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| gene therapy books free download: Gene Therapy Mauro Giacca, 2010-11-01 I entered the gene therapy field in the mid-1990s, being fascinated by the immense potential of genes as drugs for the treatment of human disease. Since then, I have experienced the ups and downs of this discipline, and tried to contribute with my work and that of my laboratory to the development of innovative approaches to the treatment of cardiovascular disorders. During these years, I have had several opp- tunities to speak on gene therapy at lectures and academic lessons, and have often noticed that the field is very attractive to scientists of all disciplines. However, as yet no comprehensive book on the subject has been published. Indeed, most books in the field are either a collection of gene transfer laboratory protocols or deal with the subject in a rather superficial manner. Hence the idea to write a gene therapy textbook that is broad and comprehensive, but at the same time provides sufficient molecular and clinical detail to be of interest to students, professors, and specialists in the various disciplines that contribute to gene therapy. I have tried to keep the language plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, whenever technical descriptions are required, they are provided. |
| gene therapy books free download: Gene and Cell Therapy: Biology and Applications Giridhara R. Jayandharan, 2018-09-12 Recent advances in stem cell biology, nanotechnology and gene therapy have opened new avenues for therapeutics. The availability of molecular therapeutics that rely on the delivery of DNA, RNA or proteins, harnessing enhanced delivery with nanoparticles, and the regenerative potential of stem cells (adult, embryonic or induced pluripotent stem cells) has had a tremendous impact on translational medicine. The chapters in this book cover a range of strategies for molecular and cellular therapies for human disease, their advantages, and central challenges to their widespread application. Potential solutions to these issues are also discussed in detail. Further, the book addresses numerous advances in the field of molecular therapeutics that will be of interest to the general scientific community. Lastly, the book provides specific examples of disease conditions for which these strategies have been transferred to the clinic. As such, it will be extremely useful for all students, researchers and clinicians working in the field of translational medicine and molecular therapeutics. |
| gene therapy books free download: A Guide to Human Gene Therapy Roland W. Herzog, Sergei Zolotukhin, 2010 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 |
| gene therapy books free download: A Handbook of Gene and Cell Therapy Clévio Nóbrega, Liliana Mendonça, Carlos A. Matos, 2021-07-12 This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field. |
| gene therapy books free download: Lentiviral Vectors and Gene Therapy David Escors, Karine Breckpot, Frederick Arce, Grazyna Kochan, Holly Stephenson, 2012-03-22 Gene therapy was conceived during the early and mid part of the 20th century. At first, it was considered a revolutionary biomedical procedure, which could potentially cure any disease for which the molecular bases were understood. Since then, gene therapy has gone through many stages and has evolved from a nearly unrealistic perspective to a real life application. Clinical efficacy in humans was demonstrated at the beginning of this century after its successful application in small-scale clinical trials to cure severe immunodeficiency in children. However, their successes were overshadowed some time later by the occurrence of vector-related leukaemia in a number of treated children. It is in this context that lentiviral vectors have appeared, with improved efficiency and, possibly, increased biosafety. Very recently, the first clinical trials with lentivectors have been carried out with some success. This Brief firstly defines gene therapy, and places lentivectors within this fascinating therapeutic strategy. Then follows a comprehensive description of the development of retroviral and lentiviral vectors and how to specifically target distinct cell types and tissues. The authors also discuss the application of lentivector gene therapy for the treatment of cancer and autoimmune diseases, ending with the application of lentivectors in human gene therapy clinical trials. |
| gene therapy books free download: Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders Nicola Brunetti-Pierri, 2017-05-22 In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields. |
| gene therapy books free download: Regulatory Aspects of Gene Therapy and Cell Therapy Products Maria Cristina Galli, Mercedes Serabian, 2015-09-15 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. |
