Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of childhood and currently stands as an incurable condition. This authoritative guide provides a clear overview of the latest current and experimental approaches to the treatment of DMD and examines the clinical, genetic, and pathophysiological aspects of the disease i
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.