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In den letzten zehn Jahren hat sich die Gentherapie als eine bahnbrechende neue Behandlungsoption entwickelt, insbesondere durch die Zulassung von Produkten für die Hämophilie A und B. Diese Therapieform verspricht eine weitgehende Unabhängigkeit von regelmäßiger Substitutionstherapie bei niedrigen Blutungsraten, dank der Verwendung von adeno-assoziierten Viren (AAV) als Vektoren. Das vorliegende Buch vermittelt die aktuellen Kenntnisse zum Potenzial der neuen Gentherapeutika in der Behandlung von Hämophilie A und B. Es deckt dabei ein breites Spektrum ab: von den Grundlagen der Hämophilie über die verschiedenen gentherapeutischen Ansätze (AAV, Lentivirus, Gene Editing) bis hin zu spezifischen Aspekten wie der Auswahl geeigneter Patienten für die Gentherapie, den Auswirkungen auf Gerinnungsfaktorwerte, leberbezogenen Fragestellungen und der Immunantwort auf AAV-vermittelte Gentherapie. Es erläutert außerdem die Notwendigkeit einer interdisziplinären Zusammenarbeit und diskutiert das innovative “Hub and Spoke”-Modell. Dieses Buch soll als Brücke dienen, die die Lücke zwischen dem aktuellen Wissenstand und zukünftigen Entwicklungen schließt, und es zielt darauf ab, eine umfassende Informationsquelle für Fachleute in der klinischen Praxis und Forschung zu sein.

Treatment Landscape of Targeted Therapies in Oncology: Challenges and Opportunities provides up-to-date knowledge on antitumor-targeted therapies and immunotherapy. The book's chapters are written by researchers dynamically working/focusing on cancer treatment. The content is designed to help those who are new to the field (beginners) and various specialized scientists and researchers involved in cancer research. For decades, the hallmark of cancer treatment has been conventional chemotherapy. But with the rapid increase in our understanding of the immune system, more and more small molecules, peptides, recombinant antibodies, vaccines and cellular therapeutic modalities are being applied to manipulate the immune response for cancer treatment. - Covers basic concepts and updated knowledge of the latest anticancer drugs - Brings comprehensive understanding of the treatment of cancer in context of immunotherapy and targeted therapy - Introduces the latest drugs and advancements in the treatment of cancer - Includes detailed diagrams to give insight to the mechanisms of action

Post-transcriptional Gene Regulation in Human Disease, a new volume in the Translational Epigenetics book series, offers a thorough overview and discussion of post-transcriptional genetic control mechanisms and their roles across various pathologies and human developmental outcomes, along with regulatory mechanisms targeted for therapeutic approaches. The book is broadly divided in two parts: early chapters describe the basics of post-transcriptional gene regulation, associated epigenetic mechanisms, the role of RNA binding proteins, the evolution of post-transcriptional gene regulation, and methods to study these mechanisms. The second half of the book includes deeper discussion of post-transcriptional gene regulation across specific diseases and therapeutics targets. Various post-transcriptional events, including alternative splicing and polyadenylation, mRNA stability, and miRNAs and their involvement in the disease progression, are examined in detail. - Includes full-color imagery illustrating key concepts and post-transcriptional disease processes, as well as descriptions of methods for studying post-transcriptional gene regulation - Presents fundamental knowledge, molecular and biochemical mechanisms, and recent findings in concise and easily understandable formats - Features a summary and conclusion at the end of each chapter

Somatic genome manipulation is required when a sexual crossing approach cannot be used in breeding or genetic treatment of an individual organism. Examples can include gene- or cell-therapy of a person to correct disease, genetic improvement of vegetatively propagated plants, and genetic replacement of cytoplasm without significantly modifying the nuclear genome. The advantage of somatic genome manipulation is maintenance of the general genotype while correcting one or more traits. Somatic genome manipulation is also an option for genetic improvement of sexually propagated plants in polyploidy breeding or in overcoming issues of sexual incompatibility. Recent novel technologies in somatic genome manipulation are developing quickly but much of this literature is fragmented and difficult or inconvenient to access. This book represents the first attempt to assemble updated reviews, detailed protocols, and their applications in all fields in which somatic genome manipulation has thrived. This is a truely one-of-a-kind work that brings together the most important and relevant advances in somatic genome manipulation in plants, algae, microorganisms, humans and animals, and demonstrates where the science interacts and where it diverges. The chapters are written by experts on the topic with ready-to-use protocols that were originally developed or adapted from the literature in their laboratories. We expect that this book will be useful for students, researchers, and teachers in both plant and animal research as a resource for the latest information on somatic genome manipulation and for its useful laboratory methods.