| gene therapy books free download: Stem Cell Biology and Gene Therapy Peter J. Quesenberry, Gary S. Stein, Bernard G. Forget, Sherman M. Weissman, 1998-09-10 STEM CELL BIOLOGY AND GENE THERAPY Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide. Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies. This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including: * Fundamental regulatory mechanisms that operate in stem cells * Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy * Delivery systems for therapeutic genes, including an overview of the most promising vectors * Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine. |
| gene therapy books free download: An Introduction to Molecular Medicine and Gene Therapy Thomas F. Kresina, 2001 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. |
| gene therapy books free download: Cell Therapy Adrian Gee, 2009-09-18 Cell Therapy: cGMP Facilities and Manufacturing is the source for a complete discussion of facility design and operation with practical approaches to a variety of day-to-day activities, such as staff training and competency, cleaning procedures, and environmental monitoring. This in-depth book also includes detailed reviews of quality, the framework of regulations, and professional standards. It meets a previously unmet need for a thorough facility-focused resource, Cell Therapy: cGMP Facilities and Manufacturing will be an important addition to the cell therapy professional’s library. Additional topics in Cell Therapy: cGMP Facilities and Manufacturing...Standard operating procedures - Supply management - Facility equipment - Product manufacturing, review, release and administration - Facility master file. |
| gene therapy books free download: Precision Medicine, CRISPR, and Genome Engineering Stephen H. Tsang, 2017-11-11 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. |
| gene therapy books free download: Viral Vectors for Gene Therapy Otto-Wilhelm Merten, Mohamed Al-Rubeai, 2011-05-19 The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular BiologyTM series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective. |
| gene therapy books free download: Life with Epidermolysis Bullosa (EB) Jo-David Fine, Helmut Hintner, 2009-04-02 Epidermolysis bullosa (EB) is a heterogenous group of genodermatoses characterized by the formation of blisters and erosions on skin and mucous membrans from birth on. The cause are mutations in the genes of structural proteins of the junction between epidermis and dermis. This book deals with the treatment of this skin disease itself and its many extracutaneous complications. There is no previous book which has been focused on the therapy and it will be based on evidence-based data derived from the world ́s largest cohort of inherited EB-patients, the American EB Registry. An important chapter will discuss gene therapy in hereditary EB which has been recently successfully performed within a localized skin site on a single EB patient as a proof-of-principle test. Given its unique collective contents, the monograph will provide the primary source for clinical informations of this oftentimes severe multiorgan disease. |
| gene therapy books free download: Gene and Cellular Immunotherapy for Cancer Armin Ghobadi, John F. DiPersio, 2022-01-01 Clinical and preclinical exploration of gene and cellular immunotherapy have seen rapid growth and interest with the development and approval of five Chimeric Antigen Receptor T-cell (CAR-T) products for lymphoma and myeloma and one Bispecific T-Cell Engager (BiTE) for acute lymphoblastic leukemia (ALL). These advances have dramatically improved the management of patients with relapsed refractory lymphoma, myeloma and leukemia. Gene and Cellular Immunotherapy for Cancer offers readers a comprehensive review of current cellular and gene-based immunotherapies. Divided into eighteen cohesive chapters, this book provides an in-depth and detailed look into cellular-based immunotherapies including CAR-T, TCR-T, TIL, Viral CTLs, NK cells in addition to T/NK cell engagers, focusing on their historical perspectives, biology, development and manufacturing, toxicities and more. Edited by two leading experts on gene and cellular immunotherapy, the book will feature chapters written by a diverse collection of recognized and up-and-coming experts and researchers in the field, providing oncologists, immunologists, researchers and clinical and basic science trainees with a bench to bedside view of the latest developments in the field. |
| gene therapy books free download: Lentivirus Gene Engineering Protocols Maurizio Federico, 2008-02-03 Cell gene engineering is emerging as a field with outstanding impact, not only in medicine/biology, but also, and perhaps most importantly, in agriculture and in all those food sciences involved in the fight against world hunger. Lentivirus vector-based technologies represent the last frontier in the development of powerful and reliable methods for both in vitro and in vivo gene transfer in eukaryotic animal cells. Although the design of lentivirus vectors is closely reminiscent of those already successfully applied to the construction of oncoretroviral vectors, some unique features, e.g., the ef- ciency in transducing both postmitotic and stem cells, render