Download or read book Gene Therapy Technologies Applications and Regulations written by Anthony Meager and published by John Wiley & Sons. This book was released on 1999 with total page 450 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene Therapy Technologies, Applications and Regulations From Laboratory to Clinic Edited by Anthony Meager Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK The development of gene-based technologies has been rapid over the past decade and has consequently resulted in a surge of interest in human gene therapy, the deliberate transfer of genes to somatic cells to cure or alleviate disease symptoms. Hundreds of clinical protocols involving variously designed vectors for efficient gene transfer have been developed. However, the use of such complex 'gene medicines' containing potentially heritable genes has raised numerous concerns regarding quality, efficacy and safety. Encompassing recent developments in the field and addressing current concerns this book: surveys many of the current technologies for preparing vectors for use in gene therapy protocols reviews the application of gene-mediated therapies to a range of medical conditions considers the regulatory aspects of gene therapy including product quality and safety requirements appraises the transfer of technologies from laboratory to clinic with regard to the attendant requirements and facilities for: good laboratory practice (GLP) conditions in the R&D laboratory large-scale production methods and good manufacturing practice (GMP) current in-process and final product testing Written by international experts knowledgeable about many aspects of human somatic gene therapy, this book will be an essential guide for those embarking on gene therapy technologies relevant to specifications of production and testing of products (and procedures) required to meet existing regulations, including quality, efficacy and safety considerations.
Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.
Download or read book Human Gene Therapy Current Opportunities and Future Trends written by G. M. Rubanyi and published by Springer Science & Business Media. This book was released on 2013-04-17 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: Advances in genetics, molecular biology and gene delivery technologies in recent years have led to new gene therapy strategies for treatment of a variety of diseases. This book gives a comprehensive overview of the present status and future directions of gene delivery systems and therapeutic strategies for the clinical application of gene therapy in cancer, cardiovascular and central nervous system diseases. Stem cell-based therapies and gene expression regulatory systems as novel platform technologies for various gene therapy applications are also discussed. Leading experts give excellent overviews of basic molecular aspects and clinical applications in this new emerging biomedical field.
Download or read book Gene Therapy written by Blankenstein and published by Birkhäuser. This book was released on 2012-12-06 with total page 374 pages. Available in PDF, EPUB and Kindle. Book excerpt: K. Sikora Gene therapy is one of the fastest developing areas in modern medical research. Transcending the classical preclinical and clinical disciplines, it is likely to have far reaching consequences in the practice of medicine, as we enter the next millennium. Currently, there are over 200 seperate active clinical trials with over 2,500 patients entered. These studies involve over 20 countries and include patients with a wide range of diseases, including cancer, HIV infection; cystic fibrosis (CF), haemophilia, diabetes, immune deficiencies, metabolic disorders, ischaemic heart disease and arthritis. Gene therapy can be defined as the deliberate transfer of DNA for therapeutic purposes. There is a further implication that only specific sequences containing rel evant genetic information are used; otherwise, transplantation procedures involving bone marrow, kidney or liver could be considered a form of gene therapy. The con cept of transfer of genetic information as a practical clinical tool arose from the gene-cloning technology, developed during the 1970s. Without the ability to isolate and replicate defined genetic sequences, it would be impossible to produce purified material for clinical use. The drive for the practical application of this technology came from the biotechnology industry with its quest for complex human biomole cules produced by recombinant techniques in bacteria. Within a decade, pharma ceutical-grade insulin, interferon, interleukin 2 and tumour necrosis factor were all involved in clinical trials. The next step was to obtain gene expression in vivo.
Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer. This book was released on 2015-09-15 with total page 235 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.
Download or read book Oversight and Review of Clinical Gene Transfer Protocols written by Institute of Medicine and published by National Academies Press. This book was released on 2014-03-27 with total page 78 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.
