Delivery Of Gene Therapies Into The Central Nervous System

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Gene Therapy of the Central Nervous System: From Bench to Bedside

Author : Michael G. Kaplitt
Publisher : Gulf Professional Publishing
ISBN 13 : 9780123976321
Total Pages : 380 pages
Book Rating : 4.9/5 (763 download)

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Book Synopsis Gene Therapy of the Central Nervous System: From Bench to Bedside by : Michael G. Kaplitt

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.

Gene Therapy for the Central and Peripheral Nervous System

Author : Andrew P. Tosolini
Publisher : Frontiers Media SA
ISBN 13 : 2889454754
Total Pages : 220 pages
Book Rating : 4.8/5 (894 download)

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Book Synopsis Gene Therapy for the Central and Peripheral Nervous System by : Andrew P. Tosolini

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.

Gene Therapy in the CNS – Progress and Prospects for Novel Therapies

Author : Casper René Gøtzsche
Publisher : Frontiers Media SA
ISBN 13 : 2889718522
Total Pages : 188 pages
Book Rating : 4.8/5 (897 download)

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Book Synopsis Gene Therapy in the CNS – Progress and Prospects for Novel Therapies by : Casper René Gøtzsche

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:

Delivery of Gene Therapies Into the Central Nervous System

Author : Peter Deng
Publisher :
ISBN 13 :
Total Pages : 0 pages
Book Rating : 4.5/5 (825 download)

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Book Synopsis Delivery of Gene Therapies Into the Central Nervous System by : Peter Deng

Download or read book Delivery of Gene Therapies Into the Central Nervous System written by Peter Deng and published by . This book was released on 2020 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Therapies based on zinc finger, transcription activator-like effectors, and CRISPR/Cas9 to act as artificial transcription factors to regulate gene expression are becoming viable strategies to treat genetically linked disorders. Effective delivery methodologies for artificial transcription factors remain a major barrier in targeting the central nervous system. Chapter 1 of this dissertation broadly reviews the use of adeno-associated virus, lipid nanoparticles, and stem cell-based systems in the gene therapy field. Chapter 2 of this dissertation discusses the development of a transcription activator-like effector for the silencing of the mutant Huntingtin gene in Huntington's Disease. It further evaluates the strengths and limitations of current in-vivo artificial transcription factor delivery strategies such as adeno-associated virus and lipid nanoparticles in preclinical models of Huntington's Disease. Chapter 3 of this dissertation describes the first reported use of a mesenchymal stem/stromal cell-based delivery system for the secretion of a zinc finger protein in transgenic Angelman Syndrome mouse models and young rhesus monkeys. These secreted zinc finger proteins widely distribute in the murine brain and partially restore motor deficits in Angelman syndrome mouse models. Chapter 4 this dissertation discusses the identification and development of lead zinc finger proteins to disrupt UBE3A-ATS expression and activate paternal UBE3A expression in Angelman Syndrome patient cell models. Finally, Chapter 5 summarizes this dissertation work as a whole and speculates on future directions of the work described.

Gene Delivery Into the Central Nervous System (CNS) Using AAV Vectors

Author : Kōichi Miyake
Publisher :
ISBN 13 :
Total Pages : pages
Book Rating : 4.:/5 (115 download)

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Book Synopsis Gene Delivery Into the Central Nervous System (CNS) Using AAV Vectors by : Kōichi Miyake

Download or read book Gene Delivery Into the Central Nervous System (CNS) Using AAV Vectors written by Kōichi Miyake and published by . This book was released on 2015 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: Application of gene therapies is a promising approach to the treatment of various neurological disorders, including Parkinson's disease, amyotrophic lateral sclerosis (ALS), and lysosomal storage disorders, which are not treatable by any other means. However, the blood-brain barrier (BBB) is a key obstacle to gene delivery to the central nervous system (CNS). Adeno-associated virus (AAV) vectors have emerged as a promising tool for gene delivery to the CNS, thanks to their safety and ability to transduce non-dividing neuronal cells. In this chapter, we discuss strategies for delivering genes across the BBB, focusing especially on potential routes of administration of AAV vectors and promising applications of AAV vectors to the treatment of CNS disorders.

Drug and Gene Delivery to the Central Nervous System for Neuroprotection

Author : Hari S. Sharma
Publisher : Springer
ISBN 13 : 3319576968
Total Pages : 237 pages
Book Rating : 4.3/5 (195 download)

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Book Synopsis Drug and Gene Delivery to the Central Nervous System for Neuroprotection by : Hari S. Sharma

Download or read book Drug and Gene Delivery to the Central Nervous System for Neuroprotection written by Hari S. Sharma and published by Springer. This book was released on 2017-06-05 with total page 237 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309495849
Total Pages : 93 pages
Book Rating : 4.3/5 (94 download)

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Book Synopsis Advancing Gene-Targeted Therapies for Central Nervous System Disorders by : National Academies of Sciences, Engineering, and Medicine

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.