the use of lentivirus vectors invaluable. It has been a great pleasure to edit Lentivirus Gene Engineering Pro- cols, owing in part to the high level of enthusiasm that the authors dem- strated in contributing to this book. The fact that so many outstanding scientists engaged in lentivirus vector research have provided articles renders it so- thing more than a technical handbook. In addition to detailed descriptions of the most innovative methodologies, the reader may find very informative ov- views concerning both theoretical and practical aspects of the origin and the development of diverse lentivirus vector types. This, in my opinion, rep- sents a unique added value of this volume, which should help our work resist the passage of time, to which books such as this are particularly sensitive. |
| gene therapy books free download: Textbook of Gene Therapy Kewal K. Jain, 1998 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. |
| gene therapy books free download: Genetic Twists of Fate Stanley Fields, Mark Johnston, 2013-02-08 How tiny variations in our personal DNA can determine how we look, how we behave, how we get sick, and how we get well. News stories report almost daily on the remarkable progress scientists are making in unraveling the genetic basis of disease and behavior. Meanwhile, new technologies are rapidly reducing the cost of reading someone's personal DNA (all six billion letters of it). Within the next ten years, hospitals may present parents with their newborn's complete DNA code along with her footprints and APGAR score. In Genetic Twists of Fate, distinguished geneticists Stanley Fields and Mark Johnston help us make sense of the genetic revolution that is upon us. Fields and Johnston tell real life stories that hinge on the inheritance of one tiny change rather than another in an individual's DNA: a mother wrongly accused of poisoning her young son when the true killer was a genetic disorder; the screen siren who could no longer remember her lines because of Alzheimer's disease; and the president who was treated with rat poison to prevent another heart attack. In an engaging and accessible style, Fields and Johnston explain what our personal DNA code is, how a few differences in its long list of DNA letters makes each of us unique, and how that code influences our appearance, our behavior, and our risk for such common diseases as diabetes or cancer. |
| gene therapy books free download: Plasmids for Therapy and Vaccination Martin Schleef, 2001 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. |
| gene therapy books free download: Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies (Second Edition) Daniel Scherman, 2019-07-16 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 for day-to-day cell biology experiments and swift development of gene therapy, which is already revolutionizing medicine. In this advanced textbook, more than 30 leading scientists come together to explore these topics.This educational introduction provides the background material needed to further explore the subject as well as relevant research literature. It is an invaluable resource to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide or develop a gene therapy strategy. The second edition's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome. |
| gene therapy books free download: Gene Therapy - Principles and Challenges , 2015 |
| gene therapy books free download: Genome Editing in Neurosciences Rudolf Jaenisch, Feng Zhang, Fred Gage, 2018-08-11 This book is open access under a CC BY 4.0 license. CRISPR-Cas9 is a rapid, efficient, versatile and relatively cheap method for dissecting the molecular pathways that are the basis of life, as well as for investigating and potentially rectifying faults in these pathways that result in disease. This book reviews how CRISPR-Cas9 and other genome editing techniques are advancing our understanding of development and function in the nervous system, uncovering the molecular causes of neurological disorders and providing tools for gene therapy. |
| gene therapy books free download: The Human Genome in Health and Disease Tore Samuelsson, 2019-02-07 The human genome is a linear sequence of roughly 3 billion bases and information regarding this genome is accumulating at an astonishing rate. Inspired by these advances, The Human Genome in Health and Disease: A Story of Four Letters explores the intimate link between sequence information and biological function. A range of sequence-based functional units of the genome are discussed and illustrated with inherited disorders and cancer. In addition, the book considers valuable medical applications related to human genome sequencing, such as gene therapy methods and the identification of causative mutations in rare genetic disorders. The primary audiences of the book are students of genetics, biology, medicine, molecular biology and bioinformatics. Richly illustrated with review questions provided for each chapter, the book helps students without previous studies of genetics and molecular biology. It may also be of benefit for advanced non-academics, which in the era of personal genomics, want to learn more about their genome. Key selling features: Molecular sequence perspective, explaining the relationship between DNA sequence motifs and biological function Aids in understanding the functional impact of mutations and genetic variants Material presented at basic level, making it accessible to students without previous studies of genetics and molecular biology Richly illustrated with questions provided to each chapter |
