Introduction.-Probing Astrocyte Function in Fragile X Syndrome.- Neural Stem Cells.- Fragile X Mental Retardation Protein (FMRP) and the Spinal Sensory System. The Role of the Postsynaptic Density in the Pathology of the Fragile X Syndrome.- Behavior in a Drosophila model of Fragile X.- Molecular and Genetic Analysis of the Drosophila Model of Fragile X Syndrome.- Fragile X Mental Retardation Protein and Stem Cells.- Manipulating the Fragile X Mental Retardation Proteins in the Frog.- Exploring the Zebra finch Taeniopygia gutta as a Novel Animal Model for the Speech-language Deficit of Fragile X Syndrome.- Neuroendocrine Alterations in the Fragile X Mouse.- Taking STEPs forward to understanding Fragile X Syndrome.- Fmr-1 as an Offspring Genetic and a Maternal Environmental Factor in Neurodevelopmental Disease.- Mouse Models of the Fragile X Premutation and the Fragile X Associated Tremor/Ataxia Syndrome.- Clinical Aspects of the Fragile X Syndrome.- Fragile X Syndrome: A Psychiatric Perspective.- Fragile X Syndrome and Targeted Treatment Trials.- The Fragile X-associate Tremor Ataxia Syndrome.- Vignettes: Models in Absentia."

This book is a collection of preclinical and clinical reports on the appli cation of gene therapy to human disease. The focus of these studies is on cancer and cardiovascular disease. There are two fundamental technologies for delivering therapeutic genes to diseased ceHs: either viral vectors, as discussed by Dr. Bal main, or non-viral vector systems, as discussed by Dr. Felgner. The strengths and limitations of each of these delivery systems are charac terized. The use of a therapeutic gene to treat a disease has taken two general approaches. The first is to introduce anormal (i. e., wild type) gene into the patient that will restore normal gene function. Dr. Weiss man has characterized the tumor suppressor gene (pS3), and has shown that it can restore normal ceH function in cancer cells. The second ap proach is to treat the disease with antisense molecules. Abnormal gene expression can be down-regulated and selectively inhibited by anti sense molecules, which can reverse the pathologie process in cancer cells. Dr. Gewirtz has demonstrated this with anti sense genes on leuke mia, while Dr. Scanlon has applied this principle using ribozymes in human carcinomas. During this symposium, Dr. Engler described clinical studies of gene therapy using growth factors to stimulate new blood vessels in patients with cardiovascular disease. Several gene therapy strategies were used for cancer: overcoming drug resistance by Dr. Bertino, a pro-drug strategy with ganciclovir by Dr."

In this monograph about gene therapy of autoimmune and inflammatory d- orders we have gathered international experts and leaders from different fields to review the state of the art advances on topics ranging from disease entities to vectors and engineered cells. The different approaches described in each chapter take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long-term genetic interventions. Gene therapy also serves as a testing ground for new therapeutic entities and helps provide proof of principle for their potential therapeutic role in animal models of disease. Scaling up from mice to men still remains an important h- dle not only from the quantitative point of view, but also for currently unknown and unexpected secondary effects of the vector or the transgene. Some of these approaches have already been tested in the clinic, but much more needs to be done to understand the human conditions treated and the n- ural history of their pathology. We are indebted to the secretarial assistance of Ms. Lin Wells (Bone and Joint Research Unit, London, UK) and the help of Hans Detlef Kluber for his help in getting this book published. We hope this book will be of interest to c- nicians and scientists and inspiring to students of the subject who will use their own ingenuity and knowledge to further forward this discipline into clinical use.

