An Introduction To Molecular Medicine And Gene Therapy

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.

Easy to read, yet comprehensive, this is the perfect introduction into the molecular basis of disease and the novel treatment options that have become available. The authors, Jens Kurreck and Cy Stein, have both long-standing teaching experience on the subject, one from a biologist's angle, the other with a medical background. Together, they have produced a modern textbook for courses in Molecular Medicine that incorporates modules from immunology to signaling, from virology to gene therapy, and the latest development in personalized medicine.

In the four years since the first edition of this book was published, the molecular revolution has continued. DNA has been named by Time maga zine as the Molecule of the Year, a Nobel Prize has been awarded to a young man for the invention of the polymerase chain reaction, and televi sion viewers have learned of the DNA fingerprint. Molecular technology in medicine is increasing. The availability of DNA probes for cancer suscepti bility is stressing our system of insurance, testing our ideas about medical ethics, and teaching us new things about cancer. In this edition, I have added a number of new sections, as well as a new chapter. New examples of molecular medicine have been added to demonstrate current applications of this technology. The basic concepts of molecular biology remain the basis for the first three chapters of the book. The excitement surrounding molec ular medicine that I mentioned in the preface to the first edition continues. It is now tinged with a touch of awe and a little bit of fear at the changes that recombinant DNA technology has brought to our society. v Preface to the First Edition This book describes the discoveries that have created a field called molecu lar medicine. The use of recombinant DNA technology in medical research and most recently in medical practice constitutes a revolutionary tool in our study of disease.

"This book should be useful to lecturers who teach senior undergraduates, graduate students, and students in the biomedical sciences in general. More globally, Greenwell and McCulley's book should encourage academicians of any stripe who for some time have been honing their lectures in a niche subject area to turn their courses into textbooks." –Biochemistry and Molecular Biology Education Molecular therapeutics refers to the developments in molecular biology that are focused on treating disease with new molecular-based drugs. By taking advantage of recent advances and increased understanding in the field of human genetics, this book provides essential background knowledge and key literature on a broad range of novel approaches and disciplines. These include making recombinant proteins, xenotransplantation gene therapy and therapeutic cloning. Molecular Therapeutics: 21st Century Medicine describes the techniques - including their associated benefits, problems, pitfalls - and discusses their applicability with respect to treating microbial, inherited, multifactoral and acquired diseases. The book also pays specific attention to the ethical issues associated with this new field. Cutting-edge topics and clinically relevant materials engage and maintain student interest Self-assessment questions are included throughout the book Features an additional web site, with a web forum, regular updates and PowerPoint slides of figures from the book Molecular Therapeutics: 21st Century Medicine is a comprehensive, accessible and engaging guide to the rapidly developing field of molecular therapeutics. It is essential reading for all students in this area of research and also of interest for health professionals involved in these novel therapies.

This popular exploration of gene therapy presents the underlying molecular biology in readily accessible form, and lays out the early efforts and future prospects for gene therapy to revolutionize the treatment of human genetic diseases.

Lecture Notes on Molecular Medicine provides a concise and straightforward introduction to molecular biology, explaining how it is used to understand and treat human disease. This new edition has been written in response to exciting changes in this fast-moving field. Fully updated, it explains the human genome project and how the sequence will change medicine. It also covers many new methods that have been introduced since the first edition was published. Beginning with first principles, the book is a useful primer for any science student new to molecular biology and genetics. It is also an invaluable resource for medical students and practicing doctors who need an understanding of how advances in molecular biology have impacted clinical medicine, especially in the fields of gene therapy and screening. For ease of use Lecture Notes on Molecular Medicine is divided into four sections: Basic Principles: describing the fundamentals of DNA structure and function that underpin molecular biology Biomolecular Tools: covering the manipulation of DNA and RNA and molecular techniques. Understanding Genetics: covering the basic principles of inheritance, biodiversity, gene mapping and expression and gene therapy. Molecular Medicine in Practice: discussing the profound effect which molecular biology has had on medical practice at all levels. This chapter has been greatly expanded in this new edition to cover all the latest developments in the field. A concise introduction to the basic principles & applications of molecular medicine. Explains complicated science in simple terms with clear diagrams. Integrates basic and clinical science by emphasising application to clinical medicine. Expanded chapter examining molecular medicine in clinical practice.

Since the discovery of the molecular structure of genes and the unveiling of the molecular basis of numerous human diseases, scientists have been fas- nated with the possibility of treating certain diseases by transducing foreign DNA into the affected cells. Initially, it was proposed that the foreign DNA could either replace defective nonfunctional genes, or code for therapeutic proteins. This concept has evolved into the rapidly growing field of gene therapy. Even though surgery, radiotherapy, and chemotherapy are widely ava- able and routinely used for cancer treatment, these therapies fail to cure approximately 50 percent of cancer patients. Therefore, since it is a disease characterized by aberrant gene expression, cancer has been a target of gene therapy research since the inception of this treatment modality. Numerous cancer gene therapy strategies are currently being investigated, including gene replacement therapy, the regulation of gene expression to modulate immu- logical responses to tumors, the direct killing of tumor cells, and direct int- ference with tumor growth. In this context, gene transfer systems, tumor-specific expression vectors, and novel therapeutic genes have been extensively st- ied. All these strategies aim for the selective destruction of human malignant disease while circumventing the destruction of nonmalignant cells and tissues thereby minimizing toxicity to the patient.