Download or read book Viral Vectors for Treating Diseases of the Nervous System written by David S. Latchman and published by Gulf Professional Publishing. This book was released on 2003-08-22 with total page 360 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy offers considerable potential for the treatment of various incurable diseases of the nervous system. Viral Vectors for Treating Disease of the Nervous System describes a number of different viral vectors developed for achieving high efficiency gene delivery to the brain. Vectors described include those based on adenovirus, adeno-associated virus, Herpes Simplex Virus, lentivirus, and other retroviruses. It also discusses the potential application of such viruses in treating brain tumors, Parkinson's disease, and other diseases of the nervous system. Provides up-to-date account of gene therapy approaches for incurable neurological disorders Describes a range of gene delivery methods based on different viruses

Download or read book Viral Vectors written by Michael G. Kaplitt and published by Academic Press. This book was released on 1995-08-14 with total page 486 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Advancing Gene Targeted Therapies for Central Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2019-12-01 with total page 93 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Gene Therapy of the Central Nervous System From Bench to Bedside written by Michael G. Kaplitt and published by Gulf Professional Publishing. This book was released on 2006 with total page 380 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Viral Vectors for Gene Therapy written by Curtis A. Machida and published by Springer Science & Business Media. This book was released on 2008-02-02 with total page 591 pages. Available in PDF, EPUB and Kindle. Book excerpt: Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdoctoral training. The “Notes” section contained in each chapter provides valuable troublesho- ing guides to help develop working protocols for your laboratory. With Viral Vectors for Gene Therapy: Methods and Protocols, it has been my intent to develop a comprehensive collection of modern molecular methods for the construction, development, and use of viral vectors for gene transfer and gene therapy. I would like to thank the many chapter authors for their contributions. They are all experts in various aspects of viral vectors, and I appreciate their efforts and hard work in developing comprehensive chapters. As editor, it has been a privilege to preview the development of Viral Vectors for Gene Therapy: Methods and Protocols, and to acquire insight into the various methodological approaches from the many different contri- tors.

Download or read book Adenoviral Vectors for Gene Therapy written by David T. Curiel and published by Academic Press. This book was released on 2016-03-10 with total page 868 pages. Available in PDF, EPUB and Kindle. Book excerpt: Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Download or read book Viral Vectors written by Michael G. Kaplitt and published by . This book was released on 1995 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Gene Therapy for Neurological Disorders written by Fredric P. Manfredsson and published by Humana. This book was released on 2015-11-26 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Gene Therapy for the Central and Peripheral Nervous System written by Andrew P. Tosolini and published by Frontiers Media SA. This book was released on 2018-05-10 with total page 220 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy is at the forefront of current techniques that aim to re-establish functional connectivity, after an insult to the brain, spinal cord or peripheral nerves. Gene therapy makes the most of the existing cellular machinery and anatomical networks to facilitate molecular changes in DNA, RNA and proteins aiming to repair these disrupted connections. For instance, gene therapy is currently being used to target genes in conditions including spinal cord injury, amyotrophic lateral sclerosis, spinal muscular atrophy, stroke and multiple sclerosis, amongst others. The various delivery routes include viral-vectors, genetically modified cellular implants, naked DNA/RNA, liposomes, Cre-Lox recombination, optogenetics and nanoparticles. In particular, gene therapy aims to restore function by augmenting the expression of neuroprotective/axonal growth-promoting neurotrophic factors (e.g., BDNF, CNTF, NGF and GDNF, etc.). Furthermore, the downstream intracellular signalling pathways after receptor activation can also be targeted (e.g., mTor, MAPK, etc.). On the other hand, gene therapy can also be used to downregulate and/or remove faulty mutated genes, such as those contributing to disease progression or that inhibit axonal regeneration (e.g., SOD-1, TDP-43, Nogo-A, MAG, OmGP, etc.). Depending on the methodology, these genes, for instance, can be silenced, removed or replaced to alleviate the underlying pathology. As such, gene therapy can transform a largely toxic and inhibitory milieu surrounding a neuronal/axonal insult into a growth-permissive environment that will ultimately aid neuronal survival and functional regeneration. Moreover, gene therapy has the capacity to target non-neuronal cells and can be even used for neuroanatomical tract tracing. Ultimately, the principal outcome of gene therapy is to functionally restore damaged neuronal and/or axonal connections irrespective of the system it is being introduced in to. This Research Topic is devoted to work using gene therapy for the both the central and/or peripheral nervous system.

Download or read book Viral Vector Approaches in Neurobiology and Brain Diseases written by Riccardo Brambilla and published by Humana. This book was released on 2016-08-23 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Aiming toward improvement in the safety, efficiency, and specificity of viral vectors for neurobiological research and clinical applications, Viral Vector Approaches in Neurobiology and Brain Diseases covers key aspects related to the use of viral vectors in neuroscience, with a major emphasis on basic mechanisms of synaptic plasticity, learning, and memory, as well as molecular neuropharmacology and experimental animal models of brain disorders. The volume begins by delving into features of the viral vectors currently available in neuroscience and their production methods, and it then continues onward to examples of successful applications of viral vector technology to psychiatric and memory research, current applications of viral vector technology in the context of neurological disorders, as well as various cutting-edge applications of viral vector technology to neuroscience, including optogenetics. Written for the Neuromethods series, the chapters of this book contain the kind of detailed description and implementation advice that promotes successful, repeatable results. Practical and up to date, Viral Vector Approaches in Neurobiology and Brain Diseases will be useful not only to neurobiologists wishing to routinely use viral vectors in the laboratory but also to experienced scientists needing detailed new protocols for a variety of experimental applications.

Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.

Download or read book Gene Therapy for Neurological Disorders and Brain Tumors written by E. Antonio Chiocca and published by Springer Science & Business Media. This book was released on 1997-10-23 with total page 458 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Gene Therapy in Neurological Disorders written by Mingjie Li and published by Academic Press. This book was released on 2018-05-25 with total page 450 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.

Download or read book Gene Therapy in the CNS Progress and Prospects for Novel Therapies written by Casper René Gøtzsche and published by Frontiers Media SA. This book was released on 2021-12-21 with total page 188 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Pediatric Neurology Part III written by Patrick Aubourg and published by Elsevier Inc. Chapters. This book was released on 2013-04-24 with total page 732 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Genetic Manipulation of the Nervous System written by and published by Elsevier. This book was released on 1995-11-24 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.