With the thorough understanding of stem cell biology and the advent of targeted therapeutics for cancer, stem cell-based therapeutic strategies are being increasingly explored for the treatment of various cancer types."Mesenchymal Stem Cells in Cancer Therapy" sheds light on current stem cell based targeted therapies for cancer, by focusing on the application of mesenchymal stem cells (MSC) in various cancers with emphasis on a number of aspects that are critical to the success of future stem cell based therapies for cancer. Sections of this publicationare devoted to developing stem cell based therapies for cancer with the main focus on tumoritrophic properties of stem cells, engineering targeted therapeutics, utilization of imaging techniques and the recent combination studies utilizing currently employed therapeutics with stem cells."Mesenchymal Stem Cells in Cancer Therapy"informs readers about critical and cutting edge stem cell therapies for cancer and also enables them to appreciate the vast plain of unresolved questions in stem cell research for cancer therapeutics.
Includes biological foundation on key sources of mesenchymal stem cells and the various ways they can be utilized to treat cancer.Provides examples of current MSC based cancer therapies and prospects for the future with insights from the leading lab on cancer cell therapies.Technically advanced topic written for widespread understanding for clinical and research audiences."

Androgens and androgen receptors (AR) play critical roles in the development and progression of prostate cancer, the most frequently diagnosed cancer and second leading cause of cancer death in US males. AR is an androgen-dependent DNA-binding transcription factor that regulates the expression of androgen-responsive genes. Identification and characterization of androgen-responsive genes provide insights into the cellular mechanisms of androgen action and may lead to new approaches in diagnosis, prognosis, prevention and/or treatment of prostate cancer. This volume provides critical information from well respected experts in the field. Some of the exciting topics include the new understanding of mechanisms underlining the regulation of androgen-responsive gene expression, and functions of various androgen-responsive genes in biological processes essential in carcinogenesis including cell growth, angiogenesis, and epithelial-to-mesenchyme transition (EMT). Other important aspects addressed are the current and potential clinic applications of knowledge on androgen-responsive gene regulation and function. This book is intended for researchers, scientists, faculty, and advanced graduate students with an interest in androgen action and prostate cancer.

Continued refinement ofwide-spread access to transgenic technology has allowed for new animal models have been developed that exhibit features of autoimmune disease have been developed that exhibit features of autoimmune disease. The second edition of "Autoimmunity: Methods and Protocols" researchers in the field detail many of the most up-to-date methods which are now commonly used to study autoimmunity. The first half the book focuses on methods and protocols used to assess immunological and biochemical pathways of diseases pathogenesis in human subjects. While the second half investigates treatment of inflammatory arthritis, experimental allergic encephalomyelitis (EAE), IDDM, scleroderma, and uveitis in animal models and assessment of genetic, immunological, and biochemical parameters underlying spontaneous or exogenous antigen-induced diseases. Written in the highly successful "Methods in Molecular Biology(tm)" series format, the chapters include the kind of detailed description and implementation advice that is crucial for getting optimal results in the laboratory.

Through and intuitive, "Autoimmunity: Methods and Protocols, Second Edition" seeks to aid scientists in the autoimmunity field to extract new meaning of old models and developing new ones."

During the first half century of genetics, coinciding with the first half of this cen tury, geneticists dreamt of the repair of genetic disease by altering or replacing defective genes. H. J. Muller wrote of the great advantages of mutations, "nanoneedles" in his apt term, for delicately probing physiological and chemical processes. In the same spirit, genes could be used to provide treatments of needle point delicacy. Yet, during this period no realistic possibility appeared; it remained but a dream. The situation changed abruptly at the half century. Microbial genetics and its offshoot, cell culture genetics, provided the route. Pneumococcus transformation showed that exogenous DNA could become a permanent part of the genome; yet attempts to reproduce this in animals produced a few tantalizing hints of success, but mostly failures. Transduction, using a virus as mediator, offered a better op portunity. The fITSt reproducible in vivo gene therapy in a whole animal came in 1981. This was in Drosophila, with a transposable element as carrier. Flies were "cured" of a mutant eye color by incorporation of the normal allele, and the effect was transmissible, foreshadowing not only somatic, but germ line gene therapy. At the same time, retroviruses carrying human genes were found to be ex tremely efficient in transferring their contents to the chromosomes of cultured cells."

Cardiovascular disease has evolved into an epidemic health concern. During the past decades, considerable effort has been made to develop novel and safer therapeutic options. Gene therapy has offered much renewed hope and instigated the present book. For the treatment of cardiovascular disease gene therapy has risen to an interesting and important point of view of curing disease. Although most of the studies are still experimental beneath a well defined number of clinical studies, more and more studies are designed to be carried out in humans. Based on a rich compilation of scientific as well as clinical studies around the world, this book gives insights into the general principles of gene transfer as well as therapeutic strategies including stem cell therapy as an alternative approach to gene therapy.

