Are you looking for read ebook online? Search for your book and save it on your Kindle device, PC, phones or tablets. Download Human Genome Editing PDF full book. Access full book title Human Genome Editing by National Academies of Sciences, Engineering, and Medicine. Download full books in PDF and EPUB format.
Author: National Academies of Sciences, Engineering, and Medicine Publisher: National Academies Press ISBN: 0309452880 Category : Medical Languages : en Pages : 329
Book Description
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: 0309452880 Category : Medical Languages : en Pages : 329
Book Description
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: 0309672988 Category : Medical Languages : en Pages : 127
Book Description
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.
Author: David T. Curiel Publisher: Academic Press ISBN: 0128005106 Category : Science Languages : en Pages : 870
Book Description
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
Author: P.L. Chang Publisher: CRC Press ISBN: 1351093665 Category : Science Languages : en Pages : 242
Book Description
As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.
Author: Ravin Narain Publisher: Woodhead Publishing ISBN: 0081005210 Category : Science Languages : en Pages : 303
Book Description
Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. - Explores current challenges in the research of genetic diseases - Discusses polymers for gene therapy and their function in designing advanced materials - Provides examples of organic and inorganic nanomaterials for gene therapy - Includes labeling, targeting, and assays - Looks at characterization, physico-(bio)chemical properties, and applications
Author: Manzoor M. Khan Publisher: Springer Science & Business Media ISBN: 0387779760 Category : Medical Languages : en Pages : 275
Book Description
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: Stephen H. Tsang Publisher: Springer ISBN: 3319639048 Category : Science Languages : en Pages : 180
Book Description
This book presents descriptive overviews of gene editing strategies across multiple species while also offering in-depth insight on complex cases of application in the field of tissue engineering and regenerative medicine. Chapters feature contributions from leaders in stem cell therapy and biology, providing a comprehensive view of the application of gene therapy in numerous fields with an emphasis on ophthalmology, stem cells, and agriculture. The book also highlights recent major technological advances, including ZFN, TALEN, and CRISPR. Precision Medicine, CRISPR, and Genome Engineering is part of the highly successful Advances in Experimental Medicine and Biology series. It is an indispensable resource for researchers and students in genetics as well as clinicians.
Author: Daan J. A. Crommelin Publisher: CRC Press ISBN: 9780415285018 Category : Medical Languages : en Pages : 456
Book Description
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: Institute of Medicine Publisher: National Academies Press ISBN: 0309260248 Category : Medical Languages : en Pages : 89
Book Description
The number of new drug approvals has remained reasonably steady for the past 50 years at around 20 to 30 per year, while at the same time the total spending on health-related research and development has tripled since 1990. There are many suspected causes for this trend, including increases in regulatory barriers, the rising costs of scientific inquiry, a decrease in research and development efficiency, the downstream effects of patient expirations on investment, and the lack of production models that have successfully incorporated new technology. Regardless, this trajectory is not economically sustainable for the businesses involved, and, in response, many companies are turning toward collaborative models of drug development, whether with other industrial firms, academia, or government. Introducing greater efficiency and knowledge into these new models and aligning incentives among participants may help to reverse the trends highlighted above, while producing more effective drugs in the process. Genome-Based Therapeutics explains that new technologies have the potential to open up avenues of development and to identify new drug targets to pursue. Specifically, improved validation of gene-disease associations through genomics research has the potential to revolutionize drug production and lower development costs. Genetic information has helped developers by increasing their understanding of the mechanisms of disease as well as individual patients' reactions to their medications. There is a need to identify the success factors for the various models that are being developed, whether they are industry-led, academia-led, or collaborations between the two. Genome-Based Therapeutics summarizes a workshop that was held on March 21, 2012, titled New Paradigms in Drug Discovery: How Genomic Data Are Being Used to Revolutionize the Drug Discovery and Development Process. At this workshop the goal was to examine the general approaches being used to apply successes achieved so far, and the challenges ahead.
Author: Stanton L. Gerson Publisher: Elsevier ISBN: 0080491367 Category : Science Languages : en Pages : 555
Book Description
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
Author: National Academies of Sciences, Engineering, and Medicine
Author: National Academies of Sciences, Engineering, and Medicine
Author: National Academies of Sciences, Engineering, and Medicine
Author: David T. Curiel
Author: P.L. Chang
Author: Ravin Narain
Author: Manzoor M. Khan
Author: Stephen H. Tsang
Author: Daan J. A. Crommelin
Author: Institute of Medicine
Author: Stanton L. Gerson