Clinical Trials Of Genetic Therapy With Antisense Dna And Dna Vectors

Author : Eric Wickstrom
Publisher : CRC Press
Page : 452 pages
File Size : 45,7 Mb
Release : 2020-08-13
Category : Medical
ISBN : 9781000145953

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Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors by Eric Wickstrom Pdf

An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors. Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors furnishes a historical overview of genetic therapy highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications illustrates for new practitioners how each stage of genetic therapy is developed details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy and addresses questions of preparation, delivery, toxicity, mechanism, and specificity.

Author : Manzoor M. Khan
Publisher : Springer Science & Business Media
Page : 275 pages
File Size : 55,8 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.

Author : Daan J. A. Crommelin,Robert D. Sindelar
Publisher : CRC Press
Page : 456 pages
File Size : 44,8 Mb
Release : 2002-11-14
Category : Medical
ISBN : 0415285011

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Pharmaceutical Biotechnology by Daan J. A. Crommelin,Robert D. Sindelar Pdf

The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.

Author : The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada
Publisher : Council of Canadian Academies
Page : 122 pages
File Size : 40,5 Mb
Release : 2020-11-05
Category : Medical
ISBN : 9781926522791

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From Research to Reality by The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada Pdf

From Research to Reality describes the stages involved in the approval and use of gene therapies in Canada, and examines challenges associated with regulatory oversight, manufacturing, access, and affordability, and identifies promising approaches to address them.

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 : 41,7 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.

Author : Thomas F. Kresina
Publisher : John Wiley & Sons
Page : 408 pages
File Size : 50,9 Mb
Release : 2004-03-24
Category : Science
ISBN : 9780471461043

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An Introduction to Molecular Medicine and Gene Therapy by Thomas F. Kresina Pdf

An Introduction to Molecular Medicine and Gene Therapy Edited by Thomas F. Kresina, Ph.D. Gene therapy, or the use of genetic manipulation for disease treatment, is derived from advances in genetics, molecular biology, clinical medicine, and human genomics. Molecular medicine, the application of molecular biological techniques to disease treatment and diagnosis, is derived from the development of human organ transplantation, pharmacotherapy, and elucidation of the human genome. An Introduction to Molecular Medicine and Gene Therapy provides a basis for interpreting new clinical and basic research findings in the areas of cloning, gene transfer, and targeting; the applications of genetic medicine to clinical conditions; ethics and governmental regulations; and the burgeoning fields of genomics, biotechnology, and bioinformatics. By dividing the material into three sections - an introduction to basic science, a review of clinical applications, and a discussion of the evolving issues related to gene therapy and molecular medicine-this comprehensive manual describes the basic approaches to the broad range of actual and potential genetic-based therapies. In addition, An Introduction to Molecular Medicine and Gene Therapy: * Covers new frontiers in gene therapy, animal models, vectors, gene targeting, and ethical/legal considerations * Provides organ-based reviews of current studies in gene therapy for monogenetic, multifactoral or polygenic disorders, and infectious diseases * Includes bold-faced terms, key concepts, summaries, and lists of helpful references by subject in each chapter * Contains appendices on commercial implications and a review of the history of gene therapy This textbook offers a clear, concise writing style, drawing upon the expertise of the authors, all renowned researchers in their respective specialties of molecular medicine. Researchers in genetics and molecular medicine will all find An Introduction to Molecular Medicine and Gene Therapy to be an essential guide to the rapidly evolving field of gene therapy and its applications in molecular medicine.

Author : Kam M Hui
Publisher : World Scientific
Page : 289 pages
File Size : 41,6 Mb
Release : 1994-11-29
Category : Science
ISBN : 9789814502382

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Gene Therapy - From Laboratory To The Clinic by Kam M Hui Pdf

With advances in our understanding of the molecular biology of human diseases and the development of efficient gene transfer techniques, the treatment of such diseases as cancer and infectious disease using gene therapy has progressed from a distant prospect to a distinct possibility in a very short time. The development of gene transfer methods which are suitable for different forms of therapy has been a major topic of research over the past several years. A common goal of this research has been to achieve the efficient delivery of genes into cells. The successful implementation of gene transfer as a cure for diseases, however, will continue to require the translation of preclinical studies in gene therapy into effective clinical protocols. This volume outlines the latest developments in cancer treatment using various gene delivery systems, which include cytokine gene transfer, the delivery of anti-ras DNA by retroviral vector and the injection of allogeneic HLA DNA via liposomes. Several of these molecular approaches have recently been approved by the US FDA as human clinical trial protocols in order to assess their therapeutic efficiency and safety for cancer treatment. Further developments in recombinant DNA technology within this field should ultimately lead to dramatic improvements in the practice of medicine.

