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A Guide to Human Gene Therapy

A Guide to Human Gene Therapy
  • Author : Roland W. Herzog,Sergei Zolotukhin
  • Publisher :Unknown
  • Release Date :2010
  • Total pages :388
  • ISBN : 9789814280914
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Summary : Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy - though still in its infancy - remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ( vector) to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession. This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system.Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

Gene Therapy of Cancer

Gene Therapy of Cancer
  • Author : Stanton L. Gerson,Edmund C. Lattime
  • Publisher :Unknown
  • Release Date :2002-04-04
  • Total pages :534
  • ISBN : 0080491367
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Summary : The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. Key Features * Provides in-depth description of targeted systems and treatment strategies * Explains the underlying cancer biology necessary for understanding a given therapeutic approach * Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses * Presents translational focus with emphasis on requirements for clinical implementation * Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference

Genetics and Gene Therapy

Genetics and Gene Therapy
  • Author : Sheila A.M. McLean
  • Publisher :Unknown
  • Release Date :2017-09-08
  • Total pages :568
  • ISBN : 9781351934275
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Summary : Genetics and Gene Therapy shows the wide range of the debate and the very real significance that genetics and its associated developments have for human beings, individually and collectively. Few areas of science and medicine have resulted in the volume of academic and popular literature as has genetics. The so-called revolution in understanding of the causes of disease states, and even behavioural traits, has focussed public attention on the influence of genes in making us what we are. Rapidly, however, the potential benefits of such understanding were overtaken, in the public mind at least, by the question of the possible (negative) implications of genetic knowledge and associated technologies. The chapters in this volume show just how wide-ranging concern has become, ranging from regulation to cloning, with the fear of discrimination in between. Part One begins with a range of general discussions of about the genetic enterprise itself, followed by consideration of some specific questions. Part Two then addresses cutting edge debates in genetics.

Gene Therapy

Gene Therapy
  • Author : Mauro Giacca
  • Publisher :Unknown
  • Release Date :2010-11-01
  • Total pages :303
  • ISBN : 8847016436
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Summary : I entered the gene therapy field in the mid-1990s, being fascinated by the immense potential of genes as drugs for the treatment of human disease. Since then, I have experienced the ups and downs of this discipline, and tried to contribute with my work and that of my laboratory to the development of innovative approaches to the treatment of cardiovascular disorders. During these years, I have had several opp- tunities to speak on gene therapy at lectures and academic lessons, and have often noticed that the field is very attractive to scientists of all disciplines. However, as yet no comprehensive book on the subject has been published. Indeed, most books in the field are either a collection of gene transfer laboratory protocols or deal with the subject in a rather superficial manner. Hence the idea to write a gene therapy textbook that is broad and comprehensive, but at the same time provides sufficient molecular and clinical detail to be of interest to students, professors, and specialists in the various disciplines that contribute to gene therapy. I have tried to keep the language plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, whenever technical descriptions are required, they are provided.

Gene Therapy for Viral Infections

Gene Therapy for Viral Infections
  • Author : Patrick Arbuthnot
  • Publisher :Unknown
  • Release Date :2015-06-01
  • Total pages :392
  • ISBN : 9780124114524
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Summary : Gene Therapy for Viral Infections provides a comprehensive review of the broader field of nucleic acid and its use in treating viral infections. The text bridges the gap between basic science and important clinical applications of the technology, providing a systematic, integrated review of the advances in nucleic acid-based antiviral drugs and the potential advantages of new technologies over current treatment options. Coverage begins with the fundamentals, exploring varying topics, including harnessing RNAi to silence viral gene expression, antiviral gene editing, viral gene therapy vectors, and non-viral vectors. Subsequent sections include detailed coverage of the developing use of gene therapy for the treatment of specific infections, the principles of rational design of antivirals, and the hurdles that currently face the further advancement of gene therapy technology. Provides coverage of gene therapy for a variety of infections, including HBV, HCV, HIV, hemorrhagic fever viruses, and respiratory and other viral infections Bridges the gap between the basic science and the important medical applications of this technology Features a broad approach to the topic, including an essential overview and the applications of gene therapy, synthetic RNA, and other antiviral strategies that involve nucleic acid engineering Presents perspectives on the future use of nucleic acids as a novel class of antiviral drugs Arms the reader with the cutting-edge information needed to stay abreast of this developing field

Gene Therapy

Gene Therapy
  • Author : David Cooper,Prof Nick Lemoine
  • Publisher :Unknown
  • Release Date :2020-09-11
  • Total pages :360
  • ISBN : 9781000144680
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Summary : Gene Therapy describes the delivery systems now available to target a given tissue with specific gene or oligonucleotide sequences, and explores the utility of animal modules as test systems. In the context of selected disease states, it summarises in vitro and in vivo studies and clinical trials performed to date.

