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.