Molecular and Cellular Therapies for Motor Neuron Diseases discusses the basics of the diseases, also covering advances in research and clinical trials. The book provides a resource for students that will help them learn the basics in a detailed manner that is required for scientists and clinicians. Users will find a comprehensive overview of the background of Amyotrophic Lateral Sclerosis (ALS/Lou Gehrig's Disease) and Spinal Muscular Atrophy (SMA), along with the current understanding of their genetics and mechanisms. In addition, the book details gene and cell therapies that have been developed and their translation to clinical trials. - Provides an overview of gene and cell therapies for amyotrophic lateral sclerosis (ALS) and other motor neuron diseases - Edited by a leading Neurosurgeon and two research scientists to promote synthesis between basic neuroscience and clinical relevance - Presents a great resource for researchers and practitioners in neuroscience, neurology, and gene and cell therapy

The Molecular and Cellular Basis of Neurodegenerative Diseases: Underlying Mechanisms presents the pathology, genetics, biochemistry and cell biology of the major human neurodegenerative diseases, including Alzheimer's, Parkinson's, frontotemporal dementia, ALS, Huntington's, and prion diseases. Edited and authored by internationally recognized leaders in the field, the book's chapters explore their pathogenic commonalities and differences, also including discussions of animal models and prospects for therapeutics. Diseases are presented first, with common mechanisms later. Individual chapters discuss each major neurodegenerative disease, integrating this information to offer multiple molecular and cellular mechanisms that diseases may have in common. This book provides readers with a timely update on this rapidly advancing area of investigation, presenting an invaluable resource for researchers in the field. - Covers the spectrum of neurodegenerative diseases and their complex genetic, pathological, biochemical and cellular features - Focuses on leading hypotheses regarding the biochemical and cellular dysfunctions that cause neurodegeneration - Details features, advantages and limitations of animal models, as well as prospects for therapeutic development - Authored by internationally recognized leaders in the field - Includes illustrations that help clarify and consolidate complex concepts

Spinal Muscular Atrophy: Disease Mechanisms and Therapy provides the latest information on a condition that is characterized by motoneuron loss and muscle atrophy, and is the leading genetic cause of infant mortality. Since the identification of the gene responsible for SMA in 1995, there have been important advances in the basic understanding of disease mechanisms, and in therapeutic development. This book provides a comprehensive accounting of recent advances in basic and clinical research that covers SMA clinical features and standards of care, multifaceted aspects of SMN protein functions and SMA disease pathology, various animal models, and biomarkers, as well as current therapeutic development. This title is ideal for graduate students/postdocs and principal investigators who are already in the SMA field and need to keep updated on recent findings and approaches, and for those who are new to, or would like to join, the field. Likewise, users will find an excellent source of reading for biotech/pharma scientists, clinical researchers, and practitioners, regulators, and patients and their advocacy organizations. Furthermore, this book is a handy reference for researchers and clinicians who may want to apply the research strategies and therapeutic approaches in SMA to other rare diseases. - Provides comprehensive, up-to-date reviews by leading investigators on diverse topics of SMA, including clinical features and patient care, SMN genetics and protein functions, animal models, disease pathology and mechanisms, biomarkers, current therapeutic development, and the role of non-profit organizations in therapeutic development - Written to bridge multiple disciplines and promote better communications among basic scientists, clinical researchers, and health care providers on the latest developments in SMA - Includes outstanding questions and perspectives for future investigations and key references for additional detailed study

'Motor Neuron Disease in Adults' reviews new information from 1998 as it applies to all aspects of motor neuron disease. Articles included use evidence-based methods to ensure that the new information is solid and advances the topic. The book can be used by anyone who provides any type of care to ALS patients.

Motor neuron diseases are the most catastrophic of neurodegenerative disorders. The cognitive function is spared, but the motor neuron degeneration translates into progressive muscle weakness and paralysis that propel the afflicted patient to eventual death. Neurodegenerative disorders constitute one of the major challenges of modern medicine in view of the current lack of effective therapies. The fruit fly, Drosophila melanogaster, has a distinguished history as an important model organism capable of shaping our fundamental understanding of life. Remarkably, the vast majority of all known human disease genes have a similar fly counterpart and at the molecular and physiological level, the basic principles of neuromuscular function are amazingly conserved between humans and Drosophila. Combine this with the presence of numerous genetic tools developed over the last century allowing genes and the proteins they encode to be manipulated swiftly to decipher their in vivo function and you have a superb genetic animal model organism of disease.This publication singles out the past and recent accomplishments of Drosophila in modelling motor neuron disease including amyotrophic lateral sclerosis (Lou Gehrigs disease), hereditary spastic paraplegias, Charcot-Marie-Tooth disease, spinal and bulbar muscular atrophy (Kennedys disease) and spinal muscular atrophy. The emphasis is on recent developments including the emerging molecular pathways underpinning these disorders. Genetic screens aimed at identifying novel genes that cause motor neuron degeneration or finding modifiers of the phenotype resulting from the disruption of disease-causative genes are also tackled. Importantly, this collection provides an inspiring look at the indispensability of the fruit fly, and of model organisms in general, to neuroscience research.

This simple guide to neurogenetics demystifies the overwhelming amount of information on the subject so you can identify key clinical features and understand your management options. Reach relevant differential diagnoses and provide appropriate counseling to your patients using the symptom-based approach. By integrating genetic and neurological approaches to diagnoses, this book ensures that the neurological consequences of a genetic diagnosis and the genetic consequences of a neurological diagnosis are clear and explicit. Concise and portable, this book is ideal for easy reference in clinical use. Details the underlying basic science and clinical features of genetic disorders by taking a symptom-based approach to provide you with a comprehensive understanding of the field. Focuses on the clinical application of neurogenetics to be of practical use to you in the clinic. Clarifies the neurological consequences of a genetic diagnosis and the genetic consequences of a neurological diagnosis by integrating genetic and neurological approaches to diagnoses. Discusses and evaluates necessary investigations so you know when to use them and when to refer. Highlights diagnostic and therapeutic tips so you can learn new concepts or refine your skills in practice. Refers to online sources, such as Online Mendelian Inheritance in Man (OMIM) and others, to help you supplement your knowledge.