Gene Therapy in Neurodegenerative Disorders: From Bench to Bedside offers a comprehensive review of gene therapy modalities and their applications in treating neurodegenerative disorders. Initial chapters focus on the fundamentals of gene therapy strategies, including gene delivery methods and CNS-targeted gene delivery. Subsequent chapters delve into specific neurodegenerative disorders such as Spinal Muscular Atrophy (SMA), Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), and Alzheimer's Disease (AD), highlighting the latest gene therapy approaches for each condition. Each chapter provides a detailed introduction to the relevant field before exploring specific tools and applications.
With contributions from leading experts, the book addresses current and ongoing clinical trials, potential challenges, and promising advances in gene therapy for neurodegenerative disorders. It also discusses regulatory and ethical considerations in translating research from bench to bedside. This book is an essential resource for basic science researchers in gene therapy, geneticists, biotechnologists, and clinical researchers seeking new and effective treatments for neurodegenerative diseases. It equips readers with the knowledge and tools needed to advance their work and drive innovation in gene therapy.
1. Chapter 1 Introduction - p. 1 2. Chapter 2 Gene-editing technologies and gene delivery modalities for neurodegenerative disorders: Current advances and challenges - p. 3 3. Chapter 3 Ethical and regulatory aspects of gene therapy in neurodegenerative disorders - p. 49 4. Section I: Neuromuscular disorders - p. S1 5. Chapter 4 Gene therapy in spinal muscular atrophy (SMA) - p. 79 6. Chapter 5 Gene therapy in amyotrophic lateral sclerosis (ALS) - p. 97 7. Chapter 6 Gene therapy in Duchenne muscular dystrophy (DMD) - p. 113 8. Chapter 7 Gene therapy in limb-girdle muscular dystrophy (LGMD) - p. 153 9. Section II: Lysosomal storage disorders - p. S2 10. Chapter 8 Gene therapy in leukodystrophies - p. 175 11. Chapter 9 Gene therapy in neuronal ceroid lipofuscinosis (NCL) - p. 203 12. Section III: Neurodegenerative disorders - p. S3 13. Chapter 10 Gene therapy in Alzheimer’s disease (AD) - p. 231 14. Chapter 11 Gene therapy in Parkinson’s disease (PD) - p. 269 15. Chapter 12 Gene therapy in Huntington’s disease (HD) - p. 311 16. Chapter 13 Gene therapy in spinocerebellar ataxia (SCA) - p. 359 17. Index - p. 375
Gene Therapy in Neurodegenerative Disorders: From Bench to Bedside offers a comprehensive review of gene therapy modalities and their applications in treating neurodegenerative disorders. Initial chapters focus on the fundamentals of gene therapy strategies, including gene delivery methods and CNS-targeted gene delivery. Subsequent chapters delve into specific neurodegenerative disorders such as Spinal Muscular Atrophy (SMA), Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), and Alzheimer's Disease (AD), highlighting the latest gene therapy approaches for each condition. Each chapter provides a detailed introduction to the relevant field before exploring specific tools and applications.
With contributions from leading experts, the book addresses current and ongoing clinical trials, potential challenges, and promising advances in gene therapy for neurodegenerative disorders. It also discusses regulatory and ethical considerations in translating research from bench to bedside. This book is an essential resource for basic science researchers in gene therapy, geneticists, biotechnologists, and clinical researchers seeking new and effective treatments for neurodegenerative diseases. It equips readers with the knowledge and tools needed to advance their work and drive innovation in gene therapy.
1. Chapter 1 Introduction - p. 1 2. Chapter 2 Gene-editing technologies and gene delivery modalities for neurodegenerative disorders: Current advances and challenges - p. 3 3. Chapter 3 Ethical and regulatory aspects of gene therapy in neurodegenerative disorders - p. 49 4. Section I: Neuromuscular disorders - p. S1 5. Chapter 4 Gene therapy in spinal muscular atrophy (SMA) - p. 79 6. Chapter 5 Gene therapy in amyotrophic lateral sclerosis (ALS) - p. 97 7. Chapter 6 Gene therapy in Duchenne muscular dystrophy (DMD) - p. 113 8. Chapter 7 Gene therapy in limb-girdle muscular dystrophy (LGMD) - p. 153 9. Section II: Lysosomal storage disorders - p. S2 10. Chapter 8 Gene therapy in leukodystrophies - p. 175 11. Chapter 9 Gene therapy in neuronal ceroid lipofuscinosis (NCL) - p. 203 12. Section III: Neurodegenerative disorders - p. S3 13. Chapter 10 Gene therapy in Alzheimer’s disease (AD) - p. 231 14. Chapter 11 Gene therapy in Parkinson’s disease (PD) - p. 269 15. Chapter 12 Gene therapy in Huntington’s disease (HD) - p. 311 16. Chapter 13 Gene therapy in spinocerebellar ataxia (SCA) - p. 359 17. Index - p. 375
