Gene Delivery And Therapy For Neurological Disorders PDF Download
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Author | : Xuenong Bo |
Publisher | : Humana |
Total Pages | : 0 |
Release | : 2016-10-05 |
Genre | : Medical |
ISBN | : 9781493953165 |
Download Gene Delivery and Therapy for Neurological Disorders Book in PDF, ePub and Kindle
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.
Author | : Mingjie Li |
Publisher | : Academic Press |
Total Pages | : 450 |
Release | : 2018-05-25 |
Genre | : Medical |
ISBN | : 012809821X |
Download Gene Therapy in Neurological Disorders Book in PDF, ePub and Kindle
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
Author | : P. R. Lowenstein |
Publisher | : |
Total Pages | : 468 |
Release | : 1996-07-25 |
Genre | : Medical |
ISBN | : |
Download Protocols for Gene Transfer in Neuroscience Book in PDF, ePub and Kindle
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.
Author | : E. Antonio Chiocca |
Publisher | : Springer Science & Business Media |
Total Pages | : 458 |
Release | : 1997-10-23 |
Genre | : Medical |
ISBN | : 1592594786 |
Download Gene Therapy for Neurological Disorders and Brain Tumors Book in PDF, ePub and Kindle
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.
Author | : Fredric P. Manfredsson |
Publisher | : Humana |
Total Pages | : 0 |
Release | : 2015-11-26 |
Genre | : Medical |
ISBN | : 9781493932702 |
Download Gene Therapy for Neurological Disorders Book in PDF, ePub and Kindle
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.
Author | : Michael G. Kaplitt |
Publisher | : Gulf Professional Publishing |
Total Pages | : 380 |
Release | : 2006 |
Genre | : Medical |
ISBN | : 9780123976321 |
Download Gene Therapy of the Central Nervous System: From Bench to Bedside Book in PDF, ePub and Kindle
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.
Author | : Hari S. Sharma |
Publisher | : Springer |
Total Pages | : 237 |
Release | : 2017-06-05 |
Genre | : Medical |
ISBN | : 3319576968 |
Download Drug and Gene Delivery to the Central Nervous System for Neuroprotection Book in PDF, ePub and Kindle
This book summarizes the latest research on drug and gene delivery to the central nervous system (CNS). The chapters address safety concerns regarding the nanotechnology that is needed to develop nanomedicine for clinical practice. Particular focus is given to new technologies that have emerged in recent years to deliver therapeutic materials, such as genes, drugs, and other agents using nanotechnologies of diverse origin. This is an ideal book for students, teachers, researchers, and clinicians interested in a deeper understanding of nanotechnological advances in therapeutic medicine. This book also: Broadens readers’ understanding of viral vector gene delivery to the brain for treating neurogenetic diseases as well as targeted gene delivery into the brain using microbubble-facilitated focused ultrasound Covers in detail the latest developments in delivering therapeutic materials, such as siRNA delivery to the brain for treating neurological diseases, neuroprotective effects of gelatin nanoparticles in stroke, and nanowired drug delivery for brain diseases, heat stroke, and CNS injury Enriches understanding of new technologies for delivering therapeutic materials treating Alzheimer’s Disease, including targeted nanodrug delivery through the blood-brain barrier and the superior neuroprotective effects of nanowired drug delivery in Alzheimer’s Disease
Author | : Fredric P. Manfredsson |
Publisher | : |
Total Pages | : 493 |
Release | : 2016 |
Genre | : Biomedicine |
ISBN | : 9781493932719 |
Download Gene Therapy for Neurological Disorders Book in PDF, ePub and Kindle
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.
Author | : |
Publisher | : Elsevier |
Total Pages | : 297 |
Release | : 1995-11-24 |
Genre | : Science |
ISBN | : 0080532403 |
Download Genetic Manipulation of the Nervous System Book in PDF, ePub and Kindle
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.
Author | : National Academies of Sciences, Engineering, and Medicine |
Publisher | : National Academies Press |
Total Pages | : 93 |
Release | : 2019-10-31 |
Genre | : Medical |
ISBN | : 0309495873 |
Download Advancing Gene-Targeted Therapies for Central Nervous System Disorders Book in PDF, ePub and Kindle
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.