| gene therapy books free download: Gene Biotechnology William Wu, Helen H. Zhang, Michael J. Welsh, Peter B. Kaufman, 2016-04-19 Covering state-of-the-art technologies and a broad range of practical applications, the Third Edition of Gene Biotechnology presents tools that researchers and students need to understand and apply today's biotechnology techniques. Many of the currently available books in molecular biology contain only protocol recipes, failing to explain the princ |
| gene therapy books free download: The Ethics of Human Gene Therapy LeRoy Walters, Julie Gage Palmer, 1997 The authors of this absorbing new book describe the science of gene therapy in terms easily accessible to the non-specialist, and focus on the controversial ethical and public policy issues surrounding human interventions in human heredity. After a brief survey of the structure and functions of DNA, genes, and cells, Walters and Palmer discuss three major types of potential genetic intervention: somatic cell gene therapy, germ line gene therapy, and genetic enhancements. They start with the current techniques of gene addition, using non-reproductive (somatic) cells in an effort to cure or treat disease. Next they address the technical problems and moral issues facing attempts to prevent disease through genetically modifying early human embryos or sperm and egg cells. These changes would be passed on to future generations. Chapter 4, in many ways the most original part of this volume, confronts the issue of employing genetic means to improve human abilities and appearance. Depending on the techniques employed, such enhancements could affect not only the individuals receiving the intervention but their offspring as well. Three types of genetic enhancements are considered: physical alterations to improve size, reduce the need for sleep, and decelerate aging; intellectual enhancements of memory and general cognitive ability; and moral enhancements for control of violently aggressive behavior. The authors maintain that genetic modifications should be evaluated individually rather than be condemned in principle or as a group. The final chapter summarizes the public review process that human gene therapy proposals have been undergoing in the United States since 1990. Five appendices, providing technical background information along with a complete list of questions raised in the national public review process, supplement the discussion. |
| gene therapy books free download: Gene and Cell Therapy Nancy Smyth Templeton, 2003-12-17 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. |
| gene therapy books free download: Textbook of Hemophilia Christine A. Lee, Erik E. Berntorp, W. Keith Hoots, 2008-04-15 The only up-to-date definitive reference source onhemophilia This book is an invaluable resource that provides an overview ofall aspects of the care of patients with haemophilia. Covering how to assess both bleeding children and adults,Haemophilia A and B, molecular basis of the disease, the role offactors in coagulation, epidemiology, pharmacokinetics, andtreatment of inhibitors. There will also be a section onmusculoskeletal aspects of haemophilia as well as newerdevelopments such as gene therapy and rare bleedingdisorders. Textbook of Hemophilia is ideal for: Trainees and residents in hematology Hematologists in practice Specialists working in thrombosis and hemostasis as well astransfusion medicine Why Buy This Book? The only up-to-date definitive reference source onhemophilia Essential for all those managing hemophilia patients Detailed guidance on assessment, diagnosis, management andtreatment Advice for everyday clinical questions Edited by three of the world’s leading experts onhemophilia |
| gene therapy books free download: Decoding the Language of Genetics David Botstein, 2015 This is a book about the conceptual language of genetics. There is a need for special words and terms to deal with some of the essential abstractions in genetics; these are the focus of this book. It is intended to help readers with diverse interests and experience to think about genetic analysis in a more sophisticated and creative way.--Publisher information. |
| gene therapy books free download: Exploring Novel Clinical Trial Designs for Gene-Based Therapies National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Forum on Regenerative Medicine, 2020-08-27 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. |
