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A Guide to Human Gene Therapy

A Guide to Human Gene Therapy Book
Author : Roland W. Herzog,Sergei Zolotukhin
Publisher : World Scientific
Release : 2010
ISBN : 9814280917
Language : En, Es, Fr & De

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Book Description :

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.

A Handbook of Gene and Cell Therapy

A Handbook of Gene and Cell Therapy Book
Author : Clévio Nóbrega,Liliana Mendonça,Carlos A. Matos
Publisher : Springer Nature
Release : 2020-06-27
ISBN : 3030413330
Language : En, Es, Fr & De

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Book Description :

This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field.

Gene Therapy for Cancer

Gene Therapy for Cancer Book
Author : Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Publisher : Springer Science & Business Media
Release : 2007-10-26
ISBN : 159745222X
Language : En, Es, Fr & De

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Book Description :

The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.

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

Gene Therapy Book
Author : David Cooper,Prof Nick Lemoine
Publisher : Garland Science
Release : 2020-09-11
ISBN : 1000144682
Language : En, Es, Fr & De

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Book Description :

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.

Adenoviral Vectors for Gene Therapy

Adenoviral Vectors for Gene Therapy Book
Author : David T. Curiel
Publisher : Academic Press
Release : 2016-03-10
ISBN : 0128005106
Language : En, Es, Fr & De

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Book Description :

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

Translating Gene Therapy to the Clinic

Translating Gene Therapy to the Clinic Book
Author : Jeffrey Laurence,Michael Franklin
Publisher : Academic Press
Release : 2014-11-14
ISBN : 0128005645
Language : En, Es, Fr & De

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Book Description :

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 Book
Author : Mauro Giacca
Publisher : Springer Science & Business Media
Release : 2010-11-01
ISBN : 8847016436
Language : En, Es, Fr & De

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Book Description :

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

Gene Therapy Book
Author : Surendra Nimesh
Publisher : Woodhead Pub Limited
Release : 2013
ISBN : 9781907568404
Language : En, Es, Fr & De

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Book Description :

Gene therapy is emerging as a new class of therapeutics for the treatment of inherited and acquired diseases. However, poor cellular uptake and instability of DNA in the physiological milieu limits its therapeutic potential, hence a vector which can protect and efficiently transport DNA to the target cells must be developed. Nanotechnology-based non-viral vectors have been proposed as potential candidates. Various polymeric nanoparticles have been shown to be suitable, with high cellular uptake efficiencies and reduced cytotoxicity. These delivery vectors form condensed complexes with DNA which result in shielding against enzymatic degradation and enhanced cellular targeting. Advantages including easy manipulatibility, high stability, low cost and high payload, mean that nanoparticles from various polymers have been exploited. Gene therapy gives a systematic account of the many aspects of nanotechnology mediated gene therapy, from the preparation of nanoparticles to physicochemical characterization, and follows with applications in in vitro and in vivo models. This book emphasizes the various aspects of nanotechnology-based gene therapy, with initial chapters detailing the tools and techniques available for preparation and in vitro and in vivo characterization of nanoparticles. Later chapters provide exhaustive details on polymeric systems employed for gene therapy. Provides an overview of nanotechnology applications in gene therapy, from preparation of nanoparticles to in vitro and in vivo studies Details the tools and techniques available for preparation, characterization and in vitro and in vivo study of nanoparticles Details the limitations of nanoparticle-mediated gene therapy and proposes ways in which they may be overcome

Human Gene Therapy

Human Gene Therapy Book
Author : Eve K. Nichols
Publisher : Harvard University Press
Release : 1988
ISBN : 9780674414808
Language : En, Es, Fr & De

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Book Description :

Introduces the concept of gene therapy as a new medical treatment, explains how genetic diseases are now diagnosed and treated, and discusses the issues surrounding human genetic manipulation

Viral Vectors for Gene Therapy

Viral Vectors for Gene Therapy Book
Author : Fredric P. Manfredsson,Matthew J. Benskey
Publisher : Unknown
Release : 2019
ISBN : 9781493990658
Language : En, Es, Fr & De

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Book Description :

This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. 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. Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.

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.

