Duchenne Muscular Dystrophy

This volume explores experimental approaches used to study Duchenne muscular dystrophy (DMD), an X-linked degenerative skeletal muscle disease caused by mutations in the dystrophin gene. Including the latest progress and scientific achievements, the book covers recent discoveries achieved through in vivo gene editing which have proven to be promising in restoring dystrophin expression, at least in ameliorating skeletal muscle symptoms, and the contents focus on Omics techniques in gene expression, protein expression, miRNAs, and long non-coding RNA analysis, as well as experimental studies of the structural/functional changes affecting the skeletal and cardiac muscles and ongoing preclinical studies and clinical trials. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshootingand avoiding known pitfalls.
Authoritative and practical, Duchenne Muscular Dystrophy: Methods and Protocols serves as a guide for researchers exploring the complicated nature of dystrophin in the hope of helping the victims of this disorder.

Includes cutting-edge methods and protocols for DMD research Provides step-by-step detail essential for reproducible results Contains key notes and implementation advice from the experts Includes supplementary material: sn.pub/extras

Inhalt

An Overview of Recent Therapeutics Advances for Duchenne Muscular Dystrophy.- Clinical Manifestations and Overall Management Strategies for Duchenne Muscular Dystrophy.- Cardiac Involvement in Duchenne Muscular Dystrophy and Related Dystrophinopathies.- Characterization of the Inflammatory Response in Dystrophic Muscle Using Flow Cytometry.- Imaging Analysis of the Neuromuscular Junction in Dystrophic Muscle.- System Biology Approach: Gene Network Analysis for Muscular Dystrophy.- Proteomic Profiling of the Dystrophin-Deficient Brain.- Probing the Pathogenesis of Duchenne Muscular Dystrophy Using Mouse Models.- Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy.- Designing Effective Antisense Oligonucleotides for Exon Skipping.- Identification of Splicing Factors Involved in DMD Exon Skipping Events Using an In Vitro RNA Binding Assay.- The Use of Antisense Oligonucleotides for the Treatment of Duchenne Muscular Dystrophy.- PMO Delivery System Using Bubble Liposomes and Ultrasound Exposure for Duchenne Muscular Dystrophy Treatment.- Proton Nuclear Magnetic Resonance (1H NMR) Spectroscopy-Based Analysis of Lipid Components in Serum / Plasma of Patients with Duchenne Muscular Dystrophy (DMD).- Test of Anti-Fibrotic Drugs in a Cellular Model of Fibrosis Based on Muscle-Derived Fibroblasts from Duchenne Muscular Dystrophy Patients.- Flow Cytometry-Defined CD49d Expression in Circulating T-Lymphocytes is a Biomarker for Disease Progression in Duchenne Muscular Dystrophy.- Advanced Methods to Study the Cross-Talk Between Fibro-Adipogenic Progenitors and Muscle Stem Cells.- AAV6 Vector Production and Purification for Muscle Gene Therapy.- From gRNA Identification to the Restoration of Dystrophin Expression: A Dystrophin Gene Correction Strategy for Duchenne Muscular Dystrophy Mutations Using the CRISPR-Induced Deletion Method.