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.
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.
Table of Contents
1. Gene Delivery Modalities for Neurodegenerative Disorders2. Current Advances and Challenges Associated with CNS-targeted Gene Delivery Overview of Gene therapy in Neurodegenerative Disorders
3. Gene Therapy in Spinal Muscular Atrophy (SMA)
4. Gene Therapy in Amyotrophic Lateral Sclerosis (ALS)
5. Gene Therapy in Duchenne Muscular Dystrophy (DMD)
6. Gene Therapy in Neuronal Ceroid Lipofuscinosis (NCL)
7. Gene Therapy in Mucopolysaccharidoses (MPSs)
8. Gene Therapy in Giant Axonal Neuropathy (GAN)
9. Gene Therapy in Limb-Girdle Muscular Dystrophy (LGMD)
10. Gene Therapy in Pompe Disease
11. Gene Therapy in Huntington’s Disease (HD)
12. Gene Therapy in Spinocerebellar Ataxia (SCA)
13. Gene Therapy in Alzheimer’s Disease (AD)
14. Gene Therapy in Parkinson’s Disease (PD)
15. Gene Therapy in Facioscapulohumeral Muscular Dystrophy (FSHD)
