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Gene Therapy in Neurological Disorders

Gene Therapy in Neurological Disorders
  • Author : Mingjie Li,B. Joy Snider
  • Publisher :Unknown
  • Release Date :2018-05-25
  • Total pages :450
  • ISBN : 9780128098219
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Summary : Gene therapy has tremendous potential for the treatment of neurological disorders. There has been substantial progress in the development of gene therapy strategies for neurological disorders over the last two decades. Gene Therapy in Neurological Disorders thoroughly reviews currently available gene therapy tools and presents examples of their application in a variety of neurological diseases. The book begins with general reviews of gene therapy strategies with a focus on neurological disorders. The remainder of the chapters present approaches to specific neurological disorders. Each chapter gives an in-depth introduction to the relevant field before diving into the specific tool or application. The book aims to help investigators, students and research staff better understand the principles of gene therapy and its application in the nervous system. Provides background information and experimental details of gene therapy tools applied for neuroscience research and neurological disorders Covers a broad range of gene delivery and regulation tools, therapeutic agents, and target cells, including emerging new technologies such as CRISPR/Cas9 genome editing Discusses applications of gene therapy tools to neurological disorders including neurodegeneration, muscular dystrophy, trauma and chronic pain, and neoplastic diseases

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders
  • Author : Pedro R. Lowenstein,Maria G. Castro
  • Publisher :Unknown
  • Release Date :2006
  • Total pages :354
  • ISBN : 0429136242
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Summary :

Gene Therapy for Neurological Disorders and Brain Tumors

Gene Therapy for Neurological Disorders and Brain Tumors
  • Author : E. Antonio Chiocca,Xandra O. Breakefield, Ph.D.,Xandra O. Breakefield
  • Publisher :Unknown
  • Release Date :1998
  • Total pages :458
  • ISBN : UOM:39015040033220
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Summary : The authoritative contributions provide cutting-edge reviews of the vectors available for gene transfer to the central nervous system, the strategies against CNS tumors, the potential strategies against neurologic disorders, and the limitations of today's gene therapy approaches. Also discussed are significant applications of gene therapy to brain tumors, Parkinson's disease, ischemia, and Huntington's chorea.

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders
  • Author : Pedro R. Lowenstein,Maria G. Castro
  • Publisher :Unknown
  • Release Date :2006-05-01
  • Total pages :384
  • ISBN : 0824728475
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Summary : Coedited by internationally recognized leaders in gene therapy research, this guide supplies the most recent advances, studies, and expert opinion on gene therapy for neurological disorders. Spanning conditions such as Parkinsons's and Alzheimer's disease, multiple sclerosis, brain tumors, stroke, epilepsy, and brain and spinal cord trauma, this reference presents the basic science and applications of recombinant gene products, immune and stem cell-mediated therapeutic strategies, and brain tumor gene therapy.

Rosenberg's Molecular and Genetic Basis of Neurological and Psychiatric Disease

Rosenberg's Molecular and Genetic Basis of Neurological and Psychiatric Disease
  • Author : Roger N. Rosenberg,Juan M. Pascual
  • Publisher :Unknown
  • Release Date :2014-10-28
  • Total pages :1464
  • ISBN : 9780124105492
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Summary : Rosenberg’s Molecular and Genetic Basis of Neurologic and Psychiatric Disease, Fifth Edition provides a comprehensive introduction and reference to the foundations and key practical aspects relevant to the majority of neurologic and psychiatric disease. A favorite of over three generations of students, clinicians and scholars, this new edition retains and expands the informative, concise and critical tone of the first edition. This is an essential reference for general medical practitioners, neurologists, psychiatrists, geneticists, and related professionals, and for the neuroscience and neurology research community. The content covers all aspects essential to the practice of neurogenetics to inform clinical diagnosis, treatment and genetic counseling. Every chapter has been thoroughly revised or newly commissioned to reflect the latest scientific and medical advances by an international team of leading scientists and clinicians. The contents have been expanded to include disorders for which a genetic basis has been recently identified, together with abundant original illustrations that convey and clarify the key points of the text in an attractive, didactic format. Previous editions have established this book as the leading tutorial reference on neurogenetics. Researchers will find great value in the coverage of genomics, animal models and diagnostic methods along with a better understanding of the clinical implications. Clinicians will rely on the coverage of the basic science of neurogenetics and the methods for evaluating patients with biochemical abnormalities or gene mutations, including links to genetic testing for specific diseases. Comprehensive coverage of the neurogenetic foundation of neurological and psychiatric disease Detailed introduction to both clinical and basic research implications of molecular and genetic understanding of the brain Detailed coverage of genomics, animal models and diagnostic methods with new coverage of evaluating patients with biochemical abnormalities or gene mutations