| gene therapy books free download: Emerging Medical Technologies Gennady Ermak, 2015-08-27 The book provides an overview of new technologies in medicine. It defines each technology and gives an account of its history, and the principles, technical problems, and applications as well as controversies involved. The goal of the book is to provide a comprehensive overview of the emerging medical technologies in a simple way that is understandable to non-specialists.Biomedical science and medical technologies are developing at such a fast pace that it is hard to follow even for specialists. In addition, they have become more and more complex. This book provides readers with a basic understanding of these technologies and helps them to keep up with new developments. The value of the book is that it is comprehensive and yet concise, which makes the learning process fast and easy. |
| gene therapy books free download: Bio-Carrier Vectors Kumaran Narayanan, 2022-01-01 This detailed book explores techniques used in research efforts to adopt and combine the best capabilities from natural and artificial vector systems to assemble improved delivery technologies aimed for specific applications. The collection discusses vectors other than the traditional viral and non-viral systems and explores ideas for blending the best features of bacteria, nanoparticles, peptides, and hybrid systems for the delivery of biomaterials into cells, as well as a chapter on the application of shotgun proteomics and mass spectrometry as a tool to analyze the proteomic profile changes in cells that result from these interventions. Written in the highly successful Methods in Molecular Biology format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Bio-Carrier Vectors: Methods and Protocols serves as an ideal guide for researchers working toward harnessing the power of multiple vectors in gene and drug delivery. |
| gene therapy books free download: The ABCs of Gene Cloning Dominic Wong, 2007-12-31 Clear and concise, this easy-to-use text offers an introductory course on the language of gene cloning, covering microbial, plant, and animal systems. The essential concepts in biology relevant to the understanding of gene cloning are presented in a well-organized and accessible manner. This updated version of the first edition is an invaluable book for nonscientists as well as scientists with little background knowledge in gene cloning, providing a wealth of information for anyone wishing to gain proficiency in reading and speaking the language of gene cloning. |
| gene therapy books free download: Cancer Gene Therapy David T. Curiel, Joanne T. Douglas, 2007-11-03 A complete introduction and guide to the latest developments in cancer gene therapy-from bench to bedside. The authors comprehensively review the anticancer genes and gene delivery methods currently available for cancer gene therapy, including the transfer of genetic material into the cancer cells, stimulation of the immune system to recognize and eliminate cancer cells, and the targeting of the nonmalignant stromal cells that support their growth. They also thoroughly examine the advantages and limitations of the different therapies and detail strategies to overcome obstacles to their clinical implementation. Topics of special interest include vector-targeting techniques, the lessons learned to date from clinical trials of cancer gene therapy, and the regulatory guidelines for future trials. Noninvasive techniques to monitor the extent of gene transfer and disease regression during the course of treatment are also discussed. |
| gene therapy books free download: Nanomedicine for Drug Delivery and Therapeutics Ajay Kumar Mishra, 2013-02-01 This book describes a broad area of nanomedicine which involves mainly applications, diseases, and diagnostics. The comprehensive coverage provides researchers, academics, and health specialists with a great tool, that includes techniques applicable to various uses. |
| gene therapy books free download: Gene Therapy Applications Chunsheng Kang, 2011-08-23 The aim of our book is to provide a detailed discussion of gene therapy application in human diseases. The book brings together major approaches: (1) Gene therapy in blood and vascular system, (2) Gene therapy in orthopedics, (3) Gene therapy in genitourinary system, (4) Gene therapy in other diseases. This source will make clinicians and researchers comfortable with the potential and problems of gene therapy application. |
| gene therapy books free download: Pharmaceutical Biotechnology Daan J. A. Crommelin, Robert D. Sindelar, 2002-11-14 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. |
| gene therapy books free download: Prenatal Gene Therapy Charles Coutelle, Simon N. Waddington, 2012-05-31 The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions. In Prenatal Gene Therapy: Concepts, Methods and Protocols, expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These include detailed protocols for vector production, for breeding and husbandry of the animal models, for the surgical procedures of gene delivery in large and small animals and for the methods of gene transfer analysis. Written in the highly successful Methods in Molecular BiologyTM series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls. Thorough and intuitive, Prenatal Gene Therapy: Concepts, Methods and Protocols seeks to aid scientists in the further study of prenatal disease and gene therapy projects beyond the scope of fetal medicine. |
