Gene Therapy In Neurological Disorders
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Gene Therapy In Neurological Disorders
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Author : Mingjie Li
language : en
Publisher: Academic Press
Release Date : 2018-05-25
Gene Therapy In Neurological Disorders written by Mingjie Li and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-05-25 with Medical categories.
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
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Author : E. Antonio Chiocca
language : en
Publisher: Springer Science & Business Media
Release Date : 1997-10-23
Gene Therapy For Neurological Disorders And Brain Tumors written by E. Antonio Chiocca and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 1997-10-23 with Medical categories.
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 For Neurological Disorders
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Author : Fredric P. Manfredsson
language : en
Publisher:
Release Date : 2016
Gene Therapy For Neurological Disorders written by Fredric P. Manfredsson and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2016 with Biomedicine categories.
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 For Neurological Disorders
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Author : Pedro R. Lowenstein
language : en
Publisher: CRC Press
Release Date : 2006-05-01
Gene Therapy For Neurological Disorders written by Pedro R. Lowenstein and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2006-05-01 with Medical categories.
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.
Gene Delivery And Therapy For Neurological Disorders
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Author : Xuenong Bo
language : en
Publisher: Humana
Release Date : 2016-10-05
Gene Delivery And Therapy For Neurological Disorders written by Xuenong Bo and has been published by Humana this book supported file pdf, txt, epub, kindle and other format this book has been release on 2016-10-05 with Medical categories.
This volume aims to explore the latest developments in adeno-associated viral and lentiviral vectors as well as the gene therapy strategies for the most common neurological disorders, followed by chapters that include step-by-step guides to viral vector-based gene delivery in animal models used in the authors’ laboratories. Although safe gene manipulation in neural cells can be achieved, it may still be years away from efficacious gene-based treatment of neurological disorders such as Parkinson's and Alzheimer’s diseases due to the complexity of the underlying genetic/molecular mechanisms and the difficulty of developing reliable animal models. Gene Delivery and Therapy for Neurological Disorders seeks to aid researchers in this vital work. Written in the popular Neuromethods series format, chapters include the kind of detailed description and expert implementation advice that leads to success in the lab. Meticulous and authoritative, Gene Delivery and Therapy for Neurological Disorders serves as an ideal guide for researchers attempting to explore the potentials of gene therapy for neurological disorders.
Advancing Gene Targeted Therapies For Central Nervous System Disorders
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Author : National Academies of Sciences, Engineering, and Medicine
language : en
Publisher: National Academies Press
Release Date : 2019-12-01
Advancing Gene Targeted Therapies For Central Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-12-01 with Medical categories.
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.
Gene Therapy For Neurological Disorders
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Author : Fredric P. Manfredsson
language : en
Publisher: Humana
Release Date : 2015-11-26
Gene Therapy For Neurological Disorders written by Fredric P. Manfredsson and has been published by Humana this book supported file pdf, txt, epub, kindle and other format this book has been release on 2015-11-26 with Medical categories.
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.
Protocols For Gene Transfer In Neuroscience
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Author : P. R. Lowenstein
language : en
Publisher:
Release Date : 1996-07-25
Protocols For Gene Transfer In Neuroscience written by P. R. Lowenstein and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1996-07-25 with Medical categories.
Protocols for Gene Transfer in Neuroscience Towards Gene Therapy of Neurological Disorders Edited by P. R. Lowenstein University of Manchester, UK and L. W. Enquist Princeton University, USA The brain and the cells that compose it have always presented unique barriers to any kind of outside interventions, including drug delivery, surgical procedures and the genetic manipulation of cells. However, these barriers are now being bypassed by the new technology of gene delivery. Protocols for Gene Transfer in Neuroscience is the first practical guide to gene delivery, providing over 150 easy to follow, step-by-step protocols, together with troubleshooting hints and the theoretical background needed for neuroscientists to understand, adapt and expand the technology as needed. Written by a team of international expert neuroscientists, geneticists, molecular biologists and biochemists, Protocols for Gene Transfer in Neuroscience describes the latest techniques from the top gene-delivery labs, ranging from physical transfection techniques, through the use of a wide range of viral vectors, to retrovirally transduced cell and bone marrow transplantation. Several variations of a single protocol are given, thus demonstrating how different labs have solved a common problem in different ways. The protocols are also indexed and cross-referenced so that an investigator can find all the details required for a particular problem.
Genetic Manipulation Of The Nervous System
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Author :
language : en
Publisher: Elsevier
Release Date : 1995-11-24
Genetic Manipulation Of The Nervous System written by and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 1995-11-24 with Science categories.
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.
Pediatric Neurology Part Iii
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Author : Patrick Aubourg
language : en
Publisher: Elsevier Inc. Chapters
Release Date : 2013-04-24
Pediatric Neurology Part Iii written by Patrick Aubourg and has been published by Elsevier Inc. Chapters this book supported file pdf, txt, epub, kindle and other format this book has been release on 2013-04-24 with Medical categories.
Though heavily hyped in the early 1990s as the answer to many genetic diseases, gene therapy has largely failed to live up to its promises up to now. The scientific challenges of conveying replacement genes into the brain have prevented it from having an impact on more than a handful of conditions. In addition, gene therapy has been beset by safety problems. During the last 5 years, new vectors and techniques have started to resolve not only safety issues that once held gene therapy back but also markedly to improve efficient gene delivery within the brain and even the spinal cord. Brain gene therapy will not be suitable for every neurological disease, but considerable progress made in the field means we can now envisage that CNS gene therapy will be able to treat many neurological conditions that are not obviously treatable in any other way.