There is no doubt that the study of the muscular dystrophies in recent years has been exciting and rewarding. It has attracted the attention of many investigators of international repute, and this is reflected in the various contributions to this volume. Molecular Mechanisms of Muscular Dystrophies represents a personal collection of chapters cover
A practical resource for everyone involved in the gene therapy field and in the design of effective gene delivery systems, this volume presents an overview and update of recent advances in the field of non-viral methods for the in vivo transfer of therapeutic genes to biological targets using conventional routes of administration. Methods to control the spatial and temporal modulation of gene function in vivo as well as the level, duration, specificity, and fidelity of gene expression are described. The rational design and the applications of a variety of non-viral gene delivery systems, such as cationic lipid-, polymer-, and (poly) peptide-based systems, are exemplified for the control of location of therapeutic genes administered by various routes. Current and potential clinical applications of gene-based medicines are presented for the prevention, correction or modulation of diseases. Examples of current applications of plasmid-based systems for genetic vaccination, treatment of genetic disorders such as cystic fibrosis, and treatment of acquired diseases such as cancer are also provided.
Advances in Genetics increases its focus on modern human genetics and its relation to medicine with the merger of this long-standing serial with Molecular Genetic Medicine. This merger affirms the Academic Press commitment to publish important reviews of the broadest interest to geneticists and their colleagues in affiliated disciplines. This volume of Advances in Genetics continues the series goal to present both human and molecular genetic reviews in a timely fashion. Anderson and Kay investigate CAB gene expression in the regulation of gene transcriptions in plants. Harmon and Allan offer a historical overview of apoptosis and its recent heightened interest. Developments involving four X-linked disorders and their resulting increased susceptibility to infection are presented by Smithand Notarangelo. Dickson and colleagues present research on the role of dystrophin in Duchenne muscular dystrophy and the potential feasibility of gene therapy in curing this disease as well as other diseases involving the heart and central nervous system. The relationship between mice and humans and the ways in which they help each other understand gene function and relationship is presented by Elizabeth Fisher. Timmons and Shearn summarize what information has been learned about prune, Killer of prune, and the prune/Killer of prune lethal interactions: rare genetic phenomena in Drosophila melanogaster. Meiotic recombination, via the breaking and rejoining of DNA and the subsequent reciprocal exchange and the cytological evidence of the exchange, are explored by Ross et al. Last, Coonar and McKenna discuss the four major cardiomyopathies, focusing on recent advances in hypertrophic cardiomyopathy.
The absence of dystrophin is the underlying cause of Duchenne muscular dystrophy. This is the first book to address the structure, function, and distribution of dystrophin and its associated proteins in muscle and brain, rather than concentratiing primarily on the disease. It covers an exciting and rapidly expanding field that promises to have important and widespread implications for many aspects of cell biology.
Culling together excerpts from a wide range of writings by Dr. Kewal K. Jain on biotechnology topics as they relate to disorders of the nervous system, Applications of Biotechnology in Neurology covers a variety of applications for those working in life sciences and the pharmaceutical sciences, particularly those developing diagnostics and therapeutics for the nervous system. This detailed volume delves into areas such as neurobiotechnology, like neurogenomics and neuroproteomics, molecular diagnostics, various methods of improving systemic administration of drugs for targeted delivery to the nervous system, including the use of nanobiotechnology, biotechnology-based strategies and products for neuroprotection, as well as chapters on neurosurgery and personalized neurology. Thorough, cutting-edge, and thoughtfully organized, Applications of Biotechnology in Neurology serves as an ideal guide, supplemented by 75 tables and 16 figures as well as numerous references from recent literature on this topic, which are appended to each chapter.
Angiogenesis and Cardiovascular Disease provides a comprehensive review of the basic scientific and clinical advances in the field of angiogenesis, and its role in the human cardiovascular system. The material presented draws from multiple disciplines, integrating information in a single source. Topics addressed focus first on aspects of development and normal biology of the vasculature, and then on angiogenesis and how it is involved in the pathophysiology and therapy of ischemic cardiovascular disease. Vascular development, endothelial cell biology, the vascular matrix, and growth factors are discussed. Up to date information on current clinical trials, and practical advice concerning application to cardiovascular therapeutic options, are also stressed.
Building upon the success of the Handbook of Laboratory Animal Science and completing Volumes I and II of the Second Edition, Handbook of Laboratory Animal Science, Second Edition: Animal Models, Volume III provides the final component to present a comprehensive overview of animal models in biomedical research.As with Volume II, this new volume add
International Review of Cytology presents current advances and comprehensive reviews in cell biology – both plant and animal. Authored by some of the foremost scientists in the field, each volume provides up-to-date information and directions for future research. Articles in this volume address therapeutic strategies for duchenne and becker dystrophies, cellular distribution and functions of P2 receptor subtypes in different systems, neuropeptides in hypothalamic neuronal disorders, and multiple parallelisms in animal cytokinesis.
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.