Genome Editing for Neurodegenerative Diseases: From Concept to Clinical Trials examines recent advances in neurodegenerative disease research and clinical challenges in practical applications. With an emphasis on genome editing tools such as ZFNs, TALENSs and CRISPR-Cas, this book sheds light on the possibilities to counteract genetic and neurodegenerative diseases. It will be of interest to researchers in neuroscience and biopharmaceuticals, as well as clinicians specializing in neurodegenerative diseases. - Covers basic mechanisms of genome-editing tools and their therapeutic potential - Highlights current research progress and therapeutic strategies to use genome-editing tools against neurodegenerative diseases - Includes research on genome-editing tool development, from basic concept to clinical trials

This reference book provides a comprehensive overview of models and therapeutic approaches against neurodegenerative diseases, including Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, and amyotrophic lateral sclerosis. It explores models based on the chemical, induced, cellular, genetic, transgenic, and 3D organoid approaches in neurodegenerative diseases. The book also reviews advantages and limitations of these models in designing the treatment strategies. Additionally, the book covers the emerging field of bioinformatics and its application in modeling various neurodegenerative diseases. Towards the end, the book highlights the role of holistic management, precision medicine, OMICS, and gene therapy against neurodegenerative disorders. It examines the implications and significance of stem cells therapy in translational models of neurodegenerative diseases. This book is an invaluable resource for researchers, neuroscientists, and neurosurgeons for getting in-depth information on the neurodegenerative models and therapeutic approaches. Key Features: Provides a comprehensive overview of neurodegenerative diseases and their models Examines the limitations associated with modeling neurodegenerative diseases Presents novel treatment strategies for Alzheimer's disease using cellular models Reviews importance of 3D organoid models for therapeutic approaches in Parkinson's disease Covers modeling techniques in understanding prion diseases Explores the role of genetic models in understanding Huntington's disease

Neurological illnesses pose one of the biggest hazards to the healthcare system today. This new book brings together the latest methods of gene therapy that can be used to treat both inherited and sporadic neurodegenerative diseases. It presents the most significant advances in gene transfer methods as well as the most recent understandings of the mechanisms behind specific neurodegenerative illnesses, placing these into the context of gene therapy approaches for the central nervous system. The book introduces the basics of neurodegenerative conditions and the physiological basis for their occurrence in humans. It also discusses the various proteins useful in neuro diseases and their viral applications. The book explores how stem cells can be used to learn about the future of gene therapy along with the significance of the sequences that silence genes. It discusses the functions of astrocytes in different brain regions, as well as in-vivo research in gene therapy for neurodegenerative disorders.

Provides a timely overview of critical advances in molecular and cellular neurobiology, covers key methodologies driving progress, and highlights key future directions for research on neuronal injury and neurodegeneration relevant to neuronal brain pathologies. The editors bring together contributions from internationally recognized workers in the field to provide an up to date account of how and why molecular and cellular neurobiology is such an important area for clinical neuroscience. Understanding the molecular aspects of a number of neurodegenerative conditions such as Parkinson's or Alzheimer's disease for the purpose of improving patient management remains a major challenge of neurobiology be it from the basic or clinical perspective. A strategic evaluation of research contributions and the power of modern methods will help advance knowledge over the next years.

Handbook of Innovations in CNS Regenerative Medicine provides a comprehensive overview of the CNS regenerative medicine field. The book describes the basic biology and anatomy of the CNS and how injury and disease affect its balance and the limitations of the present therapies used in the clinics. It also introduces recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies. Finally, the book presents successful cases of translation of basic research to first-in-human trials and the steps needed to follow this path. Areas such as cell transplantation approaches, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies are key in regenerative medicine are covered in the book, along with regulatory and ethical issues. - Describes the basic biology and anatomy of the CNS and how injury and disease affect its balance - Discusses the limitations of present therapies used in the clinics - Introduces the recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies, and enabling technologies - Presents successful cases of translation of basic research to first-in-human trials, along with the steps needed to follow this path

This book explores the pivotal role of synaptic plasticity in the pathogenesis, progression, and potential treatment of neurodegenerative disorders. The initial chapter provides an in-depth understanding of the complexity and impact of neurodegenerative conditions. It discusses the association of mitochondrial dysfunction, epigenetic influences, and neuroinflammation with synaptic plasticity in neurodegenerative diseases. The following chapters review the dynamic changes that occur at the cellular and synaptic levels in Parkinson's disease, Alzheimer's disease, and Huntington's disease, paving the way for innovative therapeutic strategies. Furthermore, the book presents various computational tools and methodologies essential for enhancing our understanding of synaptic plasticity. It examines the transformative role of artificial intelligence tools in addressing synaptic impairment across various neurodegenerative diseases. Discusses the role of synaptic plasticity in neurodegenerative diseases, shedding light on how dynamic changes occur at the cellular and synaptic levels Explores the transformative role of artificial intelligence tools in addressing synaptic impairment across various neurodegenerative diseases Provides a comprehensive overview of neurodegenerative disorders, including pathogenesis, etiology, and treatment strategies Presents tools and techniques used to simulate the complex system biology of synaptic plasticity Examines the role of computational neuroscience in understanding and potentially treating conditions such as multiple sclerosis and amyotrophic lateral sclerosis Toward the end, the book explores the role of synaptic impairment and computational neuroscience in understanding and potentially treating conditions such as multiple sclerosis and amyotrophic lateral sclerosis. With its multifaceted approach, this book serves as a useful resource for researchers, clinicians, and students in the fields of neuroscience, computational biology, and neurology.

Disease-Modifying Targets in Neurodegenerative Disorders: Paving the Way for Disease-Modifying Therapies examines specific neurodegenerative disorders in comprehensive chapters written by experts in the respective fields. Each chapter contains a summary of the disease management field, subsequently elaborating on the molecular mechanisms and promising new targets for disease-modifying therapies. This overview is ideal for neuroscientists, biomedical researchers, medical doctors, and caregivers, not only providing readers with a summary of the way patients are treated today, but also offering a glance at the future of neurodegenerative disorder treatment. - Provides a comprehensive overview of how key proteins in neurodegenerative disorders can be used as targets to modify disease progress - Summarizes how patients are treated today, providing a glance at future disease management - Includes intelligible and informative information that is perfect for non-specialists, medical practitioners, and scientists - Written and peer reviewed by outstanding scientists in their respective fields

Neurodegenerative diseases share the common property of neuronal loss in the higher-order association and limbic cortices or the extrapyramidal and pyramidal motor systems. In addition, oligodendroglia, astrocytes, and microglia have been implicated in fundamental abnormalities of virtually every neurodegenerative disorder. The particular system affected, more importantly the distribution of the pathology, determines the clinical presentation. While the most common dementia and movement disorders, such as Alzheimer’s disease, Lewy body disease, and frontotemporal lobar degeneration with TDP-43 pathology, including amyotrophic lateral sclerosis, have been extensively studied, many less common, even rare neurodegenerative disorders have gained more attention in recent years. This shift in focus is perhaps driven, in part, by the severely underestimated financial costs associated with these diseases, as well as the immense emotional burden they impose on patients and their caregivers. This book presents the most recent developments in rare neurodegenerative disorders. Insights gained from the investigation of pathophysiological mechanisms of these rare disorders may lead to the development of therapeutic strategies for more prevalent neurodegenerative disorders. In addition to highlighting advancements in research, the book discusses the significant challenges faced by researchers and healthcare professionals in diagnosing and treating rare diseases. It emphasizes the critical need for continued funding and support for research, which is essential to improving patient outcomes and advancing our understanding of these complex conditions.