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Gene Therapy of Autoimmune Disease

Author : Gerald J. Prud'homme
Publisher : Springer Science & Business Media
Page : 148 pages
File Size : 39,51 MB
Release : 2007-02-26
Category : Medical
ISBN : 0387286705

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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.

Lentiviral Vectors and Gene Therapy

Author : David Escors
Publisher : Springer Science & Business Media
Page : 110 pages
File Size : 42,78 MB
Release : 2012-03-23
Category : Medical
ISBN : 3034804016

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Gene therapy was conceived during the early and mid part of the 20th century. At first, it was considered a revolutionary biomedical procedure, which could potentially cure any disease for which the molecular bases were understood. Since then, gene therapy has gone through many stages and has evolved from a nearly unrealistic perspective to a real life application. Clinical efficacy in humans was demonstrated at the beginning of this century after its successful application in small-scale clinical trials to cure severe immunodeficiency in children. However, their successes were overshadowed some time later by the occurrence of vector-related leukaemia in a number of treated children. It is in this context that lentiviral vectors have appeared, with improved efficiency and, possibly, increased biosafety. Very recently, the first clinical trials with lentivectors have been carried out with some success. This Brief firstly defines gene therapy, and places lentivectors within this fascinating therapeutic strategy. Then follows a comprehensive description of the development of retroviral and lentiviral vectors and how to specifically target distinct cell types and tissues. The authors also discuss the application of lentivector gene therapy for the treatment of cancer and autoimmune diseases, ending with the application of lentivectors in human gene therapy clinical trials.

Biologic and Gene Therapy of Autoimmune Disease

Author : C. Garrison Fathman
Publisher : Karger Medical and Scientific Publishers
Page : 229 pages
File Size : 22,52 MB
Release : 2000-01-01
Category : Medical
ISBN : 3805569491

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The clinical management of autoimmune diseases has proven to be extremely difficult. Current therapies focus on trying to alleviate symptoms, but fail to correct the fundamental immune defects that lead to pathology. To achieve this goal, it is necessary to understand much of the biology of antigen presentation, lymphocyte activation and the effects of cytokines. The articles in this book provide an up-to-date review of current innovative therapies using both biologic and gene therapy for the treatment of selected autoimmune diseases. Therapeutical approaches discussed include oral tolerance, the use of anti-CD4 monoclonal antibodies, IL-10 and anti-TNFa antibodies, DNA vaccination, and gene therapy applied to organ-specific autoimmune disease. Although some of these techniques are still in their infancy, their potential efficacy has been demonstrated in several animal models of autoimmune disease, holding great promise for the future development of treatments. Written by recognized experts in the field, the chapters in this book illustrate the concept of technology transfer from bench to bedside and provide a valuable update for clinicians and scientists in clinical immunology.

Gene Therapy in Inflammatory Diseases

Author : Christopher H. Evans
Publisher : Birkhäuser
Page : 278 pages
File Size : 16,75 MB
Release : 2012-12-06
Category : Medical
ISBN : 3034884788

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Gene therapy for inflammatory diseases is a new , burgeoning field of medicine. Edited by the undisputed pioneers of this area of research, this volume is the first devoted to its topic. It contains thirteen chapters, each written by leaders in their respective fields, that summarize the state of the art in developing novel, gene based treatments for inflammatory diseases. As well as providing an introduction to the basic concepts of gene therapy and the use of naked DNA approaches, the book describes the advances that have been made in applying them to arthritis, lupus, multiple sclerosis, diabetes, Sjogren`s syndrome and transplantation.One chapter is devoted to discussing the first human clinical trials that apply gene therapy to the treatment of an inflammatory disease. As well as providing novel therapeutic approaches, gene therapy facilitates the development of new and improved animal models of disease; a chapter describing these advances is also included. As an up-to-date, timely book written by th

Immunopharmacology

Author : Manzoor M. Khan
Publisher : Springer Science & Business Media
Page : 275 pages
File Size : 37,38 MB
Release : 2008-12-19
Category : Medical
ISBN : 0387779760

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During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.

Gene Therapy for HIV

Author : Gerhard Bauer
Publisher : Springer Science & Business Media
Page : 77 pages
File Size : 30,7 MB
Release : 2014-02-07
Category : Medical
ISBN : 1493904345

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This Brief describes the concept and realization of gene therapy for HIV from the unique historic perspective and insight of two pioneers of the clinical applications of stem cell gene therapy for HIV. Gerhard Bauer applied ribozyme-anti-HIV and other vectors to manufacture clinical grade, HIV-resistant hematopoietic stem cells for the first patients that received stem cell gene therapy for HIV, including the first child in the world and the first fully marrow-ablated HIV infected patient. Joseph Anderson developed the most recent and most potent combination anti-HIV lentiviral vectors and pluripotent stem cell applications for HIV gene therapy and tested these in the appropriate in vitro and vivo models, paving the way for novel HIV gene therapy approaches to possibly cure patients. In Gene Therapy for HIV, Bauer and Anderson discuss the unique aspects of this therapy, including its limitations and proper safety precautions and outline a path for a possible functional cure for HIV using stem cell gene therapy based on a cure already achieved with a bone marrow stem cell transplantation performed in Germany using donor stem cells with a naturally arising CCR5 mutation. In addition, the Brief provides a thorough and methodical explanation of the basics of gene therapy, gene therapy vector development, in vitro and in vivo models for HIV gene therapy and clinical applications of HIV gene therapy, including Good Manufacturing Practices.

Gene Therapy of Autoimmune Disease

Author : Gerald J. Prud'homme
Publisher : Springer
Page : 149 pages
File Size : 39,87 MB
Release : 2005-07-13
Category : Medical
ISBN : 9780306479915

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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.

A Guide to Human Gene Therapy

Author : Roland W. Herzog
Publisher : World Scientific
Page : 415 pages
File Size : 40,15 MB
Release : 2010
Category : Medical
ISBN : 9814280917

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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

Viruses in Human Gene Therapy

Author : J. Vos
Publisher : Springer Science & Business Media
Page : 228 pages
File Size : 32,39 MB
Release : 2012-12-06
Category : Science
ISBN : 9401105553

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W. French Anderson, M.D. The publication of this book comes at an opportune time for the young field of human gene therapy. After a decade of long struggle at the laboratory bench and many long hours under the harsh lights of the federal review process, gene therapy has emerged as a legitimate scientific discipline. It is now time to move away from the period of questioning whether gene therapy will be a useful part of the physician armamentarium to begin to actively teach the concepts and practices that make gene therapy a reality. This book is a comprehensive collection of chapters that describe the basic biology and potential application of viruses as gene transfer reagents. It is not a coincidence that a modified virus was the reagent used in the first human gene therapy trials. Viruses have evolved with the human species (and most likely with all forms of life) to be the masters of gene transfer.