This book has been written in response to the many physicians and scien tists working on the development of biological approaches to providing therapies for many orthopaedic disorders as well as to improving the healing of many tissues of the musculoskeletal system. The first goal of this book is to make the language compatible between the bench scientist and the clinician working in orthopaedic and sports medicine in order to cover specific areas of the orthopaedic discipline where the treatment can be improved and/or changed by the advancements in molecular medicine. Advancements in molecular biology, which encompass the study of the genetic basis of disease, have produced new diagnostic methods and drug therapies for genetic diseases and acquired disorders. The growth in the understanding of human genetics has also led to the initiation of many human gene therapy experiments. Although many approved therapeutic clinical trials using this new technology have been performed in the last ten years, the first clinical trial using this technology in the area of orthopaedics was performed at the University of Pittsburgh."

The creation of the science on stem cells and development of its theoretical bases is a prevalent topic today, taking into account comparative evolutionary cell biology and the cardinal problem of the developmental biology. This allows revealing correlations and studying correlative dependencies of various structures at different levels of biohierarchy. The creation of every science is impossible without the application of methodology. This book examines the system of non-traditional ideas about the nature and role of stem cells in ontogenesis, reproduction and evolution of plants. The main properties of plant stem cells have been developed, which has shown the integrity of morphogenous and reproductive processes at all stages of plant's life cycle.

This book presents and discusses current research in the study of stem cells. Topics discussed include research on adult stem cells; cell therapy applications; somatic cell nuclear transfer; stem cell research in Europe; an artificial niche for expansion of long-term engraftable hematopoietic cells; global characterisation and genomic stability of human MultiStem; haematopoietic and stromal stem cell regulation by extracellular matrix components and growth factors and stem cell divisions.

The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular Biology (TM) series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.

MicroRNAs (miRNAs), endogenous noncoding regulatory mRNAs of around 22-nucleotides long, have rapidly emerged as one of the key governors of the gene expression regulatory program in cells of varying species, with ever-increasing implications in the control of the fundamental biological processes and in the pathogenesis of adult humans. The exciting findings in this field have inspired us with a premise and a promise that miRNAs will ultimately be taken to the heart for therapy of human disease. While miRNAs have been considered potential therapeutic targets for disease treatment, it remains obscured what strategies we can use to achieve the goal. In the past years, we have witnessed a rapid evolving of many creative, innovative, inventive strategies and methodologies pertinent to miRNA research and applications. These technologies have convincingly demonstrated their efficacy and reliability in producing gain-of-function or loss-of-function of miRNAs through targeting miRNA expression/biogenesis/function, providing new tools for elucidating miRNA functions and opening up a new avenue for the development of new agents targeting miRNAs for therapeutic aims. The present book provides comprehensive descriptions of these technologies and their applications to miRNA research and to new drug design for miRNA-related diseases. It starts with an overview of up-to-date knowledge of miRNA biology and the potential of miRNAs as therapeutic targets for human disease, followed by an introduction of the new concept of miRNA interference (miRNAi) and the perspectives of miRNAi technologies in general terms. In the following, each chapter introduces one of the miRNAi technologies with detailed descriptions of state-of-the-art design, procedures, principles and applications to basic research, R and D and clinical therapy.

