Assessment of Gene Therapy in the Canine DMD Model.- MRI Evaluation of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy.- Assessment of the Gene Therapy Immune Response in the Canine Muscular Dystrophy Model.- Use of Mesenchymal Stem Cells to Enhance the Efficacy of Gene Therapy.- Exon-Skipping for a Pathogenic COL6A1 Variant in Ullrich CMD.- CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice by a Self-Complementary AAV Delivery System.- Preparation of NanoMEDIC Extracellular Vesicles to Deliver CRISPR-Cas9 Ribonucleoproteins for Genomic Exon Skipping.- Restoration of Dystrophin Expression in Mdx-Derived Muscle Progenitor Cells Using CRISPR/Cas9 System and Homology-Directed Repair Technology.- Effects of Glucocorticoids in Murine Models of Duchenne and Limb-Girdle Muscular Dystrophy.- High-Throughput Screening to Identify Modulators of Sarcospan.- Identifying FDA-Approved Drugs that Upregulate Utrophin A as a Therapeutic Strategy for Duchenne Muscular Dystrophy.- Monitoring Membrane Injury-Triggered Endocytosis at Single Cell and Single Vesicle Resolution.- Evaluation of hiPSC-Derived Muscle Progenitor Cell Transplantation in a Mouse Duchenne Muscular Dystrophy Model.- Quantification of Muscle Satellite Stem Cell Divisions by High Content Analysis.- Systemic Delivery of a Monoclonal Antibody to Immunologically Block Myostatin in the A17 Mouse Model of OPMD.