Stem Cell and Gene Therapy for Cardiovascular Disease

Stem Cell and Gene Therapy for Cardiovascular Disease Book
Author : Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson
Publisher : Academic Press
Release : 2015-08-21
ISBN : 0128018631
Language : En, Es, Fr & De

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Book Description :

Stem Cell and Gene Therapy for Cardiovascular Disease is a state-of-the-art reference that combines, in one place, the breadth and depth of information available on the topic. As stem cell and gene therapies are the most cutting-edge therapies currently available for patients with heart failure, each section of the book provides information on medical trials from contributors and specialists from around the world, including not only what has been completed, but also what is planned for future research and trials. Cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals will find this book an invaluable resource for further study on the topic. Provides information on stem and gene therapy medical trials from contributors and specialists around the world, including not only what has been completed, but also what is planned for future research and trials Presents topics that can be applied to allogeneic cells, mesenchymal cells, gene therapy, cardiomyoctyes, iPS cells, MAPC's, and organogenesis Covers the three areas with the greatest clinical trials to date: chronic limb ischemia, chronic angina, and acute MI Covers the prevailing opinions on how to harness the body’s natural repair mechanisms Ideal resource for cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals

Polymers and Nanomaterials for Gene Therapy

Polymers and Nanomaterials for Gene Therapy Book
Author : Ravin Narain
Publisher : Woodhead Publishing
Release : 2016-01-09
ISBN : 0081005210
Language : En, Es, Fr & De

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Book Description :

Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. Explores current challenges in the research of genetic diseases Discusses polymers for gene therapy and their function in designing advanced materials Provides examples of organic and inorganic nanomaterials for gene therapy Includes labeling, targeting, and assays Looks at characterization, physico-(bio)chemical properties, and applications

Gene Therapy for Viral Infections

Gene Therapy for Viral Infections Book
Author : Patrick Arbuthnot
Publisher : Academic Press
Release : 2015-06-01
ISBN : 0124114520
Language : En, Es, Fr & De

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Book Description :

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

Fast Facts Gene Therapy

Fast Facts  Gene Therapy Book
Author : R. Herzog,L. Popplewell
Publisher : Karger Medical and Scientific Publishers
Release : 2020-04-24
ISBN : 3318066672
Language : En, Es, Fr & De

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Book Description :

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

Emerging Trends in Cell and Gene Therapy

Emerging Trends in Cell and Gene Therapy Book
Author : Michael K. Danquah,Ram I. Mahato
Publisher : Springer Science & Business Media
Release : 2013-06-14
ISBN : 162703417X
Language : En, Es, Fr & De

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Book Description :

Examples from various organs and diseases illustrate the potential benefit obtained when both therapeutic approaches are combined with delivery strategies. Representing the combined effort of several leading international research and clinical experts, this book, Emerging Trends in Cell and Gene Therapy, provides a complete account on and brings into sharp focus current trends and state-of-the-art in important areas at the interface of cell- and gene-based therapies. This book addresses the current fragmented understanding regarding these two research areas and fills the vast unmet educational need and interest of both students and researchers in academia and industry. Main features of the book: · Biological aspects of stem cell sources, differentiation and engineering. · Application of microfluidics to study stem cell dynamics · Potential clinical application of stem cells and gene therapy to specific human disease. · Utilization of biomaterials and stem cells in regenerative medicine with particular emphasis on spinal cord repair, ligament and bone tissue engineering. · Biomimetic multiscale topography for cell alignment.

Gene Therapy of Cancer

Gene Therapy of Cancer Book
Author : Stanton L. Gerson
Publisher : Unknown
Release : 2002
ISBN : 9780124375512
Language : En, Es, Fr & De

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Book Description :

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

Gene Therapy

Gene Therapy Book
Author : Nancy Smyth Templeton,D. D. Lasic
Publisher : CRC Press
Release : 2000-06-15
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

This text covers all aspects of gene therapy, including basic principles, viral and nonviral delivery systems, targeted diseases, regulatory issues, and FDA requirements. It investigates genetic bullets to block HIV-1 replication and genetic guns to deliver antiviral agents.

Human Gene Therapy

Human Gene Therapy Book
Author : Anonim
Publisher : Unknown
Release : 1996
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

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