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders
  • Author : Frederic P Manfredsson
  • Publisher :Unknown
  • Release Date :2015-11-26
  • Total pages :493
  • ISBN : 1493932705
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Summary : This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course of experimentation and will answer questions such as: What delivery vehicle do you use?; How will you ensure that your vector retains stability?; What expression system best fits your needs?; What route will you choose to deliver your gene therapy agent?; How will you model the neurodegenerative disorder that you aim to investigate and what are the proven methods to treat these disorders in preclinical models? Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols and tips on troubleshooting and avoiding known pitfalls. Authoritative and thorough, Gene Therapy for Neurological Disorders: Methods and Protocols, is a compilation of protocols and instructive chapters intended to give researchers, clinicians, and students of all levels, a foundation upon which future gene therapy experiments can be designed.

Gene Therapy of the Central Nervous System: From Bench to Bedside

Gene Therapy of the Central Nervous System: From Bench to Bedside
  • Author : Michael G. Kaplitt,Matthew During
  • Publisher :Unknown
  • Release Date :2006
  • Total pages :355
  • ISBN : 0123976324
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Summary : Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use

Selected Topics in Neonatal Care

Selected Topics in Neonatal Care
  • Author : René Mauricio Barría
  • Publisher :Unknown
  • Release Date :2018-06-27
  • Total pages :248
  • ISBN : 9781789233629
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Summary : Neonatology is one of the areas of greatest development and evolution within pediatrics. The technoscientific advances in this area have led to an increase in the survival of premature infants who sometimes require sophisticated care. However, there is essential care that must be included in all centers that care for high-risk babies. This book includes important topics related to neonatal care grouped into four sections. In 14 chapters that address relevant issues about neonatal care, the book seeks to contribute to the clinical work of the health teams of neonatal units. Specialists in the field of neonatology from different countries have developed these chapters and through them they hope to share part of their experience.

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

Advancing Gene-Targeted Therapies for Central Nervous System Disorders
  • Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
  • Publisher :Unknown
  • Release Date :2019-10-31
  • Total pages :92
  • ISBN : 9780309495875
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Summary : On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.

Molecular Neurology

Molecular Neurology
  • Author : Stephen Waxman
  • Publisher :Unknown
  • Release Date :2010-07-26
  • Total pages :600
  • ISBN : 0080546188
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Summary : Why a book on molecular neurology? Molecular neuroscience is advancing at a spectacular rate. As it does so, it is revealing important clues to the pathogenesis and pathophysiology of neurological diseases, and to the therapeutic targets that they present. Medicines work by targeting molecules. The more specific the targeting, the more specific the actions, and the fewer the side effects. Molecular Neurology highlights, for graduate and MD-PhD students, research fellows and research-oriented clinical fellows, and researchers in the neurosciences and other biomedical sciences, the principles underlying molecular medicine as related to neurology. Written by internationally recognized experts, this well-illustrated and well-referenced book presents the most up-to-date principles and disease examples relevant to molecular neurology, and reviews the concepts, strategies, and latest progress in this field. This book will interest anyone studying the molecular basis of neurology, or developing new therapies in neurology. Describes the newest molecular aspects of neurological disorders Provides an introduction to neurological disorders for basic scientists Updates clinicians and clinical researchers on the most recent developments