Recent stem cell research has revealed that miRNA and RNAi-mediated gene regulation is one of the vital determinates controlling the state of cell differentiation, with the small RNAs serving as key elements involved in regulatory network control of pluripotent cell fate determination. In RNAi and microRNA-Mediated Gene Regulation in Stem Cells: Methods, Protocols, and Applications, expert authors from laboratories across the globe contribute an accessible compendium of up-to-date, proven methods focused on the study of the titular topic. Divided into three sections, the book first gives a brief introduction to RNAi and miRNAs in stem cells, with a focus on the current status of research and future perspectives, then it continues with detailed methods and protocols for RNAi screening, transfection, and the knockdown of specific genes and pathways in several animal species, including humans and mice, concluding with a section on recently developed methods for identification of miRNAs, including a general protocol for preparation and analysis of miRNA libraries for deep sequencing, knock down of a specific gene using miRNA-based shRNA, and miRNA expression analysis using qRT-PCR. Written in the highly successful Methods in Molecular Biology (TM) series format, chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and notes highlighting tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, RNAi and microRNA-Mediated Gene Regulation in Stem Cells: Methods, Protocols, and Applications serves as a valuable resource for scientists and aspiring graduate students interested in the intersection of RNAi, miRNA, and stem cell molecular biology and the exciting areas of medicine, including regenerative medicine, aging, cancer, and neurological disorders, that can be advanced through this expanding area of research.

Cell Therapy: cGMP Facilities and Manufacturing is the source for a complete discussion of facility design and operation with practical approaches to a variety of day-to-day activities, such as staff training and competency, cleaning procedures, and environmental monitoring. This in-depth book also includes detailed reviews of quality, the framework of regulations, and professional standards. It meets a previously unmet need for a thorough facility-focused resource, Cell Therapy: cGMP Facilities and Manufacturing will be an important addition to the cell therapy professionala (TM)s library.

Additional topics in Cell Therapy: cGMP Facilities and Manufacturinga ]

• Standard operating procedures
• Supply management
• Facility equipment
• Product manufacturing, review, release and administration
• Facility master file

As the leading cause of adult disability, stroke poses substantial economic and psychological burdens to populations around the world. Against this dismal backdrop, several novel "neurorestorative" approaches are being investigated as adjunctive treatments to physiotherapy. Stroke Recovery with Cellular Therapies discusses the promising investigations around the world on cell-based therapies to enhance recovery from stroke. Throughout this groundbreaking text, chapters detail the potential benefits of various types of cells and approaches for ischemic and hemorrhagic stroke, and all studies rely upon traditional histological and functional outcomes. Among the topics detailed in this volume are transplanting immortalized neural stem cells, transplantation of bone marrow stromal cells, adipose-derived stem cells, endogenous neural stem cells in tissue remodeling, and an investigation into the ethics of stem cell research. This text provides an overview of the field, stimulates ideas for further research, and will help serve as a foundation for cell-based therapies to move forward as a viable approach for stroke recovery in the future.

Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight disease. Gene therapy is being studied in clinical trials for many different types of cancer and for numerous other diseases. The volume presents significant new research results in this promising field.

Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight disease. Gene therapy is being studied in clinical trials for many different types of cancer and for numerous other diseases. This book offers important research from around the globe dedicated to this dynamic subject.

The possibility of treating cancer, a disease frequently defined by
genetic defects, by introducing genes that target these very
alterations has generated tremendous enthusiasm. This enthusiasm,
however, has been tempered by an increasing number of obstacles to
successful therapy, including vector systems that do not reach systemic
metastases, therapeutic genes with redundant mechanisms allowing for
cellular resistance, and toxicities in clinical trials that result in
premature closure. The three comprehensive sections of this volume
present currently available cancer gene therapy techniques, with
specific attention to these trouble spots. Part I describes the various
aspects of gene delivery including vehicles, or vectors, and their
respective characteristics and production methods. In Part II, the
contributors discuss strategies and targets for the treatment of
cancer, including methods for cell-death therapies, correction of
underlying genetic defects at the molecular level, and activation of
the immune system or tumor microenvironment. The contributors provide a
succinct framework for understanding the basic underlying oncogenic
changes, which encourages the development of vectors engineered to
exploit these gene mutations through selective spread of the vector in
tumor cells with the specific changes. Finally, in Part III, experts in
clinical gene therapy trials discuss the difficulties inherent in
bringing gene therapy treatment for cancer to the clinic, and principal
investigators present gene therapy approaches in the clinical testing
stage and the results that have reached the stage ofclinical
testing.of these trials.

Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight disease. Gene therapy is being studied in clinical trials for many different types of cancer and for numerous other diseases. This new book offers the latest research in a field bursting with new developments, hope and expectations.

