Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy — though still in its infancy — remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ('vector') to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession.This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system. Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

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.

Advances in genetics, molecular biology and gene delivery technologies in recent years have led to new gene therapy strategies for treatment of a variety of diseases. This book gives a comprehensive overview of the present status and future directions of gene delivery systems and therapeutic strategies for the clinical application of gene therapy in cancer, cardiovascular and central nervous system diseases. Stem cell-based therapies and gene expression regulatory systems as novel platform technologies for various gene therapy applications are also discussed. Leading experts give excellent overviews of basic molecular aspects and clinical applications in this new emerging biomedical field.

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.

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.

This second edition of a very successful text reflects the tremendous pace of human genetics research and the demands that it places on society to understand and absorb its basic implications. The human genome has now been officially mapped and the cloning of animals is becoming a commonplace scientific discussion on the evening news. Join authors Julia Richards and Scott Hawley as they examine the biological foundations of humanity, looking at the science behind the sensation and the current and potential impact of the study of the genome on our society. The Human Genome, Second Edition is ideal for students and non-professionals, but will also serve as a fitting guide for the novice geneticist by providing a scientific, humanistic, and ethical frame of reference for a more detailed study of genetics. New in this edition: · 60% new material, including data from the Human Genome Project and the latest genetics and ethics discussions · Several new case studies and personal stories that bring the concepts of genetics and heredity to life · Simplified treatment of material for non-biology majors · New full-color art throughout the text · New co-author, Julia Richards, joins R. Scott Hawley in this revision

The idea of human gene therapy was accepted by the medical community and society at large long before believable clinical benefits began to emerge. In this book, some of the fields most distinguished contributors chronicle the evolution of this momentous direction for medicine, illustrating how imaginative concepts shaped the development of technologies and brought the daring new idea to its current position of imminent practical success. This is a book designed to endure as clinical advances accumulate, a clear-eyed work of reference that will anchor the further development of this revolution in therapy. It is an essential addition to libraries of clinical medicine, biotechnology, and public policy, and a resource that no laboratory investigator with an interest in the biology of gene transfer should be without.