Neurological Disorders

Neurological Disorders
  • Author : World Health Organization
  • Publisher :Unknown
  • Release Date :2006
  • Total pages :218
  • ISBN : 9789241563369
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Summary : Although there are several gaps in understanding the many issues related to neurological disorders, we know enough to be able to shape effective policy responses to some of the most common. This book describes and discusses the increasing public health impact of common neurological disorders such as dementia, epilepsy, headache disorders, multiple sclerosis, neuroinfections, neurological disorders associated with malnutrition, pain associated with neurological disorders, Parkinson's disease, stroke and traumatic brain injuries. It provides information and advice on public health interventions that may reduce their occurrence and consequences, and offers health professionals and planners the opportunity to assess the burden caused by these disorders. The clear message that emerges is that unless immediate action is taken globally, the neurological burden is likely to become an increasingly serious and unmanageable.

The Right Therapy for Neurological Disorders

The Right Therapy for Neurological Disorders
  • Author : E. Beghi,G. Logroscino
  • Publisher :Unknown
  • Release Date :2016-07-26
  • Total pages :168
  • ISBN : 9783318058659
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Summary : Most neurological disorders are chronic and aging-related. With the increase of life expectancy their incidence and prevalence will grow in the decades to come, which in turn will increase the load on medical and social systems worldwide. There is thus a desperate need for successful preventive and therapeutic measures based on randomized clinical trials (RTCs) conducted by independent organizations. This book provides a compendium relating most of the principles of reliable RTCs to specific neurological diseases. Contributed by specialized neurologists, the articles touch on important aspects of RCTs with a clear critical approach, highlighting their limitations as well as giving recommendations for their planning and conducting to address the variable genotypic and phenotypic aspects of neurological conditions. Consideration is also given to combining the clinical impact of the study results with patients’ values and the interests of pharmaceutical companies. Neurologists involved in clinical trials will certainly benefit from this book, which should become a basic text for all neurological courses dealing with evidence-based neurology.

Gene Therapy

Gene Therapy
  • Author : David Cooper,Prof Nick Lemoine
  • Publisher :Unknown
  • Release Date :2020-09-11
  • Total pages :360
  • ISBN : 9781000144680
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Summary : Gene Therapy describes the delivery systems now available to target a given tissue with specific gene or oligonucleotide sequences, and explores the utility of animal modules as test systems. In the context of selected disease states, it summarises in vitro and in vivo studies and clinical trials performed to date.

Gene Therapy

Gene Therapy
  • Author : Doaa Hashad
  • Publisher :Unknown
  • Release Date :2015-11-26
  • Total pages :218
  • ISBN : 9789535122210
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Summary : Gene therapy is becoming a promising technology for the management of many human diseases. Hereditary and acquired disorders can both be tackled using the technique of gene therapy. This book provides detailed, up-to-date topics addressing basic principles of gene therapy and discussing some of the challenges encountered by scientists in developing this relatively novel technology. The development of new and efficient gene transfer vectors is of utmost importance in the progress of the field of gene therapy. Both viral and non-viral vectors are extensively discussed. A detailed chapter elaborates the problem of host immune rejection of transplanted donor cells or engineered tissue that can be avoided using the encapsulation of transgenic cells, thus avoiding the use of drugs that achieve immunosuppression.