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

The fourth edition of this bestseller presents extensive background and basic information, state-of-the-art technologies, important achievements, and lingering challenges in the fields of gene and cell therapies. It continues to provide the most comprehensive coverage of these fields in one volume. Some chapters have expanded introductions, making the book even more suitable for classroom use. This edition also offers more material on the contributors'research efforts as well as current technologies, disease targets, and clinical applications.

An exploration of the raw power of genetic material to refashion itself to any purpose… Virtually all organisms contain multiple mobile DNAs that can move from place to place, and in some organisms, mobile DNA elements make up a significant portion of the genome. Mobile DNA III provides a comprehensive review of recent research, including findings suggesting the important role that mobile elements play in genome evolution and stability. Editor-in-Chief Nancy L. Craig assembled a team of multidisciplinary experts to develop this cutting-edge resource that covers the specific molecular mechanisms involved in recombination, including a detailed structural analysis of the enzymes responsible presents a detailed account of the many different recombination systems that can rearrange genomes examines the tremendous impact of mobile DNA in virtually all organisms Mobile DNA III is valuable as an in-depth supplemental reading for upper level life sciences students and as a reference for investigators exploring new biological systems. Biomedical researchers will find documentation of recent advances in understanding immune-antigen conflict between host and pathogen. It introduces biotechnicians to amazing tools for in vivo control of designer DNAs. It allows specialists to pick and choose advanced reviews of specific elements and to be drawn in by unexpected parallels and contrasts among the elements in diverse organisms. Mobile DNA III provides the most lucid reviews of these complex topics available anywhere.

Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. - Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application - Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use - Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials

This volume of Methods in Enzymology looks at Gene Transfer Vectors for Clinical Application. The chapters provide an invaluable resource for academics, researchers and students alike. With an international board of authors, this volume covers such topics as General principles of retrovirus vector design, Chronic granulomatous disease (CGD), Gene therapy for blindness, and Retrovirus genetic strategy and vector design. - Chapters provide an invaluable resource for academics, researchers and students alike - International board of authors - This volume covers such topics as general principles of retrovirus vector design, chronic granulomatous disease (CGD), gene therapy for blindness, and retrovirus genetic strategy and vector design

A renaissance of virus research is taking centre stage in biology. Empirical data from the last decade indicate the important roles of viruses, both in the evolution of all life and as symbionts of host organisms. There is increasing evidence that all cellular life is colonized by exogenous and/or endogenous viruses in a non-lytic but persistent lifestyle. Viruses and viral parts form the most numerous genetic matter on this planet.

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

Animal Cell Biotechnology: Methods and Protocols, Second Edition constitutes a comprehensive manual of state-of-the-art and new techniques for setting up mammalian cell lines for production of biopharmaceuticals, and for optimizing critical parameters for cell culture considering the whole cascade from lab to final production. The volume is divided into five parts that reflect the processes required for different stages of production. In Part I, basic techniques for establishment of production cell lines are addressed, especially transduction techniques, cells for gene therapy and antibody production. Part II addresses basic cultivation techniques, such as microcarrier culture and encapsulation. Part III covers cell characterization and analysis, including flow cytometric applications, NMR-based techniques, and biochemical and cytometric techniques. Part IV details cultivation techniques, such as disposable bioreactors, hollow fiber cell culture, fixed bed reactors, and configuration of bioreactors. Part V covers downstream techniques such as membrane filtration techniques, while Part VI describes special applications, including retroviral vectors. Animal Cell Biotechnology: Methods and Protocols, Second Edition provides a compendium of techniques for scientists in industrial and research laboratories that use mammalian cells for biotechnology purposes.

Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis.• Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells• Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis• Includes'insider'information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use

This reference is completely revised and expanded to reflect the most critical studies, controversies, and technologies impacting the medical field, including probing research on lentivirus, gutless adenovirus, bacterial and baculovirus vectors, retargeted viral vectors, in vivo electroporation, in vitro and in vivo gene detection systems, and all inducible gene expression systems. Scrutinizing every tool, technology, and issue impacting the future of gene and cell research, it is specifically written and organized for laymen, scholars, and specialists from varying backgrounds and disciplines to understand the current status of gene and cell therapy and anticipate future developments in the field.