Download or read book Gene Therapy for Cancer written by Kelly K. Hunt and published by Springer Science & Business Media. This book was released on 2007-10-26 with total page 469 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Gene and Cell Therapy written by Nancy Smyth Templeton and published by CRC Press. This book was released on 2008-10-06 with total page 1154 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.
Download or read book Gene Therapy written by United States. Congress. House. Committee on Science, Space, and Technology and published by . This book was released on 1995 with total page 176 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Development of Gene Therapies written by Avery McIntosh and published by CRC Press. This book was released on 2024-05-23 with total page 490 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx Insights into commercial models, access hurdles, and health economics of gene therapies Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective
Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Gene Therapy Prospective Technology assessment in its societal context written by Jörg Niewöhner and published by Elsevier. This book was released on 2011-08-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents work that has been conducted as part of the research project "Discourse on ethical questions of biomedicine" of the interdisciplinary Working Group Bioethics and Science Communication at the Max-Delbrueck-Center for Molecular Medicine (MDC)in Berlin-Buch, Germany. This book offers ground-breaking ideas on how the daily interworking of cutting-edge biomedical research assess the broader social context and its communication to stakeholders and the public. Editors cover three aspects: Scientific, Ethical and Legal, and Perception and Communication. This work establishes an international and interdisciplinary network of excellent researchers at the beginning of their careers, who brilliantly integrate their work into the different perspectives on gene therapy from the natural and social sciences, as well as the humanities and law. * Discusses biological and cellular barriers limiting the clinical application of nonviral gene deliverysystems* Addresses such questions as: Does patent granting hinder the development of Gene Therapy products?* Offers insight in the future of public perception of gene therapy in Europe* Provides details on how to communicate risks in gene therapy
Download or read book Gene Therapy for Viral Infections written by Patrick Arbuthnot and published by Academic Press. This book was released on 2015-06-01 with total page 391 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Download or read book Gene Therapy Protocols written by Jeffrey R. Morgan and published by Springer Science & Business Media. This book was released on 2007-10-26 with total page 521 pages. Available in PDF, EPUB and Kindle. Book excerpt: Efforts in gene therapy have grown dramatically in recent years. Basic research as well as clinical activity have made exciting progress and are beg- ning to offer renewed hope that gene therapy may be able to deliver novel approaches for the treatment of inherited as well as such acquired diseases as cardiovascular disease and cancer. With the sequencing of the human genome complete, we now have a comprehensive catalog of genes that further expands the potential role of gene therapy into such new fields as tissue engineering. Central to gene therapy is the process of gene transfer; thus, advances in the technology of gene transfer are at the heart of this field’s progress. Numerous technologies, based on a variety of methods (e.g., viral-mediated, physical/ chemical), have been developed to achieve gene transfer. Some of the earliest methods, such as recombinant retroviruses, are still widely used, have undergone significant improvements, and have given rise to new vectors based on lentiviruses.
Download or read book Emerging Medical Technologies written by Gennady Ermak and published by World Scientific Publishing Company. This book was released on 2015-08-27 with total page 152 pages. Available in PDF, EPUB and Kindle. Book excerpt: The book provides an overview of new technologies in medicine. It defines each technology and gives an account of its history, and the principles, technical problems, and applications as well as controversies involved. The goal of the book is to provide a comprehensive overview of the emerging medical technologies in a simple way that is understandable to non-specialists.Biomedical science and medical technologies are developing at such a fast pace that it is hard to follow even for specialists. In addition, they have become more and more complex. This book provides readers with a basic understanding of these technologies and helps them to keep up with new developments. The value of the book is that it is comprehensive and yet concise, which makes the learning process fast and easy.
Download or read book Adenoviral Vectors for Gene Therapy written by David T. Curiel and published by Academic Press. This book was released on 2016-03-10 with total page 870 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Download or read book Gene Therapy in Neurological Disorders written by Mingjie Li and published by Academic Press. This book was released on 2018-05-25 with total page 450 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