Current Concepts in Movement Disorder Management

Current Concepts in Movement Disorder Management
  • Author : A. Niranjan,L.D. Lunsford,R.M. Richardson
  • Publisher :Unknown
  • Release Date :2018-01-12
  • Total pages :272
  • ISBN : 9783318062021
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Summary : This book summarizes the current state of movement disorder management and the role of surgical therapies as an alternative to medication. Following a chapter on the history of movement disorder surgery, leaders in their fields describe the pathophysiology, functional neuroanatomy, clinical presentation, and medical management of Parkinson’s disease, dystonia, and essential tremor. This is followed by chapters on the spectrum of movement disorder surgery itself, from the lesioning procedures of radiofrequency ablation, stereotactic radiosurgery, and high-frequency ultrasound to the modulatory procedures of “asleep”, image-guided deep brain stimulation (DBS) and “awake”, microelectrode-guided DBS. The final chapters focus on closed-loop DBS, drug-delivery, gene therapy, and other emerging neurosurgical therapies, highlighting long-standing experimental strategies that are reaching exciting phases of clinical translation. This volume is a valuable tool for accessing the wide spectrum of concepts that currently define this dynamic field.

Disease Control Priorities, Third Edition (Volume 4)

Disease Control Priorities, Third Edition (Volume 4)
  • Author : Vikram Patel,Dan Chisholm,Tarun Dua,Ramanan Laxminarayan,Mari'a Lena Medina-Mora,Theo Vos
  • Publisher :Unknown
  • Release Date :2016-03-10
  • Total pages :304
  • ISBN : 9781464804281
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Summary : Mental, neurological, and substance use disorders are common, highly disabling, and associated with significant premature mortality. The impact of these disorders on the social and economic well-being of individuals, families, and societies is large, growing, and underestimated. Despite this burden, these disorders have been systematically neglected, particularly in low- and middle-income countries, with pitifully small contributions to scaling up cost-effective prevention and treatment strategies. Systematically compiling the substantial existing knowledge to address this inequity is the central goal of this volume. This evidence-base can help policy makers in resource-constrained settings as they prioritize programs and interventions to address these disorders.

An Introduction to Molecular Medicine and Gene Therapy

An Introduction to Molecular Medicine and Gene Therapy
  • Author : Thomas F. Kresina
  • Publisher :Unknown
  • Release Date :2004-04-07
  • Total pages :416
  • ISBN : 9780471461043
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Summary : Gene therapy, or the use of genetic manipulation for diseasetreatment, is derived from advances in genetics, molecular biology,clinical medicine, and human genomics. Molecular medicine, theapplication of molecular biological techniques to disease treatmentand diagnosis, is derived from the development of human organtransplantation, pharmacotherapy, and elucidation of the humangenome. An Introduction to Molecular Medicine and GeneTherapy provides a basis for interpreting new clinical andbasic research findings in the areas of cloning, gene transfer, andtargeting; the applications of genetic medicine to clinicalconditions; ethics and governmental regulations; and the burgeoningfields of genomics, biotechnology, and bioinformatics. By dividingthe material into three sections - an introduction to basicscience, a review of clinical applications, and a discussion of theevolving issues related to gene therapy and molecular medicine-thiscomprehensive manual describes the basic approaches to the broadrange of actual and potential genetic-based therapies. In addition, An Introduction to Molecular Medicine andGene Therapy: Covers new frontiers in gene therapy, animal models, vectors,gene targeting, and ethical/legal considerations Provides organ-based reviews of current studies in gene therapyfor monogenetic, multifactoral or polygenic disorders, andinfectious diseases Includes bold-faced terms, key concepts, summaries, and listsof helpful references by subject in each chapter Contains appendices on commercial implications and a review ofthe history of gene therapy This textbook offers a clear, concise writing style, drawingupon the expertise of the authors, all renowned researchers intheir respective specialties of molecular medicine. Researchers ingenetics and molecular medicine will all find An Introductionto Molecular Medicine and Gene Therapy to be an essentialguide to the rapidly evolving field of gene therapy and itsapplications in molecular medicine.

