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. Gene Delivery Modalities for Neurodegenerative Disorders
2. 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 Huntingtons Disease (HD)
12. Gene Therapy in Spinocerebellar Ataxia (SCA)
13. Gene Therapy in Alzheimers Disease (AD)
14. Gene Therapy in Parkinsons Disease (PD)
15. Gene Therapy in Facioscapulohumeral Muscular Dystrophy (FSHD)
Dr. Vahid Mansouri's passion for continuous learning and innovative solutions led him from an MD degree at Isfahan University of Medical Sciences, where he was honored as the Outstanding Medical Student in Iran in 2017, to a focus on translational science. Actively involved in biomedical and clinical research, particularly in pediatrics, Dr. Mansouri has authored five first-authored papers. He pursued post-doctoral studies at Tehran University of Medical Sciences' gene therapy research center, gaining diverse skills in wet-lab, dry-lab, animal, and clinical trials. Dr. Mansouri has contributed to pioneering gene therapy projects in Iran, including treatments for X-linked retinitis pigmentosa and spinal muscular atrophy, and developed a clinical trial protocol for CD19 CAR-T cells. Outside of his professional endeavors, he is a national medalist in swimming and water polo, and enjoys trekking, camping, and mountain climbing, which bring balance and vitality to his life. Dr. Maryam Bemanalizadeh is a distinguished medical professional with a Doctorate of Medicine (MD) from Isfahan University of Medical Sciences and a Master of Business Administration (MBA) in Health from Shahid Beheshti University of Medical Sciences. Renowned for her research in neurodegenerative and neurodevelopmental disorders, gene therapy, and neuroscience, she has published extensively in high-impact journals. Dr. Bemanalizadeh's work includes pioneering gene therapy projects and developing clinical trial protocols. As a Research Associate at Tehran University of Medical Sciences and a Senior Researcher at Children's Medical Center, she leads innovative studies on spinal muscular atrophy and Alzheimer's disease. Her contributions to the field are complemented by her role as a mentor at NeuroTRACT Association. Outside of her professional endeavors, Dr. Bemanalizadeh enjoys hiking, camping, traveling, and cooking. She continues to inspire through her dedication to advancing medical science and improving patient care.
