Approaches To Blocking The Immune Response To Gene Transfer With Viral Vectors

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Approaches to Blocking the Immune Response to Gene Transfer with Viral Vectors

Author : Katherine High,Roland W. Herzog,Hildegund C. Ertl
Publisher : Frontiers E-books
Page : 96 pages
File Size : 47,8 Mb
Release : 2024-07-02
Category : Electronic
ISBN : 9782889190553

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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.

Adenoviral Vectors for Gene Therapy

Author : David T. Curiel
Publisher : Academic Press
Page : 868 pages
File Size : 46,6 Mb
Release : 2016-03-10
Category : Science
ISBN : 9780128005101

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Adenoviral Vectors for Gene Therapy by David T. Curiel Pdf

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

Immunopharmacology

Author : Manzoor M. Khan
Publisher : Springer Science & Business Media
Page : 275 pages
File Size : 46,7 Mb
Release : 2008-12-19
Category : Medical
ISBN : 9780387779768

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Immunopharmacology by Manzoor M. Khan Pdf

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 Viral Infections

Author : Patrick Arbuthnot
Publisher : Academic Press
Page : 392 pages
File Size : 50,6 Mb
Release : 2015-06-01
Category : Science
ISBN : 9780124114524

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Gene Therapy for Viral Infections by Patrick Arbuthnot Pdf

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

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Publisher : National Academies Press
Page : 93 pages
File Size : 51,5 Mb
Release : 2019-12-01
Category : Medical
ISBN : 9780309495844

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Advancing Gene-Targeted Therapies for Central Nervous System Disorders by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders Pdf

On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.

Fields Virology: Emerging Viruses

Author : Peter M. Howley,David M. Knipe
Publisher : Lippincott Williams & Wilkins
Page : 2597 pages
File Size : 40,9 Mb
Release : 2020-02-11
Category : Medical
ISBN : 9781975112554

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Fields Virology: Emerging Viruses by Peter M. Howley,David M. Knipe Pdf

Now in four convenient volumes, Field’s Virology remains the most authoritative reference in this fast-changing field, providing definitive coverage of virology, including virus biology as well as replication and medical aspects of specific virus families. This volume of Field’s Virology: Emerging Viruses, 7th Edition covers recent changes in emerging viruses, providing new or extensively revised chapters that reflect these advances in this dynamic field.

Janeway's Immunobiology

Author : Kenneth Murphy,Paul Travers,Mark Walport,Peter Walter
Publisher : Garland Science
Page : 128 pages
File Size : 45,5 Mb
Release : 2010-06-22
Category : Medical
ISBN : 0815344570

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Janeway's Immunobiology by Kenneth Murphy,Paul Travers,Mark Walport,Peter Walter Pdf

The Janeway's Immunobiology CD-ROM, Immunobiology Interactive, is included with each book, and can be purchased separately. It contains animations and videos with voiceover narration, as well as the figures from the text for presentation purposes.

Advances in Biotechnology

Author : Indu Ravi,Mamta Baunthiyal,Jyoti Saxena
Publisher : Springer Science & Business Media
Page : 264 pages
File Size : 55,9 Mb
Release : 2013-10-21
Category : Science
ISBN : 9788132215547

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Advances in Biotechnology by Indu Ravi,Mamta Baunthiyal,Jyoti Saxena Pdf

The book “Advances in Biotechnology” is about recent advances in some of the important fields that are ongoing in certain biotechnological applications. Biotechnology has been quite helpful in keeping pace with the demands of every increasing human population and in improving the quality of human life. Major biotechnological achievements associated with human welfare have been from the fields like genetic engineering; transgenic plants and animals; genomics, proteomics, monoclonal antibodies for the diagnosis of disease, gene therapy etc. Fourteen authoritative chapters written by experts having experience in academics and research on current developments and future trends in biotechnology have been empathized. The book provides a detailed account of various methodologies used in biotechnology i.e. High capacity vectors, DNA sequencing dealing with next generation sequencing, Molecular markers, DNA microarray technology, as well as Proteomics that have revolutionized biotechnology with a wide array of applications. The book not only presents a well-founded explanation of the topics but also aims to present up-to-date reviews of current research efforts, some thoughtful discussions on the potential benefits and risks involved in producing biotechnological products and the challenges of bringing such products to market. It will prove to be an excellent reference work for both academicians and researchers, indicating new starting points to young researchers for new projects in the field. The book is intended for biotechnologist, biologist, researchers, teachers and students of Biosciences and Biotechnology.

