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Gene Therapy In Neurological Disorders

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Gene Therapy for Neurological Disorders and Brain Tumors

Gene Therapy for Neurological Disorders and Brain Tumors Book
Author : E. Antonio Chiocca,Xandra O. Breakefield
Publisher : Springer Science & Business Media
Release : 1997-10-23
ISBN : 1592594786
Language : En, Es, Fr & De

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Book Description :

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

Gene Therapy for Neurological Disorders Book
Author : Frederic P Manfredsson
Publisher : Humana Press
Release : 2015-11-26
ISBN : 9781493932702
Language : En, Es, Fr & De

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Book Description :

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 in Neurological Disorders

Gene Therapy in Neurological Disorders Book
Author : Mingjie Li,B. Joy Snider
Publisher : Academic Press
Release : 2018-05-25
ISBN : 012809821X
Language : En, Es, Fr & De

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Book Description :

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 Delivery and Therapy for Neurological Disorders

Gene Delivery and Therapy for Neurological Disorders Book
Author : Xuenong Bo,Joost Verhaagen
Publisher : Humana Press
Release : 2016-10-05
ISBN : 9781493953165
Language : En, Es, Fr & De

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Book Description :

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.

Protocols for Gene Transfer in Neuroscience

Protocols for Gene Transfer in Neuroscience Book
Author : P. R. Lowenstein,L. W. Enquist
Publisher : John Wiley & Sons Incorporated
Release : 1996-07-25
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

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.

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders Book
Author : Pedro R. Lowenstein,Maria G. Castro
Publisher : Unknown
Release : 2006
ISBN : 9780429136245
Language : En, Es, Fr & De

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Book Description :

Download Gene Therapy for Neurological Disorders book written by Pedro R. Lowenstein,Maria G. Castro, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Gene Therapy of the Central Nervous System From Bench to Bedside

Gene Therapy of the Central Nervous System  From Bench to Bedside Book
Author : Michael G. Kaplitt,Matthew During
Publisher : Gulf Professional Publishing
Release : 2006
ISBN : 9780123976321
Language : En, Es, Fr & De

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Book Description :

Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far 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 of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and 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 of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders Book
Author : Anonim
Publisher : Unknown
Release : 2010
ISBN : 9789186443313
Language : En, Es, Fr & De

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Book Description :

Download Gene Therapy for Neurological Disorders book written by , available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Pediatric Neurology Part III

Pediatric Neurology Part III Book
Author : Patrick Aubourg
Publisher : Elsevier Inc. Chapters
Release : 2013-04-24
ISBN : 012808457X
Language : En, Es, Fr & De

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Book Description :

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.

Proceedings of 6th International Conference and Exhibition on Cell and Gene Therapy 2018

Proceedings of 6th International Conference and Exhibition on Cell and Gene Therapy 2018 Book
Author : ConferenceSeries
Publisher : ConferenceSeries
Release : 2021-12-08
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Mar 27-28, 2017 Madrid, Spain Key Topics : Cell Therapy, Cellular Therapy Technologies, Cell Therapy of Cardiovascular Disorders, Cell Therapy for Cancer, Cell Culture & Bioprocessing:, Cell Science & Stem Cell Research:, Cell Line Development, Tissue Science & Regenerative Medicine, Gene Therapy, Viral gene therapy, Diabetis Gene Therapy, Vectors for Gene Therapy, Molecular Epigenetics, Genetics & Genomic Medicine, Gene Therapy Commercialization, Clinical trials in cell and gene therapy, Gene Therapy for rare & Common Diseases, Gene Editing Technology, Cell Therapy for Neurological Disorders, Ethical Issues in Cell & Gene Therapy, Regulatory & Safety Aspects of Cell & Gene Therapy, Clinical Trails on Cell & Gen Therapy, Markets & Future Prospects for Cell & Gene Therapy, Cell & Gene Therapy Products,

Textbook of Neurogenetics

Textbook of Neurogenetics Book
Author : Adriano Jiménez Escrig
Publisher : Unknown
Release : 2007-09
ISBN : 9788479787929
Language : En, Es, Fr & De

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Book Description :

This text constitutes a comprehensive sinopsis of our current knowledge of thegenetics on neurological diseases. The book presents the wiews from various disciplines who share a common interest on Neurogenetics. The first sever chapters review basic genetics, from historical aspectcs to molecular genetic tools, the internet resources, and the statistics and software used in research and clinical ground. The remaining fourteen chapters provide valuable information on causes, clinical aspects, genetic diagnosis, treatment strategies and management of the main hereditary neurological disorders. These chapters covers miscellaneous neurological diseases as well as those categorized into groups, review the genetics in each process and provide advice about the steps involved to enable the clinician to make clinical, genetic and differential diagnoses. Besides the practical nature of each chapter, the book aims help neurologists select the most appropriate test, and interpret the results within the context of the genetic evaluation of hereditary neurological disorders. CONTENTS: Principles of genetics. Molecular Biology Techniques in Investigation and Diagnosis. Transgenic animals in Neurology. Gene Therapy of Neurological Diseases. Overview of Computer Software for Genetics. Internet Genetic Resources. Primer of Statistical Genetics. Strategies for the Genetic Molecular Study of a Neurological Disease. Neurogenetics from a Clinical point of view. Hereditary Muscular Dystrophies. Genetics of Movement Disorders. Genetics of Dementia. Genetics of Cerebrovascular Diseases. Genetic Bases of Neurological Disorders. Etc.

