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Author : Jose Martinez-Navio,Nicole K. Paulk,Guangping Gao Publisher : Frontiers Media SA Page : 187 pages File Size : 41,7 Mb Release : 2022-02-09 Category : Medical ISBN : 9782889743063
AAV Gene Therapy: Immunology and Immunotherapeutics by Jose Martinez-Navio,Nicole K. Paulk,Guangping Gao Pdf
Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma. The other Topic Editors declare no conflict of interest with regards to the Research Topic theme
Summarises and reviews the important field of genetic therapy with respect to the latest immunological advances in the lab and clinic Unique treatment of immunology and immunotherapy in gene - approached from a vector and target organ point of view rather than from the perspective a specific diseases Broad appeal - applicable for immunology and genetics / gene therapy, recombinant DNA studies, transplantation, virology, cancer research and tumor research
Gene Therapy for Autoimmune and Inflammatory Diseases by Yuti Chernajovsky,Paul D. Robbins Pdf
In this monograph about gene therapy of autoimmune and inflammatory d- orders we have gathered international experts and leaders from different fields to review the state of the art advances on topics ranging from disease entities to vectors and engineered cells. The different approaches described in each chapter take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long-term genetic interventions. Gene therapy also serves as a testing ground for new therapeutic entities and helps provide proof of principle for their potential therapeutic role in animal models of disease. Scaling up from mice to men still remains an important h- dle not only from the quantitative point of view, but also for currently unknown and unexpected secondary effects of the vector or the transgene. Some of these approaches have already been tested in the clinic, but much more needs to be done to understand the human conditions treated and the n- ural history of their pathology. We are indebted to the secretarial assistance of Ms. Lin Wells (Bone and Joint Research Unit, London, UK) and the help of Hans Detlef Klüber for his help in getting this book published. We hope this book will be of interest to c- nicians and scientists and inspiring to students of the subject who will use their own ingenuity and knowledge to further forward this discipline into clinical use.
Approaches to Blocking the Immune Response to Gene Transfer with Viral Vectors by Katherine High,Roland W. Herzog,Hildegund C. Ertl Pdf
Viral vectors are superior tools for gene therapy and as a genetic vaccine platform because viruses have evolved to efficiently infect and transfer their genomes to cells. Several impressive successes in viral vector-based gene therapies have been reported in humans, including restoration of vision in patients with Leber’s congenital amaurosis by retinal gene transfer and cures for severe immune deficiencies by gene transfer to hematopoietic stem cells. However, the mammalian immune system has evolved in parallel to fend off invading pathogens such as viruses. Innate and antigen-specific adaptive immune responses against viral vectors and therapeutic transgene products pose serious hurdles for successful gene therapy. Pre-existing immunity in humans, resulting from prior exposure to the parent virus that forms the basis for the gene transfer vehicle may be derived from, often prevents efficient gene transfer. This problem also reduces our ability to use certain vectors for genetic vaccination or in anti-cancer therapy. For these reasons, the gene transfer community has been extensively studying the mechanisms of immune responses against viral vectors and has started to develop strategies and protocols to block or circumvent such responses. Choice, design and engineering of a vector as well as the route of administration/target tissue can be optimized/ altered to minimize immune responses or evade pre-existing immunity. Immune suppression and modulation strategies are being developed in order to minimize inflammation, prevent antibody or T cell responses against vectors, and to promote tolerance to therapeutic gene products. Combinations of these approaches will likely facilitate clinical applications of gene therapy for many target diseases and also aid in vaccine development.
Gene Therapy for HIV and Chronic Infections by Ben Berkhout,Hildegund C.J. Ertl,Marc S. Weinberg Pdf
This book centers on gene therapy and gene transfer approaches to prevent or treat chronic virus infections. The main focus is on the Big Three: human immunodeficiency virus (HIV-1), hepatitis B virus (HBV) and hepatitis C virus (HCV). Ample anti-HIV drugs are currently available in the clinic and the development of an effective combination therapy has dramatically improved the lifespan and quality of life of infected individuals. A similar trend can already be recognized for HBV and HCV: the development of multiple (directly acting) antiviral drugs and plans to control or even cure the infection. However, approaches that help prevent infection, or which provide long-lasting treatment (such as a cure) remain important goals. Immunization through gene transfer vehicles encoding immunogenic viral proteins shows promise in preventing infections with complex, highly variable, viruses such as HIV-1 or HCV. Gene therapy applications for virus infections have been discussed since the early 1990’s. Whereas a true cure seems difficult to achieve for HIV-1 due to its intrinsic property to deposit its genome into that of the host, such attempts may be within reach for HCV where spontaneous viral clearance occurs in a small percentage of the infected individuals. The prospect of original gene therapy approaches may provide alternative ways to reach the same endpoint by, for example, silencing of CCR5 expression post-transcriptionally. Many alternative antiviral strategies have been developed based on a variety of novel molecular methods: e.g. ribozymes. Some studies have progressed towards pre-clinical animal models and a few antiviral gene therapies have progressed towards clinical trials. This book provides an overview of this rapidly progressing field, while focusing on the interface of gene therapy and immunology/vaccinology.