| gene therapy books free download: Quickstart Molecular Biology Philip N. Benfey, 2014 This book is an introductory course in molecular biology for mathematicians, physicists, and engineers. It covers the basic features of DNA, proteins, and cells but in the context of recent technological advances, such as next-generation sequencing and high-throughput screens, and their applications. This enables readers to move rapidly from the b |
| gene therapy books free download: Bioelectronic Medicine Valentin A. Pavlov, 2019 Cold Spring Harbor perspectives in medicine. |
| gene therapy books free download: Ras and Cancer in the 21st Century Linda VanAelst, Frank McCormick, 2018 Ras proteins are key molecular switches in cell signaling pathways that, when activated, trigger cell growth and division. Mutations that produce abnormally active Ras proteins are common in human cancers, particularly those of the pancreas, lung, and colon. These cancers can be difficult to treat because Ras oncoproteins have long been considered 'undruggable.' Written and edited by experts in the field, this collection from Cold Spring Harbor Perspectives in Medicine covers the recent progress that has been made in understanding Ras biology, how Ras activation leads to tumorigenesis, and ways in which oncogenic Ras signaling may be targeted therapeutically. The contributors review the biochemical characteristics of the different Ras isoforms (HRAS, KRAS, and NRAS), their main effectors and signaling pathways, and the mutations that lead to their constitutive activation. Recent work showing that some Ras oncoproteins may be effectively inhibited by small molecules is covered, as is work on alternative targets (e.g., enzymes that catalyze posttranslational modifications of Ras) and inhibitors (e.g., nucleic acids). The authors also discuss how organoids and mouse models are being used to study tumor progression and therapeutic efficacy. This volume is therefore essential reading for all cancer biologists, cell and molecular biologists, and pharmacologists concerned with understanding and treating Ras-driven cancers. |
| gene therapy books free download: The Gene Book Sarah Adelaide Crawford, 2018-06-28 The Gene Book: Explorations in the Code of Life is designed to introduce undergraduate college students to foundational concepts in genetics. The text provides in-depth coverage of the essential principles of genetics, from Mendel to molecular gene therapy, and reads like a story, guiding readers through each of these areas in an interesting, engaging, and enlightening way. Milestone scientific discoveries introduce conceptual topics in each of the 10 chapters. The significance of each genetics paradigm is reinforced by the meaningful research context in which it is placed, whether the focus is single gene inheritance of disorders such as PKU and cystic fibrosis, or more complex genetic phenomena. Chromosomes, cell division, and cytogenetic disorders, including Down Syndrome and leukemia, are presented in a riveting historical context. In addition, the principles of molecular genetics are a major focus of this book. Students learn about the double helix, DNA replication, gene expression, mutation, natural selection, genomics, and the tools of molecular DNA analysis. Approachable and effective, The Gene Book is a highly readable comprehensive text on genetics principles designed to highlight essential concepts that make up their very core. The text is well suited to undergraduate genetics courses and can also be used as a primer for more advanced undergraduate and graduate courses in medical or molecular genetics. |
GeneCards - Human Genes | Gene Database | Gene Search
Mar 28, 2025 · GeneCards is a searchable, integrative database that provides comprehensive, user-friendly information on all annotated and predicted human genes. The knowledgebase …
Powerful Gene Set Analysis | GeneAnalytics - Your Gene Set, In …
GeneAnalytics enables researchers to identify compounds related to their gene sets, and further link to biochemical and pharmacological information about drugs, small molecules and …
BRCA1 Gene - GeneCards | BRCA1 Protein | BRCA1 Antibody
Mar 28, 2025 · The BRCA1 gene contains 22 exons spanning about 110 kb of DNA. The encoded protein combines with other tumor suppressors, DNA damage sensors, and signal transducers …
CTNNB1 Gene - GeneCards | CTNB1 Protein | CTNB1 Antibody
Mar 28, 2025 · Complete information for CTNNB1 gene (Protein Coding), Catenin Beta 1, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - The …
TNF Gene - GeneCards | TNFA Protein | TNFA Antibody
Mar 30, 2025 · This gene encodes a multifunctional proinflammatory cytokine that belongs to the tumor necrosis factor (TNF) superfamily. This cytokine is mainly secreted by macrophages.