Author : Alexander V. Kabanov,Philip L. Felgner,Leonard W. Seymour
Publisher : Wiley-Blackwell
Page : 492 pages
File Size : 50,6 Mb
Release : 1998-05-29
Category : Medical
ISBN : UOM:39015042032857

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Self-Assembling Complexes for Gene Delivery by Alexander V. Kabanov,Philip L. Felgner,Leonard W. Seymour Pdf

Recent years have witnessed an explosion of activity in the field of gene therapy. Following advances in our understanding of the molecular basis of disease, hopes are high that the tremendous potential market for drugs employing antisense nucleotides and genes will one day be fulfilled. However, many obstacles remain, not least problems in the technology of gene delivery systems. Much of today's research focuses on non-viral approaches to gene delivery. Of particular importance are supramolecular complexes formed between DNA and various natural and synthetic polymers and lipids, otherwise known as 'self-assembling complexes for gene delivery'. In view of this fact, the editors of this volume have assembled an international team of contributors to present up-to-date reviews of the major chemical, biological and clinical aspects of such gene delivery vectors. Reflecting the diversity of research in this field, this book comprehensively covers: * the principles of self-assembly * natural mechanisms for gene delivery to cells * cationic lipids and liposomes * polyelectrolyte DNA complexes * systemic biodistribution of drug delivery systems * targeting of conjugates for gene delivery * new approaches to gene delivery * clinical evaluation Self-assembling Complexes for Gene Delivery is an essential reference for all professionals with an interest in gene and antisense therapy or novel drug delivery systems, including medicinal and pharmaceutical chemists, clinicians, human geneticists, molecular biologists and pharmacologists.

Author : Institute of Medicine,Board on Health Sciences Policy,Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee
Publisher : National Academies Press
Page : 78 pages
File Size : 51,7 Mb
Release : 2014-03-27
Category : Medical
ISBN : 9780309296656

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Oversight and Review of Clinical Gene Transfer Protocols by Institute of Medicine,Board on Health Sciences Policy,Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee Pdf

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.

Author : Indu Ravi,Mamta Baunthiyal,Jyoti Saxena
Publisher : Springer Science & Business Media
Page : 264 pages
File Size : 49,7 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.

Author : Anonim
Publisher : Academic Press
Page : 296 pages
File Size : 42,8 Mb
Release : 2015-01-23
Category : Science
ISBN : 9780128024706

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Nonviral Vectors for Gene Therapy by Anonim Pdf

The field of genetics is rapidly evolving, and new medical breakthroughs are occurring as a result of advances in our knowledge of genetics. Advances in Genetics continually publishes important reviews of the broadest interest to geneticists and their colleagues in affiliated disciplines. Includes methods for testing with ethical, legal, and social implications Critically analyzes future directions Written and edited by recognized leaders in the field

Author : Nancy Smyth Templeton,D. D. Lasic
Publisher : CRC Press
Page : 616 pages
File Size : 42,8 Mb
Release : 2000-06-15
Category : Medical
ISBN : UOM:39015050321036

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Gene Therapy by Nancy Smyth Templeton,D. D. Lasic Pdf

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.

Author : R. Herzog,L. Popplewell
Publisher : Karger Medical and Scientific Publishers
Page : 102 pages
File Size : 50,6 Mb
Release : 2020-04-24
Category : Medical
ISBN : 9783318066678

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Fast Facts: Gene Therapy by R. Herzog,L. Popplewell Pdf

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

Author : United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions. Subcommittee on Public Health
Publisher : Unknown
Page : 132 pages
File Size : 48,7 Mb
Release : 2000
Category : Law
ISBN : STANFORD:36105062717033

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Gene Therapy by United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions. Subcommittee on Public Health Pdf

Author : Ambikanandan Misra
Publisher : Elsevier
Page : 686 pages
File Size : 43,5 Mb
Release : 2010-09-09
Category : Science
ISBN : 9780123849656

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Challenges in Delivery of Therapeutic Genomics and Proteomics by Ambikanandan Misra Pdf

Delivery of therapeutic proteomics and genomics represent an important area of drug delivery research. Genomics and proteomics approaches could be used to direct drug development processes by unearthing pathways involved in disease pathogenesis where intervention may be most successful. This book describes the basics of genomics and proteomics and highlights the various chemical, physical and biological approaches to protein and gene delivery. Covers a diverse array of topics from basic sciences to therapeutic applications of proteomics and genomics delivery Of interest to researchers in both academia and industry Highlights what’s currently known and where further research is needed