Download or read book Cell and Gene Therapies for Neurologic Diseases written by and published by Elsevier. This book was released on 2024-10-01 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cell and Gene Therapies for Neurologic Diseases, Volume 205 comprehensively covers the scientific background, translational efforts, clinical developments and registered biologics that have entered into clinical practice. Coverage includes types of therapies available and in development, and best practice uses for a variety of neurological disorders including Parkinson’s, Huntington’s, ALS, stroke, spinal cord RP, demyelination, and epilepsy. As the emergence of gene and cellular therapeutics has changed the clinical landscape for a variety of disorders, and is now ready to do so for neurological diseases, these therapeutic modalities currently complement, and may in time, supplant small molecule drugs. Summarizes advances in cell and gene therapy for neurological diseases Describes the therapies available and in development Includes surgical, ethical, and manufacturing considerations Identifies best practices for specific neurological diseases Covers Huntington’s, Parkinson’s, ALS, Stroke, Demyelination, epilepsy, and more
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 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 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 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 Advances in Cell and Gene Therapy in Treating Neural Diseases written by Raymond Ching-Bong Wong and published by Frontiers Media SA. This book was released on 2022-01-07 with total page 252 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Gene Therapy for Neurological Disorders written by Pedro R. Lowenstein and published by CRC Press. This book was released on 2006-05-01 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Current Challenges in Cell Therapy for Neurodegenerative Diseases written by and published by Academic Press. This book was released on 2022-11-22 with total page 326 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cell Transplantation and Gene Therapy in Neurodegenerative Disease, Volume 166 in the International Review of Neurobiology series, highlights new advances in the field with this new volume presenting interesting chapters written by an international board of authors who cover Challenges in translating a cell therapy to GMP, The challenges in developing a cell therapy for Huntington’s disease, Challenges of cell therapies for retinal diseases, Challenges of gene therapy in Huntington’s Disease, Technological advances and barriers to gene therapy, Considerations in the development of cell therapy modulation for spinal cord injury treatment, Challenges of developing glial cell therapy for ALS, and more. Other chapters in this comprehensive release include Exploring cell and gene therapy in current animal models of Parkinson’s and Huntington’s disease, Considerations for the use of biomaterials to support cell therapy in degenerative disease, Neurosurgical challenges/innovations in cell and gene therapy delivery, Neuroimaging: the challenge of harnessing imaging tools to facilitate cell and gene therapy in neurodegenerative diseases/The contribution and challenges for imaging in advanced therapies of movement disorders, Considerations for clinical trial design for novel advanced therapeutics in neurodegenerative disease, and More than a trial participant: The role of the patient in ATMP development and trials for neurodegenerative disease. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review on Neurobiology serials Updated release includes the latest information on Cell Transplantation and Gene Therapy in Neurodegenerative Disease
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 Stem Cell based Therapy for Neurodegenerative Diseases written by Fabin Han and published by Springer Nature. This book was released on 2020-10-26 with total page 144 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book reviews the state-of-the-art in stem-cell-based therapies for neurodegenerative diseases, and highlights advances in both animal models and clinical trials. It comprehensively discusses most neurodegenerative diseases, including such as Parkinson's, Alzheimer's and Huntington’s diseases, amyotrophic sclerosis, multiple sclerosis, muscular dystrophy and retinal degeneration, in which stem cells could potentially be used for therapy in the future. It also addresses the challenges and problems relating to the translation of stem-cell-based therapies into treatments. As such, the book will appeal to research scientists, physicians, graduate students, and medical professionals in the field of stem cells, neuroscience, neurology, neurorestoratology and major neurological disorders.
Download or read book The Prospects of Gene Therapy written by Ernst Freese and published by . This book was released on 1972 with total page 30 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Gene Delivery and Therapy for Neurological Disorders written by Xuenong Bo and published by Humana. This book was released on 2016-10-05 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
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.
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 Development of Gene Therapies written by Avery McIntosh and published by Chapman & Hall/CRC Biostatistics Series. This book was released on 2024-03-13 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development.
Download or read book Protocols for Gene Transfer in Neuroscience written by P. R. Lowenstein and published by . This book was released on 1996-07-25 with total page 468 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Gene Transfer and Therapy in the Nervous System written by Fred H. Gage and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 226 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer technology is a powerful tool for increasing our understandingof brain functions. It is also the basis of gene therapy, which is now technically possible for the correction of many human diseases, including several disorders of the nervous (and muscular) system such as Alzheimer's disease, Parkinson's disease, and dystrophy. This volume,which contains the proceedings of a symposium of the Fondation Ipsen, provides a unique view of the state of the art on different transgenes, vectors, target cells, and clinical applications related to the nervous system.