Chimpanzees in Biomedical and Behavioral Research

Author : National Research Council,Division on Earth and Life Studies,Board on Life Sciences,Institute of Medicine,Board on Health Sciences Policy,Committee on the Use of Chimpanzees in Biomedical and Behavioral Research
Publisher : National Academies Press
Page : 200 pages
File Size : 41,9 Mb
Release : 2012-01-05
Category : Science
ISBN : 9780309220392

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Chimpanzees in Biomedical and Behavioral Research by National Research Council,Division on Earth and Life Studies,Board on Life Sciences,Institute of Medicine,Board on Health Sciences Policy,Committee on the Use of Chimpanzees in Biomedical and Behavioral Research Pdf

For many years, experiments using chimpanzees have been instrumental in advancing scientific knowledge and have led to new medicines to prevent life-threatening and debilitating diseases. However, recent advances in alternate research tools have rendered chimpanzees largely unnecessary as research subjects. The Institute of Medicine, in collaboration with the National Research Council, conducted an in-depth analysis of the scientific necessity for chimpanzees in NIH-funded biomedical and behavioral research. The committee concludes that while the chimpanzee has been a valuable animal model in the past, most current biomedical research use of chimpanzees is not necessary, though noted that it is impossible to predict whether research on emerging or new diseases may necessitate chimpanzees in the future.

Gene Therapy of Autoimmune Disease

Author : Gerald J. Prud'homme
Publisher : Springer
Page : 149 pages
File Size : 48,7 Mb
Release : 2005-07-13
Category : Medical
ISBN : 0306479915

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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.

Gene Therapy of Cancer

Author : Stanton L. Gerson,Edmund C. Lattime
Publisher : Elsevier
Page : 555 pages
File Size : 52,9 Mb
Release : 2002-04-04
Category : Science
ISBN : 9780080491363

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Gene Therapy of Cancer by Stanton L. Gerson,Edmund C. Lattime Pdf

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. 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

Adenoviruses: Model and Vectors in Virus-Host Interactions

Author : Walter Doerfler,Petra Böhm
Publisher : Springer Science & Business Media
Page : 369 pages
File Size : 47,6 Mb
Release : 2013-03-09
Category : Medical
ISBN : 9783662055991

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Adenoviruses: Model and Vectors in Virus-Host Interactions by Walter Doerfler,Petra Böhm Pdf

After three volumes on adenoviruses in 1995 the past years have seen rapid progress in the field of adenovirus research. Moreover, adenoviruses have attracted considerable interest as vectors in gene transfer regimens. After the first volume on virion and structure, viral replication and host-cell interaction this second volume deals with the immune system, oncogenesis and gene therapy.

Viral Vectors for Gene Therapy

Author : Otto-Wilhelm Merten,Mohamed Al-Rubeai
Publisher : Humana
Page : 0 pages
File Size : 45,7 Mb
Release : 2011-05-19
Category : Medical
ISBN : 161779094X

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Viral Vectors for Gene Therapy by Otto-Wilhelm Merten,Mohamed Al-Rubeai Pdf

The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular BiologyTM series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.

The Molecular Basis of Human Cancer

Author : William B. Coleman,Gregory J. Tsongalis
Publisher : Humana Press
Page : 873 pages
File Size : 55,9 Mb
Release : 2016-11-11
Category : Medical
ISBN : 9781597454582

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The Molecular Basis of Human Cancer by William B. Coleman,Gregory J. Tsongalis Pdf

This book covers the concepts of molecular medicine and personalized medicine. Subsequent chapters cover the topics of genomics, transcriptomics, epigenomics, and proteomics, as the tools of molecular pathology and foundations of molecular medicine. These chapters are followed by a series of chapters that provide overviews of molecular medicine as applied broadly to neoplastic, genetic, and infectious diseases, as well as a chapter on molecular diagnostics. The volume concludes with a chapter that delves into the promise of molecular medicine in the personalized treatment of patients with complex diseases, along with a discussion of the challenges and obstacles to personalized patient care. The Molecular Basis of Human Cancer, Second Edition, is a valuable resource for oncologists, researchers, and all medical professionals who work with cancer.

Translating Gene Therapy to the Clinic

Author : Jeffrey Laurence,Michael Franklin
Publisher : Academic Press
Page : 347 pages
File Size : 40,7 Mb
Release : 2014-11-14
Category : Science
ISBN : 9780128005644

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Translating Gene Therapy to the Clinic by Jeffrey Laurence,Michael Franklin Pdf

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