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Author: Michael G. Kaplitt
Publisher: Gulf Professional Publishing
ISBN: 9780123976321
Category : Medical
Languages : en
Pages : 380
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Book Description
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.
Author: Michael G. Kaplitt
Publisher: Gulf Professional Publishing
ISBN: 9780123976321
Category : Medical
Languages : en
Pages : 380
Get Book Here
Book Description
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.
Author: Dongsheng Duan
Publisher: Springer Science & Business Media
ISBN: 144191207X
Category : Medical
Languages : en
Pages : 281
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Book Description
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.
Author: Roland W. Herzog
Publisher: World Scientific
ISBN: 9814280917
Category : Medical
Languages : en
Pages : 415
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Book Description
1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne
Author: Matthew E. Gladden
Publisher: Synthypnion Academic
ISBN: 194437308X
Category : Business & Economics
Languages : en
Pages : 314
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Book Description
This volume serves a resource for the design and analysis of neuroprosthetic supersystems, which can be defined as organizations – either small or large, simple or complex – whose human members have been neuroprosthetically augmented. While numerous other texts focus on the biomedical engineering of neuroprostheses as technological devices or on the biocybernetic engineering of the host-device system comprising a neuroprosthesis and its human host, this volume presents a unique investigation of the intentional creation of higher-order supersystems that allow multiple neuroprosthetically augmented human beings to interact with one another and with external information systems in order to accomplish some shared task. In essence, this can be understood as the work of designing and managing neuroprosthetically enhanced organizations. Individual chapters present an ontology of the neuroprosthesis as a computing device; a biocybernetic ontology of the host-device system; an ontology of the neuroprosthesis as an instrument of ‘cyborgization’; motivating and inhibiting factors for the organizational deployment of posthumanizing neuroprostheses by military organizations and other early adopters; an introduction to enterprise architecture in the context of technological posthumanization; an exploration of the implications of neuroprosthetic augmentation for enterprise architecture; and considerations for the development of effective network topologies for neuroprosthetically augmented organizations. The conceptual frameworks formulated within this book offer a wide range of tools that can be of use to policymakers, ethicists, neuroprosthetic device manufacturers, organizational decision-makers, and others who must analyze or manage the complex legal, ethical, and managerial implications that result from the use of emerging neuroprosthetic technologies within an organizational context.
Author: Jeffrey Noebels
Publisher: OUP USA
ISBN: 0199746540
Category : Medical
Languages : en
Pages : 1258
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Book Description
Jasper's Basic Mechanisms, Fourth Edition, is the newest most ambitious and now clinically relevant publishing project to build on the four-decade legacy of the Jasper's series. In keeping with the original goal of searching for "a better understanding of the epilepsies and rational methods of prevention and treatment.", the book represents an encyclopedic compendium neurobiological mechanisms of seizures, epileptogenesis, epilepsy genetics and comordid conditions. Of practical importance to the clinician, and new to this edition are disease mechanisms of genetic epilepsies and therapeutic approaches, ranging from novel antiepileptic drug targets to cell and gene therapies.
Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 030929665X
Category : Medical
Languages : en
Pages : 78
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Book Description
Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.
Author: Reinhard Merkel
Publisher: Springer Science & Business Media
ISBN: 3540464778
Category : Medical
Languages : en
Pages : 547
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Book Description
The wealth of insights into the brain’s functioning gained by neuroscience in recent years led to the development of new possibilities for intervening in the brain such as neurotransplantation, neural prostheses and brain stimulation techniques. Moreover, new and safer classes of psychopharmaceutical drugs lend themselves to neuroenhancement applications, i.e. they could be used to enhance cognitive capacities or emotional well-being without therapeutic need. This book offers extensive state-of-the-art accounts for these novel kinds of intervention, indicates future developments, and discusses the relevant philosophical, ethical and legal issues.
Author: Viral Patel
Publisher: Elsevier
ISBN: 0443238405
Category : Medical
Languages : en
Pages : 630
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Book Description
Targeted Therapy for the Central Nervous System: Formulation, Clinical Challenges, and Regulatory Strategies presents research on various delivery methods of drugs to the central nervous system and brain. This volume examines targeted therapies for neurodegenerative disorders and succinctly outlines the future of drug delivery systems, highlighting significant advancements specifically relating to central nervous system delivery. This book will be of great interest to researchers working in the field of neuroscience and pharmacology as well as clinicians (pharmacists, radiologists, psychiatrists). - Provides a current, thorough means on how drugs are delivered to the neurological system - Figures a connection amongst the physiology of drug delivery pertaining to the central nervous system, fundamentals of drug delivery, and distribution principles - Gives an accounting of clinical trials and regulatory approaches for the formulations targeting brain
Author: Gerald J. Prud'homme
Publisher: Springer
ISBN: 9780306479915
Category : Medical
Languages : en
Pages : 149
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Book Description
Autoimmune diseases are diverse and responsible for considerable morbidity. Their etiology remains largely unknown, and current therapy with anti-inflammatory drugs is prone to adverse effects, and rarely curative. New therapies with anti-cytokine antibodies or receptors are promising, but require frequent administration of expensive protein drugs. Gene Therapy of Autoimmune Diseases comprehensively reviews research in gene therapy for autoimmune diseases with viral or non-viral vectors. Gene therapy offers the possibility of long-term, continuous delivery of a wide variety of immunosuppressive, anti-inflammatory, or tolerance-inducing agents. Moreover, highly specific genetically modified cells can be produced. This book discusses the most promising avenues in this exciting new field.
Author: Matthew E. Gladden
Publisher: Defragmenter Media
ISBN: 1944373063
Category : Business & Economics
Languages : en
Pages : 444
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Book Description
What are the best practices for leading a workforce in which human employees have merged cognitively and physically with electronic information systems and work alongside social robots, artificial life-forms, and self-aware networks that are ‘colleagues’ rather than simply ‘tools’? How does one manage organizational structures and activities that span actual and virtual worlds? How are the forces of technological posthumanization transforming the theory and practice of management? This volume explores the reality that an organization’s workers, managers, customers, and other stakeholders increasingly comprise a complex network of human agents, artificial agents, and hybrid human-synthetic entities. The first part of the book develops the theoretical foundations of an emerging ‘organizational posthumanism’ and presents frameworks for understanding and managing the evolving workplace relationship between human and synthetic beings. Other chapters investigate topics such as the likelihood that social robots might utilize charismatic authority to lead human workers; potential roles of AIs as managers of cross-cultural virtual teams; the ethics and legality of entrusting organizational decision-making to spatially diffuse robots that have no discernible physical form; quantitative approaches to comparing managerial capabilities of human and artificial agents; the creation of artificial life-forms that function as autonomous enterprises competing against human businesses; neural implants as gateways that allow human users to participate in new forms of organizational life; and the implications of advanced neuroprosthetics for information security and business model design. As the first comprehensive application of posthumanist methodologies to management, this volume will interest management scholars and management practitioners who must understand and guide the forces of technologization that are rapidly reshaping organizations’ form, dynamics, and societal roles.