Genetic Manipulation of the Nervous System

Author :
Publisher : Elsevier
ISBN 13 : 0080532403
Total Pages : 297 pages
Book Rating : 4.0/5 (85 download)

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Book Synopsis Genetic Manipulation of the Nervous System by :

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 297 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.

Gene Therapy for Neurological Disorders and Brain Tumors

Author : E. Antonio Chiocca
Publisher : Springer Science & Business Media
ISBN 13 : 1592594786
Total Pages : 458 pages
Book Rating : 4.5/5 (925 download)

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Book Synopsis Gene Therapy for Neurological Disorders and Brain Tumors by : E. Antonio Chiocca

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.

Human Gene Therapy: Current Opportunities and Future Trends

Author : G. M. Rubanyi
Publisher : Springer Science & Business Media
ISBN 13 : 3662053527
Total Pages : 276 pages
Book Rating : 4.6/5 (62 download)

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Book Synopsis Human Gene Therapy: Current Opportunities and Future Trends by : G. M. Rubanyi

Download or read book Human Gene Therapy: Current Opportunities and Future Trends written by G. M. Rubanyi and published by Springer Science & Business Media. This book was released on 2013-04-17 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: Advances in genetics, molecular biology and gene delivery technologies in recent years have led to new gene therapy strategies for treatment of a variety of diseases. This book gives a comprehensive overview of the present status and future directions of gene delivery systems and therapeutic strategies for the clinical application of gene therapy in cancer, cardiovascular and central nervous system diseases. Stem cell-based therapies and gene expression regulatory systems as novel platform technologies for various gene therapy applications are also discussed. Leading experts give excellent overviews of basic molecular aspects and clinical applications in this new emerging biomedical field.

Gene Transfer and Therapy in the Nervous System

Author : Fred H. Gage
Publisher : Springer Science & Business Media
ISBN 13 : 3642848427
Total Pages : 226 pages
Book Rating : 4.6/5 (428 download)

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Book Synopsis Gene Transfer and Therapy in the Nervous System by : Fred H. Gage

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.

Pediatric Neurology Part III

Author : Patrick Aubourg
Publisher : Elsevier Inc. Chapters
ISBN 13 : 012808457X
Total Pages : 23 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis Pediatric Neurology Part III by : Patrick Aubourg

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 23 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.

Muscle Gene Therapy

Author : Dongsheng Duan
Publisher : Springer Science & Business Media
ISBN 13 : 144191207X
Total Pages : 281 pages
Book Rating : 4.4/5 (419 download)

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Book Synopsis Muscle Gene Therapy by : Dongsheng Duan

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.

Protocols for Gene Transfer in Neuroscience

Author : P. R. Lowenstein
Publisher :
ISBN 13 :
Total Pages : 468 pages
Book Rating : 4.3/5 ( download)

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Book Synopsis Protocols for Gene Transfer in Neuroscience by : P. R. Lowenstein

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.

Drug Delivery to the Central Nervous System

Author : Kewal K. Jain
Publisher : Humana Press
ISBN 13 : 9781607615286
Total Pages : 0 pages
Book Rating : 4.6/5 (152 download)

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Book Synopsis Drug Delivery to the Central Nervous System by : Kewal K. Jain

Download or read book Drug Delivery to the Central Nervous System written by Kewal K. Jain and published by Humana Press. This book was released on 2009-12-17 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Playing an important role in the treatment of neurological disorders, the delivery of drugs to central nervous system (CNS), both administered directly and administered systematically for targeted action, encounters a major challenge in the form of the blood-brain barrier (BBB), which limits the access of drugs to the brain substance. In Drug Delivery to the Central Nervous System, experts in the field present essential methods used to deliver therapeutics across the BBB, both in experimental animals and in humans. In addition to those methods, several overviews of innovative methods and their applications are presented in order to give a glimpse of the future of this research. As a volume in the successful Neuromethods series, this book presents its protocols with the kind of detailed description and implementation advice that is crucial for getting optimal results. Authoritative and cutting-edge, Drug Delivery to the Central Nervous System serves as an ideal guide to scientists continuing to pursue knowledge of the delicate interactions between pharmaceuticals and the brain.

Gene Therapy - Principles and Challenges

Author :
Publisher :
ISBN 13 : 9789535172680
Total Pages : pages
Book Rating : 4.1/5 (726 download)

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Book Synopsis Gene Therapy - Principles and Challenges by :

Download or read book Gene Therapy - Principles and Challenges written by and published by . This book was released on 2015 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Gene Therapy in Neurological Disorders

Author : Mingjie Li
Publisher : Academic Press
ISBN 13 : 012809821X
Total Pages : 450 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis Gene Therapy in Neurological Disorders by : Mingjie Li

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