Gene Therapy in Neurological Disorders
Gene Therapy in Neurological Disorders

Author: Mingjie Li

Publisher: Academic Press

Published: 2018-05-25

Total Pages: 450

ISBN-13: 012809821X

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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 and Brain Tumors
Gene Therapy for Neurological Disorders and Brain Tumors

Author: E. Antonio Chiocca

Publisher: Springer Science & Business Media

Published: 1997-10-23

Total Pages: 458

ISBN-13: 1592594786

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Leading gene therapy researchers and clinicians illuminate the field-from basic vector technology to current and future clinical applications in neurology. The authoritative contributors 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 disorder, 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. Readers will learn the current delivery methods for transgenes, will learn the characteristics of transgene delivery vectors, and come to understand the therapy for both neuro-oncologic and neurologic disorders.

Gene Therapy Methods
Gene Therapy Methods

Author: M. Ian Phillips

Publisher: Academic Press

Published: 2002-02-22

Total Pages: 770

ISBN-13: 0121822478

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This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease

Genetic Manipulation of the Nervous System
Genetic Manipulation of the Nervous System

Author:

Publisher: Elsevier

Published: 1995-11-24

Total Pages: 297

ISBN-13: 0080532403

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Neuroscience Perspectives provides multidisciplinary reviews of topics in one of the most diverse and rapidly advancing fields in the life sciences.Whether you are a new recruit to neuroscience, or an established expert, look to this series for 'one-stop' sources of the historical, physiological, pharmacological, biochemical, molecular biological and therapeutic aspects of chosen research areas.The recent development of Gene Therapy procedures which allow specific genes to be delivered to human patients who lack functional copies of them is of major therapeutic importance. In addition such gene delivery methods can be used in other organisms to define the function of particular genes. These studies are of particular interest in the nervous system where there are many incurable diseases like Alzheimer's and Parkinson's diseases which may benefit from therapies of this kind. Unfortunately gene delivery methods for use in the nervous system have lagged behind those in other systems due to the fact that the methods developed in other systems are often not applicable to cells like neurons which do not divide. This book discusses a wide range of methods which have now been developed to overcome these problems and allow safe and efficient delivery of particular genes to the brain. Methods discussed include virological methods, physical methods (such as liposomes) and the transplantation of genetically modified cells. In a single volume therefore this book provides a complete view of these methods and indicates how they can be applied to the development of therapies for treating previously incurable neurological disorders.

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

Publisher: Gulf Professional Publishing

Published: 2006

Total Pages: 380

ISBN-13: 9780123976321

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Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology 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 in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who 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 which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system.

Viral Vectors
Viral Vectors

Author: Michael G. Kaplitt

Publisher: Academic Press

Published: 1995-08-14

Total Pages: 525

ISBN-13: 008054357X

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Genetic manipulation of the adult mammalian nervous system is one of the most exciting areas in contemporary neurobiology. The explosive growth of this field has been facilitated by harnessing the power of viruses to transfer genetic material into mammalian cells.Viral Vectors: Gene Therapy and Neuroscience Applications represents the first comprehensive review of viral vector applications to the nervous system by leaders in virology, molecular neurobiology, neuroanatomy, and developmental neurobiology. It serves both as a source of fundamental information for those newly interested in viral vectors and as a compilation of state-of-the-art technologies and applications for more experienced researchers.This work provides expert background information on viral systems, and the broad range of applications will help readers appreciate the current and future impact of viral vectors in both clinical and basic neuroscience.

Genome Editing in Neurosciences
Genome Editing in Neurosciences

Author: Rudolf Jaenisch

Publisher:

Published: 2020-10-08

Total Pages: 128

ISBN-13: 9781013268564

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Innovations in molecular biology are allowing neuroscientists to study the brain with unprecedented resolution, from the level of single molecules to integrated gene circuits. Chief among these innovations is the CRISPR-Cas genome editing technology, which has the precision and scalability to tackle the complexity of the brain. This Colloque Médecine et Recherche has brought together experts from around the world that are applying genome editing to address important challenges in neuroscience, including basic biology in model organisms that has the power to reveal systems-level insight into how the nervous system develops and functions as well as research focused on understanding and treating human neurological disorders. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors.

Oligonucleotide-Based Drugs and Therapeutics
Oligonucleotide-Based Drugs and Therapeutics

Author: Nicolay Ferrari

Publisher: John Wiley & Sons

Published: 2018-07-31

Total Pages: 576

ISBN-13: 1118537335

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A comprehensive review of contemporary antisense oligonucleotides drugs and therapeutic principles, methods, applications, and research Oligonucleotide-based drugs, in particular antisense oligonucleotides, are part of a growing number of pharmaceutical and biotech programs progressing to treat a wide range of indications including cancer, cardiovascular, neurodegenerative, neuromuscular, and respiratory diseases, as well as other severe and rare diseases. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide covering all key aspects of this increasingly popular area of pharmacology and biotech and pharma research, from the basic science behind antisense oligonucleotides chemistry, toxicology, manufacturing, to safety assessments, the design of therapeutic protocols, to clinical experience. Antisense oligonucleotides are single strands of DNA or RNA that are complementary to a chosen sequence. While the idea of antisense oligonucleotides to target single genes dates back to the 1970's, most advances have taken place in recent years. The increasing number of antisense oligonucleotide programs in clinical development is a testament to the progress and understanding of pharmacologic, pharmacokinetic, and toxicologic properties as well as improvement in the delivery of oligonucleotides. This valuable book reviews the fundamentals of oligonucleotides, with a focus on antisense oligonucleotide drugs, and reports on the latest research underway worldwide. • Helps readers understand antisense molecules and their targets, biochemistry, and toxicity mechanisms, roles in disease, and applications for safety and therapeutics • Examines the principles, practices, and tools for scientists in both pre-clinical and clinical settings and how to apply them to antisense oligonucleotides • Provides guidelines for scientists in drug design and discovery to help improve efficiency, assessment, and the success of drug candidates • Includes interdisciplinary perspectives, from academia, industry, regulatory and from the fields of pharmacology, toxicology, biology, and medicinal chemistry Oligonucleotide-Based Drugs and Therapeutics belongs on the reference shelves of chemists, pharmaceutical scientists, chemical biologists, toxicologists and other scientists working in the pharmaceutical and biotechnology industries. It will also be a valuable resource for regulatory specialists and safety assessment professionals and an important reference for academic researchers and post-graduates interested in therapeutics, antisense therapy, and oligonucleotides.

Disease Control Priorities, Third Edition (Volume 4)
Disease Control Priorities, Third Edition (Volume 4)

Author: Vikram Patel

Publisher: World Bank Publications

Published: 2016-03-10

Total Pages: 307

ISBN-13: 1464804281

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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.