Gene Therapy in Neurological Disorders

Gene Therapy in Neurological Disorders

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.

Author: Mingjie Li

Publisher: Academic Press

ISBN: 9780128098219

Category: Medical

Page: 450

View: 947

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
Categories: Medical

Gene Therapy for Neurological Disorders and Brain Tumors

Gene Therapy for Neurological Disorders and Brain Tumors

Gene Therapy for Neurological Disorders and Brain Tumors illuminates the rapidly developing applications of gene transfer technologies to the treatment of a wide range of neurological disorders and brain tumors.

Author: E. Antonio Chiocca

Publisher: Springer Science & Business Media

ISBN: 9781592594788

Category: Medical

Page: 458

View: 884

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.
Categories: Medical

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders

This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results.

Author: Fredric P. Manfredsson

Publisher: Humana Press

ISBN: 1493948962

Category: Medical

Page: 493

View: 184

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.
Categories: Medical

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders

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.

Author: Pedro R. Lowenstein

Publisher: CRC Press

ISBN: 0824728475

Category: Medical

Page: 384

View: 599

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.
Categories: Medical

Gene Delivery and Therapy for Neurological Disorders

Gene Delivery and Therapy for Neurological Disorders

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 ...

Author: Xuenong Bo

Publisher: Humana Press

ISBN: 1493953168

Category: Medical

Page: 359

View: 155

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.
Categories: Medical

Pediatric Neurology Part III

Pediatric Neurology Part III

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.

Author: Patrick Aubourg

Publisher: Elsevier Inc. Chapters

ISBN: 9780128084571

Category: Medical

Page: 732

View: 421

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.
Categories: Medical

Protocols for Gene Transfer in Neuroscience

Protocols for Gene Transfer in Neuroscience

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 ...

Author: P. R. Lowenstein

Publisher: John Wiley & Sons Incorporated

ISBN: IND:30000042796585

Category: Medical

Page: 417

View: 724

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.
Categories: Medical

Advancing Gene Targeted Therapies for Central Nervous System Disorders

Advancing Gene Targeted Therapies for Central Nervous System Disorders

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 ...

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

ISBN: 9780309495844

Category: Medical

Page: 92

View: 412

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.
Categories: Medical