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A Special Report On Gene Therapy
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Book Synopsis A Special Report on Gene Therapy by : K. K. Jain
Download or read book A Special Report on Gene Therapy written by K. K. Jain and published by John Wiley & Sons. This book was released on 2000-04-14 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt: This special report on Gene Therapy Companies by K.K. Jain provides an up-to-date overview of the gene therapy industry. It contains a wealth of very practical information on 105 companies. For each company, information is available on the people to contact, their contact details, a corporate history, the company's technologies and products, financial data, patents held, products in clinical trials, collaborations with other companies and selected publications from the company's scientists. Almost 200 companies were initially considered for inclusion but just over 100 companies were finally selected as they fulfilled the necessary criteria to be classified as a 'Gene Therapy Company,', defined broadly to include cell therapy companies. Not all of the companies devote their activity exclusively to gene therapy but they have sufficient relevance to be included. As part of this project, new companies were discovered that have not been reported in other publications or on the internet. Dr. Jain has spared no effort to ensure that the information is up-to-date and accurate at the time of publishing. In most instances, the information was reviewed by the companies to correct any outdated information or to add any complementary or recent information. In addition to the detailed descriptions of each company, the data is also presented in summary tabular form so that at a glance the reader can identify collaborations within the gene therapy industry, or determine which companies are pursuing strategies involving viral, non-viral or other approaches to gene therapy, and which diseases are being targetted. Given that much of the gene therapy research and development is currently being carried out within the industrial sector, it is important for scientists, managers and investors to have the relevant information at their fingertips. This special report provides that information in one accurate, easy to consult and up-to-date source.
Book Synopsis Oversight and Review of Clinical Gene Transfer Protocols by : Institute of Medicine
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.
Author :National Academies of Sciences, Engineering, and Medicine Publisher :National Academies Press ISBN 13 :0309452880 Total Pages :329 pages Book Rating :4.3/5 (94 download)
Book Synopsis Human Genome Editing by : National Academies of Sciences, Engineering, and Medicine
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.
Author :National Academies of Sciences, Engineering, and Medicine Publisher :National Academies Press ISBN 13 :0309672988 Total Pages :127 pages Book Rating :4.3/5 (96 download)
Book Synopsis Exploring Novel Clinical Trial Designs for Gene-Based Therapies by : National Academies of Sciences, Engineering, and Medicine
Download or read book Exploring Novel Clinical Trial Designs for Gene-Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
Book Synopsis Immunopharmacology by : Manzoor M. Khan
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.
Book Synopsis Pharmaceutical Biotechnology by : Daan J. A. Crommelin
Download or read book Pharmaceutical Biotechnology written by Daan J. A. Crommelin and published by CRC Press. This book was released on 2002-11-14 with total page 456 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Book Synopsis Novel Gene Therapy Approaches by : Ming Wei
Download or read book Novel Gene Therapy Approaches written by Ming Wei and published by BoD – Books on Demand. This book was released on 2013-02-13 with total page 408 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy has the potential to be a tailor-made therapeutic with increased specificity and decreased side effects that can offer a "cure" for many disorders. The aim of this book is to provide up-to-date reviews of the rapidly growing field of gene therapy. Chapters cover a large range of topics including methods of gene delivery, and identification of targets with several papers on cancer gene therapy. If more people become aware of the true nature and potential of gene therapy, perhaps we can achieve the full benefit of such an innovative approach for the treatment of a range of diseases, including cancer.
Book Synopsis Regulatory Aspects of Gene Therapy and Cell Therapy Products by : Maria Cristina Galli
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.
Author :The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada Publisher :Council of Canadian Academies ISBN 13 :1926522796 Total Pages :122 pages Book Rating :4.9/5 (265 download)
Book Synopsis From Research to Reality by : The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada
Download or read book From Research to Reality written by The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada and published by Council of Canadian Academies. This book was released on 2020-11-05 with total page 122 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Book Synopsis Cell and Gene Therapies by : Miguel-Angel Perales
Download or read book Cell and Gene Therapies written by Miguel-Angel Perales and published by Springer. This book was released on 2018-11-27 with total page 286 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.
