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This first title on the topic provides complete coverage, including the molecular basis, production and possible biomedical applications. Written by the most prominent academic researchers in the field as well as by researchers at one of the world's leading companies in industrial production of minicircle DNA, this practical book is aimed at everyone who is directly or indirectly involved in the development of gene therapies.
Advances in molecular biology and recombinant DNA technology have accelerated progress in many fields of life science research, including gene therapy. A large number of genetic engineering approaches and methods are readily available for gene cloning and therapeutic vector construction. Significant progress is being made in genomic, DNA sequencing, gene expression, gene delivery and cloning. Thus gene therapy has already shown that it holds great promise for the treatment of many diseases and disorders. In general it involves the delivery of recombinant genes or transgenes into somatic cells to replace proteins with a genetic defect or to transfer with the pathological process of an illness...
This is the first book specializing in plasmids and their biomedical use, including all relevant aspects of production, applications, quality, and regulations. Readers will discover clinical applications for the wide range of preventive and therapeutic applications using plasmid DNA. The book describes modified vector systems based on plasmids, as well as the potency of genomic research and vector design by informatics. Using the example of fish vaccination, the application of DNA vaccination in veterinary health care is reviewed, followed by a detailed overview of plasmid production technology on an industrial scale. Finally, the book considers regulatory and quality assurance aspects of such new drugs plus thire market potential.
Studying organisms as a whole for potential metabolic(ally) engineering of organisms for production of (bio)chemicals is essential for industrial biotechnology. To this end, integrative analysis of different –omics measurements (transciptomics, proteomics, metabolomics, fluxomics) provides invaluable information. Combination of experimental top-down and bottom-up approaches with powerful analytical tools/techniques and mathematical modeling, namely (quantitative) systems biology, currently making the state of art of this discipline, is the only practice that would improve our understanding for the purpose. The use of high-throughput technologies induced the required development of many bio...
With its focus on a completely novel class of pharmaceuticals, this book collates the hitherto scarce literature about DNA drug formulation keenly desired by biotechnologists, molecular biologists and pharmacists, as well as those working in the biotechnological and pharmaceutical industries. As such, this volume presents a wide range of gene delivery systems needed for different therapeutic applications. It fills the gap between research and clinical trials and describes pharmaceutical fundamentals for the development of efficient DNA pharmaceuticals.
An introduction to the key professional skills and core laboratory techniques that underpin successful professional practice, providing a strong foundation for beginning biomedical science students.
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This third edition provides new and updated chapters on gene therapeutic strategies of cancer. Chapters guide readers through suicide and oncolytic gene therapy, gene replacement and gene suppression therapy, vector development and refinement, immunogene therapy, TCR and CAR engineering, tumor vaccination using DNA or RNA vaccines, and antitumoral immune stimulation at different levels. Written in the format of the highly successful Methods in Molecular Biology series, each chapter includes an introduction to the topic, lists necessary materials and reagents, includes tips on troubleshooting and known pitfalls, and step-by-step, readily reproducible protocols. Authoritative and cutting-edge, Gene Therapy of Cancer: Methods and Protocols, Third Edition aims to be a useful and practical guide to new researchers and experts looking to expand their knowledge.
This unique advanced textbook provides a clear and comprehensive overview of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations. The second edition features new groundbreaking material on genome editing using the recently discovered CRISPR/Cas9 system and on cancer immunotherapy by CAR-T cells. It also presents the historical milestone of gene therapy application in the field of severe combined immunodeficiency, and other fields of gene therapy and molecular medicine.The use of gene transfer is exponentially growing in the scientific and medical communities ...
Genetic / DNA immunization represents a novel approach to vaccine and immune therapeutic development. The direct injec tion of nucleic acid expression cassettes into a living host results in a limited number of its cells becoming factories for production of the introduced gene products. This host-inappropriate gene expression has important immunological consequences, resulting in the specific immune activation of the host against the gene delivered antigen. The recent demonstration by a number of laboratories that the induced immune responses are functional in experimental models against both specific infectious diseases and cancers is likely to have dramatic consequences for the develop men...