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 in the context of emerging therapeutic modalities. The only available source on the subject, this reference emphasizes the importance of accurate diagnosis, carrier detection, and genetic counseling, and supplies state-of-the-art contributions on pharmacological interventions, regenerative medicine, and gene therapy.
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.