Viral Vectors for Treating Diseases of the Nervous System

Viral Vectors for Treating Diseases of the Nervous System Book
Author : David S. Latchman
Publisher : Gulf Professional Publishing
Release : 2003-09-05
ISBN : 9780123668561
Language : En, Es, Fr & De

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Book Description :

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

Anti Apoptotic Activity of the Herpes Simplex Virus Type 2 Gene ICP10 PK

Anti Apoptotic Activity of the Herpes Simplex Virus Type 2 Gene ICP10 PK Book
Author : Dana Perkins
Publisher : Createspace Independent Publishing Platform
Release : 2009-12-30
ISBN : 9781449991319
Language : En, Es, Fr & De

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Book Description :

PhD Thesis (2002). Apoptosis is an etiologic component of neurodegenerative disorders [e.g. Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS)] and acute brain injury ischemia/hypoxia and trauma). Previous studies have shown that a Herpes Simplex Virus Type 2 (HSV-2) gene, ICP10 PK, activates the Ras/MEK/ERK pathway in non-neuronal cells. Because this pathway was implicated in neuronal cell survival, the present studies tested whether ICP10 PK blocks apoptosis in various experimental paradigms of neuronal apoptosis. The broad anti-apoptotic activity of ICP10 PK suggests that it may be used in gene therapy of neurological disorders that involve apoptosis. In addition, the gene can be delivered non-invasively to the central nervous system using a replication-deficient HSV-2 mutant (ICP10deltaRR).

Advancing Gene Targeted Therapies for Central Nervous System Disorders

Advancing Gene Targeted Therapies for Central Nervous System Disorders Book
Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Publisher : National Academies Press
Release : 2019-10-31
ISBN : 0309495873
Language : En, Es, Fr & De

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Book Description :

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

Gene Therapy Book
Author : Anonim
Publisher : Unknown
Release : 1997
ISBN : 9789055491650
Language : En, Es, Fr & De

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Book Description :

This report is primarily concerned with somatic gene therapy, which can be defined as the introduction of specific alterations in the genetic material of human body cells for the purposes of medical treatment, diagnosis or disease prevention. The issue of germ-line gene therapy is also addressed by the Committee but only in general terms. Throughout the world, approximately 250 gene therapy clinical trials are being planned or are currently under way. About 150 of these trials are carried out in the United States and over 50 in Europe. More than 70% are focused on cancer, while just under 10% are AIDS related. In addition to these areas, gene therapy is also being tested in relation to a host of other disaeses, including hereditary disorders, cardiovascular diseases, neurological disorders and rheumatism. To date, no human disease has been cured by gene therapy. The (provisional) findings of a few studies are now available. These include both promising and disappointing results. Experience in this relatively new field of medicine is still extremely limited and gene therapy is presently at a stage that it cannot be excluded that genetic modification produces unpredictable negative effects. The public has to feel confident that measures have been taken to ensure the safety of experiments and that patients taking part in such experiments enjoy sufficient legal protection. The Committee recognizes the need for a separate review of the biosafety on one hand and the medical-ethical and legal aspects on the other.

Gene Transfer and Therapy for Neurological Disorders

Gene Transfer and Therapy for Neurological Disorders Book
Author : Anonim
Publisher : Unknown
Release : 1997
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Download Gene Transfer and Therapy for Neurological Disorders book written by , available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Controlled Genetic Manipulations

Controlled Genetic Manipulations Book
Author : Alexei Morozov
Publisher : Humana Press
Release : 2016-08-23
ISBN : 9781493961962
Language : En, Es, Fr & De

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Book Description :

The current demand for the development of techniques for controlled genetic manipulations is driven by the anatomical and physiological complexity of the brain and by the need for experimental models that can address this complexity through selective manipulation of defined components of the system: specific neuronal populations or selected synapses. In Controlled Genetic Manipulations, expert researchers present detailed chapters to supply basic technical information about controlled genetic manipulations and to provide examples of creative implementation of this methodology when addressing a unique biological problem. Some chapters of the book describe the most recent developments in the basic methodology, which includes use of Cre-recombinase, methods for delivery of genetic material into the brain and the use of optogenetics, whereas other chapters focus on applying these techniques to addressing particular biological questions like structural and functional mapping of neuronal circuits, analysis of specific synaptic connections, modeling or gene therapy of neurological disorders. As part of the Neuromethods series, chapters include key implementation advice that is crucial for getting optimal results. Authoritative and practical, Controlled Genetic Manipulations serves as a valuable guide to a variety of techniques, provided by many of the pioneers of the approaches.

Experimental Gene Therapy in Animal Models of Neurological Diseases

Experimental Gene Therapy in Animal Models of Neurological Diseases Book
Author : Graham Morrissey,University of Manchester. Medical School
Publisher : Unknown
Release : 2001
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Download Experimental Gene Therapy in Animal Models of Neurological Diseases book written by Graham Morrissey,University of Manchester. Medical School, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Proceedings of 9th International Conference and Exhibition on Advanced Cell and Gene Therapy 2019

Proceedings of 9th International Conference and Exhibition on Advanced Cell and Gene Therapy 2019 Book
Author : ConferenceSeries
Publisher : ConferenceSeries
Release : 2019-03-13
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

March 21-22, 2019 , Rome, Italy Key Topics : Cell Therapy,Gene Therapy,Stem Cell Therapies,Cell Culture and Bioprocessing,Viral Gene Therapy,Cell and Gene Therapy for Rare & Common Diseases,Tissue Science & Regenerative Medicine,Molecular Basis of Epigenetics,Bioengineering Therapeutics,Cell Science and Stem Cell Research,Clinical Trials on Cell & Gene Therapy,Nano Therapy,Genetic Engineering,Advanced Gene Therapeutics,Genetics & Genomic Medicine,Ethical Issues in Cell and Gene Therapy,Cell Therapy for Cardiovascular and Neurological Disorders,Regulatory and Safety Aspects of Cell and Gene Therapy,Markets & Future Prospects for Cell & Gene Therapy,Commercialization,