This volume examines the advantages and limitations of the major gene delivery systems and offers guidelines to select the most appropriate viral or synthetic delivery system for specific therapeutic applications. It discusses advances in the design, optimization, and adaptation of gene delivery systems for the treatment of cancerous, cardiovascula

The first unequivocal success for Gene Therapy was reported in April 2000 for X-SCID patients. Pioneering stem cell/gene therapy clinical trials are the focus of this book. Therapy successes such as the X-SCID trial and improved ADA-SCID ones are presented together with pioneering angio/vasculogenic clinical trials mediated either by transient gene therapy or emerging autologous stem cell transplantation. Highlights also include 1) promises of the breakthrough combination of stem cell- and transient gene-therapy, 2) gene therapy trials for neurodegenerative disease on non-human primates where long-term gene therapy might involve brain stem cells, and 3) the first clinical trial with non-invasive monitoring of therapeutic gene expression as a prospective conclusion.

This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. - Discusses methods currently used in preclinical and clinical gene therapy - Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease

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

Significant advances in gene transfer technology and the completion of sequencing the human genome offer much renewed hope that gene therapy will provide novel approaches to the treatment of inherited and acquired diseases. In this fully updated and revised 2nd edition of the warmly received Gene Therapy Protocols, leading experts from academic and industrial laboratories around the world detail their most effective viral and nonviral methods of gene transfer, as well as discuss their applications in different organ systems. The methods range from those in which new molecular conjugates show great promise for targeting targeting gene transfer and regulating transgene expression, to those used in such exciting applications as the delivery of therapeutic proteins, vaccination, and tissue engineering. In addition, there are completely revised methods based on retroviruses and adenoviruses, as well as new and promising methods based on lentiviruses and adeno-associated viruses. The quantitative aspects of gene therapy are strongly emphasized, and the book reflects the many significant recent improvements in gene transfer efficiency, gene regulation, and vector production. Up-to-date and highly practical, Gene Therapy Protocols, 2nd Edition, offers a rich compilation of the revolutionary advances that have recently occurred in gene transfer technology, with each article providing proven step-by-step laboratory procedures that enable successful therapeutic application. --

In Gene Targeting Protocols, a panel of innovative investigators present in readily reproducible detail the latest techniques for gene replacement, gene knockout, and gene repair in both animals and plants. These expert researchers review the best gene transfer vehicles (liposomes, PEI, and EPD), discuss the parameters that affect vector uptake, and detail successful methods for gene targeting (Cre-ox, and adeno and adeno-associated viruses). The methods include two fundamentally different approaches to gene targeting- one using a fusion protein to deliver a vector to a virus for possible therapeutic effect, and one entailing an episomal-based Epstein-Barr vector that can modulate the chromatin assembly process, and thus overcome a serious barrier to therapeutic gene targeting. The book also has several techniques for using oligonucleotides in gene targeting, a methodology that may revolutionize the entire field. Innovative and wide ranging, Gene Targeting Protocols offers today's researchers robust and proven gene-targeting techniques that are essential to understanding and regulating biological processes at the genetic level.

This book combines an up-to-date summary of how best to genetically engineer viruses with an overview of basic virology. This unique combination makes it an invaluable research tool for virologists and molecular biologists seeking to exploit viruses for a range of applications. Written by highly respected authors, the book also provides comparisons to and guidelines for the use of viruses in difference applications.

Neuroscience Perspectives provides multidisciplinary reviews of topics in one of the most diverse and rapidly advancing fields in the life sciences.Whether you are a new recruit to neuroscience, or an established expert, look to this series for'one-stop'sources of the historical, physiological, pharmacological, biochemical, molecular biological and therapeutic aspects of chosen research areas.The recent development of Gene Therapy procedures which allow specific genes to be delivered to human patients who lack functional copies of them is of major therapeutic importance. In addition such gene delivery methods can be used in other organisms to define the function of particular genes. These studies are of particular interest in the nervous system where there are many incurable diseases like Alzheimer's and Parkinson's diseases which may benefit from therapies of this kind. Unfortunately gene delivery methods for use in the nervous system have lagged behind those in other systems due to the fact that the methods developed in other systems are often not applicable to cells like neurons which do not divide. This book discusses a wide range of methods which have now been developed to overcome these problems and allow safe and efficient delivery of particular genes to the brain. Methods discussed include virological methods, physical methods (such as liposomes) and the transplantation of genetically modified cells. In a single volume therefore this book provides a complete view of these methods and indicates how they can be applied to the development of therapies for treating previously incurable neurological disorders.