TGFB1 Gene - GeneCards | TGFB1 Protein | TGFB1 Antibody
Mar 30, 2025 · Complete information for TGFB1 gene (Protein Coding), Transforming Growth Factor Beta 1, including: function, proteins, disorders, pathways, orthologs, and expression.
GeneAnalytics™ - Powerful Gene Set Analysis - GeneAnalytics
GeneAnalytics™ – GeneAnalytics supports analysis of gene sets with multiple genes, and enables matching of gene sets to tissues and cells within LifeMap Discovery.
PathCards - Human Biological Pathway Unification
Mar 28, 2025 · Human pathways were clustered into SuperPaths based on gene content similarity. Each PathCard provides information on one SuperPath which represents one or …
VEGFA Gene - GeneCards | VEGFA Protein | VEGFA Antibody
Mar 30, 2025 · Complete information for VEGFA gene (Protein Coding), Vascular Endothelial Growth Factor A, including: function, proteins, disorders, pathways, orthologs, and expression.
TP53 Gene - GeneCards | P53 Protein | P53 Antibody
Mar 30, 2025 · This gene encodes a tumor suppressor protein containing transcriptional activation, DNA binding, and oligomerization domains. The encoded protein responds to …
GeneCards - Human Genes | Gene Database | Gene Search
Mar 28, 2025 · GeneCards is a searchable, integrative database that provides comprehensive, user-friendly information on all annotated and predicted human genes. The knowledgebase …
Powerful Gene Set Analysis | GeneAnalytics - Your Gene Set, In …
GeneAnalytics enables researchers to identify compounds related to their gene sets, and further link to biochemical and pharmacological information about drugs, small molecules and …
BRCA1 Gene - GeneCards | BRCA1 Protein | BRCA1 Antibody
Mar 28, 2025 · The BRCA1 gene contains 22 exons spanning about 110 kb of DNA. The encoded protein combines with other tumor suppressors, DNA damage sensors, and signal transducers …
CTNNB1 Gene - GeneCards | CTNB1 Protein | CTNB1 Antibody
Mar 28, 2025 · Complete information for CTNNB1 gene (Protein Coding), Catenin Beta 1, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - The …
TNF Gene - GeneCards | TNFA Protein | TNFA Antibody
Mar 30, 2025 · This gene encodes a multifunctional proinflammatory cytokine that belongs to the tumor necrosis factor (TNF) superfamily. This cytokine is mainly secreted by macrophages.
TGFB1 Gene - GeneCards | TGFB1 Protein | TGFB1 Antibody
Mar 30, 2025 · Complete information for TGFB1 gene (Protein Coding), Transforming Growth Factor Beta 1, including: function, proteins, disorders, pathways, orthologs, and expression.
GeneAnalytics™ - Powerful Gene Set Analysis - GeneAnalytics
GeneAnalytics™ – GeneAnalytics supports analysis of gene sets with multiple genes, and enables matching of gene sets to tissues and cells within LifeMap Discovery.
PathCards - Human Biological Pathway Unification
Mar 28, 2025 · Human pathways were clustered into SuperPaths based on gene content similarity. Each PathCard provides information on one SuperPath which represents one or …
VEGFA Gene - GeneCards | VEGFA Protein | VEGFA Antibody
Mar 30, 2025 · Complete information for VEGFA gene (Protein Coding), Vascular Endothelial Growth Factor A, including: function, proteins, disorders, pathways, orthologs, and expression.
TP53 Gene - GeneCards | P53 Protein | P53 Antibody
Mar 30, 2025 · This gene encodes a tumor suppressor protein containing transcriptional activation, DNA binding, and oligomerization domains. The encoded protein responds to …