Biologic and Gene Therapy of Autoimmune Disease by C. Garrison Fathman Pdf
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 of Autoimmune Disease by Gerald J. Prud'homme Pdf
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.
ince the early 1980s, the HIV epidemic has been raging within the S 1 United States and around the world. Drug therapy for HIV infection has not been curative, prompting the search for alternative strategies to control HIV infection within infected persons. One potential alterna tive to drug therapy is a developing medical technology termed gene therapy. 2 Gene therapy involves introducing genetic elements into popu lations of cells in order to correct or prevent a pathologic process. A large number of gene therapy strategies have been developed in an at tempt to inhibit HIV expression and spread. These strategies fall into two general categories, genetic modification of cells in order to elicit an immune response against HIV and genetic modification of the target cells of HIV infection in order to block HIV expression and reproduction. In the first strategy, termed genetic immunotherapy by some, genetic material encoding HIV proteins is introduced into patient's cells in order to stimulate a cellular immune response above and beyond 3 5 that stimulated by the viral infection itself. - Two general genetic im munotherapy strategies have been developed. Genes encoding HIV pro teins have been directly injected into the dermis or muscle tissue of patients. These genes have been encoded in plasmids or viral DNA and have been injected either in the form of naked DNA or complexed with lipids.
The Skin and Gene Therapy by Ulrich R. Hengge,Beatrix Volc-Platzer Pdf
Basic Aspects.- 1 The Epidermal Barrier and Strategies for Surmounting It: An Overview.- 2 Stem Cells, Differentiation and Renewal Kinetics of Keratinocytes: Implications for Cutaneous Gene Therapy.- 3 Relevant Animal Models for Skin Gene Therapy.- 4 Nonviral Gene Transfer into the Skin.- 5 Safety and Pharmacokinetics of Naked Plasmid DNA: Studies on Dissemination and Ectopic Expression.- 6 Uptake of DNA by Keratinocytes.- Treatment of Skin Diseases.- Gene Therapy of Inherited Skin Diseases.- Gene Transfer Strategies in Tissue Repair.- Systemic Effects of Skin Gene Therapy.- The Use of Skin-Directed Gene Therapy in the Treatment of Systemic Diseases.- Keratinocyte Gene Therapy Using Cytokine Genes.- Genetic Vaccination Using the Skin.- Principles of Genetic Immunization.- Systematic Modulation of Immune Responses by CpG DNA.- Genetic and Dendritic Cell Vaccination as a Novel Therapy for Melanoma.- Molecular Strategies Interfering with Tumor Progression of Melanoma and Improving Anti-Tumor Immunity.- Prophylactic and Therapeutic DNA Vaccines Against Infectious Diseases.
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.
Gene Technology by Axel R. Zander,Wolfram Ostertag,Boris V. Afanasiev,Frank Grosveld Pdf
The 11 th meeting in "Modern Trends in Human Leukemia" took place from June 19 to 21, 1994 in Wilsede in the middle of the Liineburger Heide, South of Hamburg. Interwoven with the Leukemia program was the Nato-sponsored Symposium of the ASI-Series "Gene Technology in Analysis of Malignant and Inherited Human Diseases Related to Development" . The Wilsede meeting was continued on a ship of the Neva leading through lake Ladoga and lake Onega. The topics of both meetings included discussion on recent progress isolation and development of hematopoietic stem cells, genes crucial for development and diseases, methods of gene transfer, application of gene transfer; oncogenes and anti-oncogenes as targets for gene therapy; receptors and their ligands in normal development and diseases, immunology and immunotherapy, radiation biology, clinical leukemias and bone marrow transplantation. The Nato workshop concentrated not only on analysis of cell systems useful for somatic gene therapy, but also on actual themes directly related to correction of human diseases. The latter aspects emphasized themes related to biotechnology, the first part was by nature more general. We also included a few contributions that discussed perspectives for the future of gene therapy and possible relationships to evolution.
Lentiviral Vectors and Gene Therapy by David Escors,Karine Breckpot,Frederick Arce,Grazyna Kochan,Holly Stephenson Pdf
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.
With the coming of the new millennium we are witnessing a revolution in our understanding of cancer genetics. These are very exciting times. Today we have at our disposal the technology to diagnose abnormalities in our cancer genes and the means to correct the deficit and very soon we will have the complete sequence of the human genome. With the use of gene chip technology the way doctors will be able to assess patients will change completely. Today we can diagnose abnormalities in ten thousand genes and within a short period of time we will be able to screen through our genome and discover potential abnormalities in our proto-oncogenes, tumour suppressor genes, differentiating genes, apoptotic genes and pro-inflammatory genes. In this book various authors have highlighted specific genes that could be expressed, overexpressed, neutralised or h- nessed to achieve cancer control. The problem of transferring the therapeutic gene into the cancer cell has been partly addressed with major developments in the field of naked plasmid DNA, adenovirus, retrovirus and adeno-associated viruses. However, further improvements are yet to be made to achieve significant gene transfer. Gene expression, in particular specificity of gene transfer, is obviously an important issue and one which is highlighted in this book by the use of specific promoter.
Author : Gerhard Bauer,Joseph S. Anderson Publisher : Springer Science & Business Media Page : 72 pages File Size : 47,6 Mb Release : 2014-02-07 Category : Medical ISBN : 9781493904341
Gene Therapy for HIV by Gerhard Bauer,Joseph S. Anderson Pdf
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.