Neurobiology of Brain Disorders

Neurobiology of Brain Disorders
  • Author : Michael J. Zigmond,Joseph T. Coyle,Lewis P. Rowland
  • Publisher :Unknown
  • Release Date :2014-12-03
  • Total pages :824
  • ISBN : 9780123982803
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Summary : Neurobiology of Brain Disorders is the first book directed primarily at basic scientists to offer a comprehensive overview of neurological and neuropsychiatric disease. This book links basic, translational, and clinical research, covering the genetic, developmental, molecular, and cellular mechanisms underlying all major categories of brain disorders. It offers students, postdoctoral fellows, and researchers in the diverse fields of neuroscience, neurobiology, neurology, and psychiatry the tools they need to obtain a basic background in the major neurological and psychiatric diseases, and to discern connections between basic research and these relevant clinical conditions. This book addresses developmental, autoimmune, central, and peripheral neurodegeneration; infectious diseases; and diseases of higher function. The final chapters deal with broader issues, including some of the ethical concerns raised by neuroscience and a discussion of health disparities. Included in each chapter is coverage of the clinical condition, diagnosis, treatment, underlying mechanisms, relevant basic and translational research, and key unanswered questions. Written and edited by a diverse team of international experts, Neurobiology of Brain Disorders is essential reading for anyone wishing to explore the basic science underlying neurological and neuropsychiatric diseases. Links basic, translational, and clinical research on disorders of the nervous system, creating a format for study that will accelerate disease prevention and treatment Covers a vast array of neurological disorders, including ADHD, Down syndrome, autism, muscular dystrophy, diabetes, TBI, Parkinson, Huntington, Alzheimer, OCD, PTSD, schizophrenia, depression, and pain Illustrated in full color Each chapter provides in-text summary points, special feature boxes, and research questions Provides an up-to-date synthesis of primary source material

Disease and Mortality in Sub-Saharan Africa

Disease and Mortality in Sub-Saharan Africa
  • Author : Dean T. Jamison
  • Publisher :Unknown
  • Release Date :2006-01-01
  • Total pages :387
  • ISBN : 9780821363980
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Summary : Current data and trends in morbidity and mortality for the sub-Saharan Region as presented in this new edition reflect the heavy toll that HIV/AIDS has had on health indicators, leading to either a stalling or reversal of the gains made, not just for communicable disorders, but for cancers, as well as mental and neurological disorders.

Current Therapy in Neurologic Disease

Current Therapy in Neurologic Disease
  • Author : Richard Tidball Johnson,John W. Griffin,Justin C. McArthur
  • Publisher :Unknown
  • Release Date :2006
  • Total pages :447
  • ISBN : 0323034322
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Summary : Neurologists present their own experiences and step-by-step guidelines on the management and treatment of specific disorders affecting the nervous system. Diagnosis, epidemiology, and pathophysiology are discussed only when they affect treatment strategies and decisions. Treatment algorithms and patient resource information complement each chapter.

Principles of Molecular Neurosurgery

Principles of Molecular Neurosurgery
  • Author : Andrew Freese
  • Publisher :Unknown
  • Release Date :2005-01-01
  • Total pages :659
  • ISBN : 9783805577847
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Summary : The future of neurosurgery will be characterized by less invasive, molecular technologies that promise to revolutionize the field of neurosurgery and impact the treatment of additional neurological disorders, including neurometabolic diseases, stroke, dementias, affective and psychiatric diseases, movement disorders, epilepsy, and others. This book encompasses developing an understanding of the principles underlying the advent of novel molecular approaches to neurological and neurosurgical diseases. It Identifies key principles that will allow dramatic improvement in the treatment and outcomes of patients suffering from a varity of disorders affecting the central nervous system and spinal axis. This volume gives neurosurgeons an excellent understanding of the development of novel molecular and cellular technologies that will markedly change the way neurosurgery is practiced in the near future. It is also of special interest to neurologists, psychiatrists, physiatrists, spinal orthopaedic surgeons, neurobiologists and gene therapy research scientists.