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Since the first concepts of gene therapy were formulated, the hemopoietic system has been considered the most natural first target tissue for genetic manipulation. The reasons for this include the fact that a very large number of inherited disorders (including some of the most common disorders, such as the hemoglobinopathies) are disorders of the hemopoietic system, and the large amount of experience in hematopoietic transplantation biology. The consequence of this resulted in the first clinical trial of gene therapy in 1989, where two children suffering from severe combined immune deficiency (ADA-SCID) were transplanted with T-cells express ing adenosine deaminase (the defective enzyme in p...
The aim of Molecular Cardiology: Methods and Protocols is to document state-of-the-art molecular and genetic techniques in the area of cardiology. These modern approaches enable researchers to readily study heart diseases at the molecular level and will promote the development of new therapeutic str- egies. Methods for genetic dissection, signal transduction, and microarray analysis are excellent tools for the study of the molecular mechanisms of cardiovascular diseases. Protocols for transgenesis take advantage of recent advances in many areas of molecular and cell biology. Transgenic models of heart diseases (cardiac hypertrophy, cardiac dysfunction, and so on. ) are powerful tools for the...
Pancreatic ductal adenocarcinoma is the fourth leading cause of cancer death in the United States. Annually approximately 30,000 Americans are diagnosed with the disease and most will die from it within five years. P- creatic ductal adenocarcinoma is unique because of its late onset in age, high mortality, small tumor samples infiltrated with normal cells, and a lack of both early detection and effective therapies. Some of these characteristics have made studying this disease a challenge. Pancreatic cancer develops as a result of the accumulation of genetic alterations in cancer-causing genes, such as the oncogenes and the tumor-s- pressor genes. In the last decade, major progress has been m...
With sequencing of the human genome now complete, deciphering the role of gene function in human neurological pathophysiology is a promise that has yet to be realized. More than most diseases, stroke has been keenly studied from a genomic perspective. Studies are numerous and incorporate data on stroke inheritance, chromosomal loci of risk, preclinical models of stroke, and differential gene expression of brain injury, repair, and recovery. The problem is no longer a lack of information but one of interpretation and prioritization of what we do know. The aims of Stroke Genomics: Methods and Reviews are twofold. First, it aims to provide the reader with cutting-edge reviews of clinical and pr...
Essential practical manual for all those working in modern transfusion medicine Why Buy This Book? Concise and 'user friendly’ guide to transfusion medicine Provides guidance for everyday clinical questions Revised and updated throughout to reflect rapid developing areas Scope broadened by including experts from the USA
Gene therapy has expanded rapidly over the last decade. The number of clinical trials reported by 2001 included 532 protocols and 3436 patients. Phase I trials predominate with 359 trials of 1774 patients versus Phase II (57 trials with 507 patients) and Phase III (3 trials of 251 patients). The disease overwhelmingly targeted by gene therapy is cancer: involving 331 trials with 2361 patients. Despite the somewhat disappointing results of clinical trials to date, gene therapy offers tremendous promise for the future of cancer therapy. The area of gene therapy is vast, and both malignant and nonmalignant cells can be targeted. Suicide Gene Therapy: Methods and Reviews covers gene therapy that...
Despite the availability of an effective vaccine, there are still 400 million people, worldwide who are chronically infected with hepatitis B virus (HBV). For them, the vaccine, as currently applied, has no value. Given the possible consequences of HBV infection, the number of those chronically infected with HBV presents an enormous public health challenge. For example, the major etiology of hepatocellular carcinoma (HCC) is chronic infection with HBV. Although fifth in cancer incidence, worldwide, HCC/liver cancer is the third leading cause of cancer death. The high mortality as- ciated with HCC arises because the disease is often detected late and is unresponsive to treatment. The number o...
Opioid research is one of the multidisciplinary research areas that involve advanced techniques ranging from molecular genetics to neuropharmacology, and from behavioral neuroscience to clinical medicine. In current opioid research, it has become increasingly important to use multiple approaches at molecular, cellular, and system levels for investigations on a specific opio- related target system. That often requires understanding and applying cro- field techniques and methods for the success of one’s research projects. Through its broad spectrum of coverage, Opioid Research: Methods and Protocols provides a comprehensive collection of major laboratory methods and protocols in current opio...
“Research is to see what everybody else has seen, and to think what nobody else has thought. ” — Albert Szentgyörgyi Autoimmunity: Methods and Protocols is intended to serve as a ready-to-use guide to establish and interrogate human and animal models of autoimmune diseases. The first chapter, “Pathogenesis and Spectrum of Autoimmunity,” discusses major hypotheses driving this most tantalizing area of research since Paul Ehrlich proposed the concept of autoimmunity in 1900. Considering the great diversity and ever-changing spectrum of autoimmunity, it has not been possible to include models and experimental protocols for each known disorder. Rather, several chapters have been devot...
“Rule IV. There is need of a method for finding out the truth. Rule V. Method consists entirely in the order and disposition of the objects toward which our mental vision must be directed if we would find out any truth. We shall comply with it exactly if we reduce involved and obscure propositions step be step to those that are s- pler, and then starting with the intuitive apprehension of all those that are absolutely simple, attempt to ascend to the knowledge of all others by precisely similar steps. ” —Rene Descartes, Rules for the Direction of Mind “...Perhaps he would sooner satisfy himself by resolving light into colours as far as may be done by Art, and then by examining the properties of those colours apart, and afterwards by trying the effects of reconjoyning two or more or all of those, and lastly by separating them again to examine what changes that reconjunction had wrought in them. This will prove a tedious and difficult task to do it as it ought to be done but I could not be satisfied till I had gone through it. ” —From Newton’s letter, quoted in The Life of Isaac Newton by Richard Westfall. Cambridge University Press, 1993.