This set reports the results of the 10th International Histocompatibility Workshop, in which 362 laboratories collaborated over a three year period in research projects on the classification of HLA genes and their products. "Volume 1" describes the experimental design of the workshop studies and their results. "Volume 2" is a collection of papers on the latest developments in the molecular biology of HLA systems. Immunobiology of HLA is a valuable reference for tissue typing laboratories, blood banks, and general research programs on HLA and related diseases because it identifies common sources of HLA genes and gene products to be used as reference reagents, and because it is the only complete compilation of the latest research and results in the field.

Stem cells are the building blocks of the body and can develop into any of the cells that make up our bodies. Stem cells carry a lot of hope for the treatment of a broad range of diseases and injuries, spanning from cancers, diabetes, genetic diseases, graft-versus-host disease, eye, heart and liver diseases, inflammatory and autoimmune disorders, to neurological diseases and injuries, particularly neurodegenerative diseases, like Alzheimer's and Parkinson's disease and many others. This book provides an overview and in-depth analysis of recent developments in stem cell research and therapy. It is composed of recently published review articles that went through a peer-review process.

Even as classic cytogenetics has given way to molecular karyotyping, and as new deletion and duplication syndromes are identified almost every day, the fundamental role of the genetics clinic remains mostly unchanged. Genetic counselors and medical geneticists explain the "unexplainable," helping families understand why abnormalities occur and whether they're likely to occur again. Chromosome Abnormalities and Genetic Counseling is the genetics professional's definitive guide to navigating both chromosome disorders and the clinical questions of the families they impact. Combining a primer on these disorders with the most current approach to their best clinical approaches, this classic text is more than just a reference; it is a guide to how to think about these disorders, even as our technical understanding of them continues to evolve. Completely updated and still infused with the warmth and voice that have made it essential reading for professionals across medical genetics, this edition of Chromosome Abnormalities and Genetic Counseling represents a leap forward in clinical understanding and communication. It is, as ever, essential reading for the field.

This book summarizes microRNA (miRNA) biology in a variety of pathological processes, emphasizing the significant potential applications of miRNA in diagnostics and prognostics, as well as novel drug targets. The conventional techniques used for miRNA detection including standard PCR, Northern blotting, microarray and clone methods are addressed. Recent emerging strategies in miRNA detection and quantification with superior flexibility and adaptability, such as novel molecular biological techniques and locked nucleic acid (LNA) modified probes, as well as nanotechnology-based approaches, are also included. The book also highlights the latest advances in clinical-related miRNA detection methods in living cells, circulating blood and tissue, such as in situ hybridization (ISH) and molecular imaging techniques, which are useful to elucidate the biogenesis and biological function of miRNAs in vivo. Finally, the respective advantages and drawbacks of various detection techniques in this fast-moving field are discussed, along with the challenges and promising new directions. This book offers a valuable resource for analytical chemists, biologists and physicians involved in miRNA research. Dr. Xueji Zhang and Dr. Haifeng Dong are Professors at the School of Chemistry & Biological Engineering, University of Science & Technology Beijing (USTB), China. Dr. Yaping Tian is a Professor at the Department of Clinical Biochemistry, Chinese PLA General Hospital and Military Medical School, China.

"Gene Therapy for Cartilage and Bone Tissue Engineering" outlines the tissue engineering and possible applications of gene therapy in the field of biomedical engineering as well as basic principles of gene therapy, vectors and gene delivery, specifically for cartilage and bone engineering. It is intended for tissue engineers, cell therapists, regenerative medicine scientists and engineers, gene therapist and virologists. Dr. Yu-Chen Hu is a Distinguished Professor at the Department of Chemical Engineering, National Tsing Hua University and has received the Outstanding Research Award (National Science Council), Asia Research Award (Society of Chemical Engineers, Japan) and Professor Tsai-Teh Lai Award (Taiwan Institute of Chemical Engineers). He is also a fellow of the American Institute for Medical and Biological Engineering (AIMBE) and a member of the Tissue Engineering International & Regenerative Medicine Society (TERMIS)-Asia Pacific Council.