Muscular Dystrophy Therapeutics -

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Résumé :
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....

Sommaire:
This detailed book presents a comprehensive collection of state-of-the-art protocols on muscular dystrophy therapeutics, covering therapeutics using antisense oligonucleotides, gene replacement, genome editing, small molecules, stem cells, and antibodies. Written by leaders in the field, the volume explores techniques that are currently in use and are starting an exciting therapeutic revolution in muscular dystrophy. As a part of the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step and readily reproducible laboratory protocols, as well as tips on troubleshooting and avoiding known pitfalls.