This book delivers a collection of organized and succinct reviews in the field of therapeutic genetic medicine presented by a carefully selected group of top experts. Each chapter focuses on a single, current topic and explains issues in the development of genetic treatments, critical challenges and strategies for implementing them, and future directions in research and translational applications. The book is complemented with uniformly designed illustrations and a supplementary Web site with a PowerPoint presentation for educators.
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.
Publisher: Karger Medical and Scientific Publishers
ISBN: 9783805590358
Category: Medical
Page: 127
View: 989
This volume reviews current concepts and recent research findings of the application of gene therapy to the inner ear. Various forms of gene therapy, disorders that are potential targets for gene therapy, techniques for gene delivery, and mechanisms of gene targeting, and also obstacles that have yet to be overcome are discussed.
Gene Therapy is expected to revolutionize the practice of medicine at the turn of the third Millennium. Therapeutic/prophylactic benefits should arise from both gene transfer and gene repair/inactivation protocols devised for patient's somatic cells. Gene expression cassettes, designed for the production of therapeutic proteins and non-coding RNA, are thus experimented together with emerging gene repair/inactivation techniques on a variety of inherited, acquired and infectious/parasitic diseases, including complex neuro-degenerative processes. This book presents a collection of chapters on the main aspects of Gene Therapy, some of which have already been treated in the past, and updates and further develops the current survey. It is aimed at understanding why Human Gene Therapy is likely to be a medical breakthrough, although definitive clinical success still needs time to accomplish. The contributions focus on both technical/biomedical concepts and on experimental/clinical data that appear to confer potential universality to Gene Therapy. This book does not claim to provide an exhaustive review of the pathologies, which are currently approached with Gene Therapy. The presentation of current and emerging approaches, together with problems and tentative issues aims to serve as a booster for the development of new applications in every field of medicine and will, therefore, be of value and interest to researchers in this exciting field.
Gene therapy for inflammatory diseases is a new , burgeoning field of medicine. Edited by the undisputed pioneers of this area of research, this volume is the first devoted to its topic. It contains thirteen chapters, each written by leaders in their respective fields, that summarize the state of the art in developing novel, gene based treatments for inflammatory diseases. As well as providing an introduction to the basic concepts of gene therapy and the use of naked DNA approaches, the book describes the advances that have been made in applying them to arthritis, lupus, multiple sclerosis, diabetes, Sjogren`s syndrome and transplantation.One chapter is devoted to discussing the first human clinical trials that apply gene therapy to the treatment of an inflammatory disease. As well as providing novel therapeutic approaches, gene therapy facilitates the development of new and improved animal models of disease; a chapter describing these advances is also included. As an up-to-date, timely book written by th
Publisher: Karger Medical and Scientific Publishers
ISBN: 9783805590662
Category: Medical
Page: 138
View: 394
Genetically modified organisms (GMO) raise societal, political and ethical concerns. They inspire strong resistance or, conversely, enthusiastic assent. The aim of this publication is to give an overview of genetic engineering, starting with the history of the discovery of restriction enzymes continuing with technical aspects of transgenesis to its applications in research and ethical considerations. Be it the use of single engineered cells or GMO, these applications cover a broad array, ranging from disease-oriented research (but not only), to the promising perspectives of gene therapy. Historical and technical aspects give insights into the problems inherent to the creation of GMO, and illustrate the links and limits between genetic engineering, GMOs and gene therapy. A summary article in English and French structures the links between the different chapters and concepts.Scientists interested in genetic engineering of single cells or animal models, as well as in gene therapy, will find an up-to-date review on the use and perspectives of transgenesis. However, this publication is also recommended to the public interested in the definition of GMO, which encompasses a much broader array than the genetically modified crops covered by media.
Genetic engineering has become a very important field of study with its growing applications in biological engineering, medical science and other related fields. This book brings forth some of the most innovative concepts and elucidates the unexplored aspects of genetic engineering such as advanced artificial synthesis of genes, gene therapy, genetic cloning and applications of genetic engineering in various fields like agriculture, medical and biomedical science, etc. It will also provide interesting topics for research which readers can take up.
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.
“... an excellent book... achieves all of its goals with style, clarity and completeness... You can see the power and possibilities of molecular genetics as you read...” –Human Genetics "This volume hits an outstanding balance among readability, coverage, and detail." –Biochemistry and Molecular Biology Education Rapid advances in a collection of techniques referred to as gene technology, genetic engineering, recombinant DNA technology and gene cloning have pushed molecular biology to the forefront of the biological sciences. This new edition of a concise, well-written textbook introduces key techniques and concepts involved in cloning genes and in studying their expression and variation. The book opens with a brief review of the basic concepts of molecular biology, before moving on to describe the key molecular methods and how they fit together. This ranges from the cloning and study of individual genes to the sequencing of whole genomes, and the analysis of genome-wide information. Finally, the book moves on to consider some of the applications of these techniques, in biotechnology, medicine and agriculture, as well as in research that is causing the current explosion of knowledge across the biological sciences. From Genes to Genomes: Concepts and Applications of DNA Technology, Second Edition includes full two-colour design throughout. Specific changes for the new edition include: Strengthening of gene to genome theme Updating and reinforcing of material on proteomics, gene therapy and stem cells More eukaryotic/mammalian examples and less focus on bacteria This textbook is must-have for all undergraduates studying intermediate molecular genetics within the biological and biomedical sciences. It is also of interest for researchers and all those needing to update their knowledge of this rapidly moving field.
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.
In the field of medicine, the emerging frontier of gene therapy holds great potential in the treatment of many human diseases and conditions. It is characterized by the intent of direct therapeutic effect and the precision of the procedure. In this model, nucleic acid is delivered into a patient's cells which results in the replacement or knocking out of genes that cause a disease. Gene therapy can be classified into somatic and germline gene therapy. In somatic cell gene therapy, therapeutic genes are transferred into the somatic cells of the body and the resulting modifications affect the patient only, without being passed on to the offspring. In germline gene therapy, the germline itself is modified due to the introduction of functional genes into the genome of germ cells. Such changes are inherited by generations of individuals. This book covers in detail some existing theories and innovative concepts revolving around gene therapy. Some of the diverse topics covered in this book address the varied biomedical applications of gene therapy. As this field is emerging at a fast pace, this book will help the readers to better understand the advanced concepts of this field.
