Duchenne Muscular Dystrophy 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.

Features:

• Discusses challenges to the development of treatments for DMD
• Contains chapters on diagnostic modalities and innovations in molecular diagnostics… focuses on viral and nonviral gene therapy research, as well as promising stem cell therapeutics and potential targets for conventional drug approaches
• Considers intersections between different approaches to treatment and within the broader scope of the emerging field of regenerative medicine
• Highlights pathways for future research in the field

Contents

DMD Background

• Clinical Overview
• The Functional Biology of Dystrophin

Diagnostic Considerations

• Diagnostic Principles: Importance for Targeted Therapeutics
• Mutation Detection
• Protein Analysis

Current Therapeutics

• Medical Management
• Rehabilitation Management
• Orthopedic Management

Experimental Therapeutics

• 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 Gene Repair and Modulation
• Intravascular Delivery of Naked DNA
• Adenoviral-Mediated Gene Therapy
• Retroviridae-based Gene Transfer Vectors
• Adeno-associated Viral Vectors for DMD Gene Therapy
• Regional and Systemic Gene Delivery using Viral Vectors

Index

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