Duchenne Muscular Dystrophy

Jeffrey S. Chamberlain, Thomas A. Rando

Clinical Overview. The Functional Biology of Dystrophin. Duchenne Muscular Dsytrophy and Becker Muscular Dystrophy: Diagnostic Principles. Mutation Detection. Protein Studies. Medical Management. Rehabilitation Management. A Review of Current Practice and Clinical Outcomes. Therapeutic Principles and Challenges. Experimental Pharmacologic Therapies. Utrophin: The Intersection between Pharmacological and Genetic Therapy. Regenerative Therapy. Cellular-Mediated Delivery: The Intersection between Regenerative Medicine and Genetic Therapy. Oligonucleotide-Mediated Rxon Skipping and Gene Editing. Intravascular Delivery of Naked DNA. Adenoviral-Mediated Gene Therapy. Retroviridae-based Gene Transfer Vectors. Gene Therapy Using Adeno-Associated Viral Vectors. Regional and Systemic Gene Delivery using Viral Vectors.