Book Synopsis Gene Therapy for Viral Infections by : Patrick Arbuthnot
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
Book Synopsis Heritable Human Genome Editing by : The Royal Society
Download or read book Heritable Human Genome Editing written by The Royal Society and published by National Academies Press. This book was released on 2021-01-16 with total page 239 pages. Available in PDF, EPUB and Kindle. Book excerpt: Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight.
Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog
Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Book Synopsis Assessing Genetic Risks by : Institute of Medicine
Download or read book Assessing Genetic Risks written by Institute of Medicine and published by National Academies Press. This book was released on 1994-01-01 with total page 353 pages. Available in PDF, EPUB and Kindle. Book excerpt: Raising hopes for disease treatment and prevention, but also the specter of discrimination and "designer genes," genetic testing is potentially one of the most socially explosive developments of our time. This book presents a current assessment of this rapidly evolving field, offering principles for actions and research and recommendations on key issues in genetic testing and screening. Advantages of early genetic knowledge are balanced with issues associated with such knowledge: availability of treatment, privacy and discrimination, personal decision-making, public health objectives, cost, and more. Among the important issues covered: Quality control in genetic testing. Appropriate roles for public agencies, private health practitioners, and laboratories. Value-neutral education and counseling for persons considering testing. Use of test results in insurance, employment, and other settings.
Book Synopsis Mutation-Specific Gene Editing for Blood Disorders by : Carsten Werner Lederer
Download or read book Mutation-Specific Gene Editing for Blood Disorders written by Carsten Werner Lederer and published by Frontiers Media SA. This book was released on 2021-11-25 with total page 218 pages. Available in PDF, EPUB and Kindle. Book excerpt: Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com.
Author :United States. President's Commission for the Study of Ethical Problems in Medicine and Biomedical and Behavioral Research Publisher : ISBN 13 : Total Pages :128 pages Book Rating :4.:/5 (319 download)
Book Synopsis Splicing Life by : United States. President's Commission for the Study of Ethical Problems in Medicine and Biomedical and Behavioral Research
Download or read book Splicing Life written by United States. President's Commission for the Study of Ethical Problems in Medicine and Biomedical and Behavioral Research and published by . This book was released on 1982 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Textbook of Gene Therapy by : Kewal K. Jain
Download or read book Textbook of Gene Therapy written by Kewal K. Jain and published by Seattle ; Toronto : Hogrefe & Huber. This book was released on 1998 with total page 460 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the past eight years following initial gene therapy experiments, more than 200 clinical protocols have been submitted worldwide and more than 2000 patients have been treated. Although a lot remains to be accomplished before gene therapy becomes a standard medical practice, enough information has accumulated to justify a textbook on the subject. Some of the procedures and products are expected to enter the marketplace and medical practice by the year 2000. The public is very much aware of the potential of gene therapy and the medical profession should prepare itself for the new developments by learning about the basics and applications of these techniques. Much of there-search has been conducted in the bio-pharmaceutical industry by gene therapy companies and the commercial opportunities for gene therapy are enormous. A large segment of the society thus has an interest in gene therapy. This book presents a unique critical review of the tremendous progress which has been made in this field. The textbook is an effort to bring a wide range of important developments together in an accessible format. This book is aimed at physicians, gene therapists, molecular biologists, nurse practitioners and students in these fields, as well as other healthcare professionals interested in developments in the field of gene therapy and its impact, both short- and long-term, on the practice of medicine. Industrial executives planning long-term strategies in gene therapy will find this handy textbook to be a comprehensive source of information on the subject and the companies involved in developing it. Prof. K. K. Jain is a neurosurgeon trained in Canada and the United States. He has heldacademic positions in major medical centers around the world. For the last decade, he has been a consultant to a number of major biopharmaceutical companies and now runs his own biotechnology company specializing in gene therapy research and documentation.