Somatic Gene Therapy

Somatic Gene Therapy
  • Author : P.L. Chang
  • Publisher :Unknown
  • Release Date :2018-01-17
  • Total pages :313
  • ISBN : 9781351085212
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Summary : As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.

Muscle Gene Therapy

Muscle Gene Therapy
  • Author : Dongsheng Duan
  • Publisher :Unknown
  • Release Date :2009-11-26
  • Total pages :275
  • ISBN : 144191207X
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Summary : 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.

Translating Gene Therapy to the Clinic

Translating Gene Therapy to the Clinic
  • Author : Jeffrey Laurence,Michael Franklin
  • Publisher :Unknown
  • Release Date :2014-11-14
  • Total pages :346
  • ISBN : 9780128005644
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Summary : Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials

Gene Therapy

Gene Therapy
  • Author : United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions. Subcommittee on Public Health
  • Publisher :Unknown
  • Release Date :2000
  • Total pages :126
  • ISBN : PSU:000046314529
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Summary :

Gene Therapy

Gene Therapy
  • Author : United States,United States. Congress. House. Committee on Science, Space, and Technology
  • Publisher :Unknown
  • Release Date :1995
  • Total pages :160
  • ISBN : UCR:31210014030975
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Summary :

Gene Therapy

Gene Therapy
  • Author : Rex A. Hudson,United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions. Subcommittee on Public Health
  • Publisher :Unknown
  • Release Date :2000
  • Total pages :75
  • ISBN : PSU:000047030688
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Human Gene Therapy

Human Gene Therapy
  • Author : United States. Congress. Office of Technology Assessment
  • Publisher :Unknown
  • Release Date :1984
  • Total pages :105
  • ISBN : UCR:31210024829408
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Summary :

Adenoviral Vectors for Gene Therapy

Adenoviral Vectors for Gene Therapy
  • Author : David T. Curiel
  • Publisher :Unknown
  • Release Date :2016-03-10
  • Total pages :868
  • ISBN : 9780128005101
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Summary : Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Human Gene Therapy

Human Gene Therapy
  • Author : Anonim
  • Publisher :Unknown
  • Release Date :1987
  • Total pages :229
  • ISBN : UOM:39015045603795
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Summary :

Fast Facts: Gene Therapy

Fast Facts: Gene Therapy
  • Author : R. Herzog,L. Popplewell
  • Publisher :Unknown
  • Release Date :2020-04-24
  • Total pages :104
  • ISBN : 9783318066678
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Summary : Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments

Gene Therapy in Neurological Disorders

Gene Therapy in Neurological Disorders
  • Author : Mingjie Li,B. Joy Snider
  • Publisher :Unknown
  • Release Date :2018-05-25
  • Total pages :450
  • ISBN : 9780128098219
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Summary : 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

Adeno-associated Viral Vectors for Gene Therapy

Adeno-associated Viral Vectors for Gene Therapy
  • Author : Kenneth I. Berns,Terence Robin Flotte
  • Publisher :Unknown
  • Release Date :2005
  • Total pages :283
  • ISBN : OCLC:1131955937
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Summary :

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

Gene Therapy of the Central Nervous System: From Bench to Bedside
  • Author : Michael G. Kaplitt,Matthew During
  • Publisher :Unknown
  • Release Date :2006
  • Total pages :355
  • ISBN : 0123976324
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Summary : 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 and Gene Delivery Systems

Gene Therapy and Gene Delivery Systems
  • Author : Nedim E. Altaras
  • Publisher :Unknown
  • Release Date :2005-12-20
  • Total pages :285
  • ISBN : 3540284044
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Summary : 1 D.V. Schaffer, W. Zhou: Gene Therapy as Future Human Therapeutics.- 2 J. Heidel, S. Mishra, M.E. Davis: Molecular Conjugates.- 3 M. Manthorpe, P. Hobart, G. Hermanson, M. Ferrari, A. Geall, B. Goff, A. Rolland: Plasmid Vaccines and Therapeutics: From Design to Applications.- 4 S.R. Little, R. Langer: Non-Viral Delivery of Cancer Genetic Vaccines.- 5 J.C. Grieger, R.J. Samulski: Adeno-Associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications.- 6 J.H. Yu, D.V. Schaffer: Advanced Targeting Strategies for Murine Retroviral and Adeno-Associated Viral Vectors.- 7 N. Loewen, E.M. Poeschla: Lentiviral Vectors.- 8 N.E. Altaras, J.G. Aunins, R.K. Evans, A. Kamen, J.O. Konz, J.J. Wolf: Production and Formulation of Adenovirus Vectors.-

Gene Therapy

Gene Therapy
  • Author : Evelyn B. Kelly
  • Publisher :Unknown
  • Release Date :2007
  • Total pages :198
  • ISBN : 0313337608
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Summary : Presents an introduction to gene therapy, discussing its scientific background, the types of disorders which it can potentially treat, the ethical, social, and religious issues surrounding its use, and the role of government regulation.