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The growing knowledge about disturbances of epigenetic gene regulation in hematopoietic stem cell disorders such as myeloid leukemias and preleukemias (myelodysplasias) is now being translated into treatment approaches that target the epigenetic defects pharmacologically. Such approaches include reactivation of tumor suppressor genes and other antiproliferative genes by reversal of DNA hypermethylation through azanucleosides, and use of HDAC inhibitors to reverse gene silencing mediated by chimeric, leukemia-specific transcription factors. This book first presents the latest evidence derived from preclinical models regarding the epigenetic regulation of hematopoietic stem cell differentiation and hemoglobin production. The significance of DNA methylation abnormalities in hematopoietic disorders and of epigenetic disturbances in lung cancer and other solid tumors is then discussed. A major part of the book, however, relates specifically to the translation of basic research and drug development to clinical applications, and in this context both present and future clinical strategies are considered. Individual chapters are devoted to the use of DNA hypomethylating agents and chromatin-modifying agents, and the treatment of hematologic malignancies and solid tumors by means of epigenetic agents is discussed in detail.
Cancer --- Gene therapy. --- Hematology. --- Oncology. --- Treatment. --- Haematology --- Therapy, Gene --- Cancer therapy --- Cancer treatment --- Therapy --- Medicine. --- Medicine & Public Health. --- Gene Therapy. --- Tumors --- Internal medicine --- Blood --- Genetic engineering --- Therapeutics --- Diseases --- Oncology .
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This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations. The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to pro
Genetic transformation. --- Gene therapy. --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Gene transfer --- Transformation (Genetics) --- Genetic recombination --- Microbial genetics --- Nucleic acids --- Transfection
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Progress in molecular and cellular biology has greatly enhanced our ability to accurately diagnose diseases that are caused by gene mutations, changes in genome structures, and altered gene expression; increased emphasis is now placed on translational research and the clinical treatment of these genetically determined diseases. Seeking Cures outlines the progress and implications of science's quest to identify therapeutic targets and initiate novel treatments at the gene, RNA, protein, and physiological levels. Also considered are aspects of treatment at the cellular level (e.g., those with he
Genetic disorders. --- Gene therapy. --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Congenital diseases --- Disorders, Genetic --- Disorders, Inherited --- Genetic diseases --- Hereditary diseases --- Inherited diseases --- Diseases --- Medical genetics
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This book will contain the proceedings of the XV International Symposium on Retinal Degeneration (RD2012). A majority of those who will speak and present posters at the meeting will contribute to this volume. The blinding diseases of inherited retinal degenerations have no treatments, and age-related macular degeneration has no cures, despite the fact that it is an epidemic among the elderly, with 1 in 3-4 affected by the age of 70. The RD Symposium will focus on the exciting new developments aimed at understanding these diseases and providing therapies for them. Since most major scientists in the field of retinal degenerations attend the biennial RD Symposia, they are known by most as the “best” and “most important” meetings in the field. The volume will present representative state-of-the-art research in almost all areas of retinal degenerations, ranging from cytopathologic, physiologic, diagnostic and clinical aspects; animal models; mechanisms of cell death; candidate genes, cloning, mapping and other aspects of molecular genetics; and developing potential therapeutic measures such as gene therapy and neuroprotective agents for potential pharmaceutical therapy. While advances in these areas of retinal degenerations will be described, there will be many new topics that either were in their infancy or did not exist at the time of the last RD Symposium, RD2010. These include the role of inflammation and immunity, as well as other basic mechanisms, in age-related macular degeneration, several new aspects of gene therapy, and revolutionary new imaging and functional testing that will have a huge impact on the diagnosis and following the course of retinal degenerations, as well as to provide new quantitative endpoints for clinical trials. The retina is an approachable part of the central nervous system (CNS), and there is a major interest in neuroprotective and gene therapy for CNS diseases and neurodegenerations, in general. It should be noted that with successful and exciting initial clinical trials in neuroprotective and gene therapy, including the restoration of sight in blind children, the retinal degeneration therapies are leading the way towards new therapeutic measures for neurodegenerations of the CNS. Many of the successes recently reported in these areas of retinal degeneration sprang from collaborations established at previous RD Symposia, and many of those will be reported at the RD2010 meeting and included in the proposed volume. We anticipate the excitement of those working in the field and those afflicted with retinal degenerations will be reflected in the volume.
Retinal degeneration. --- Dystrophy, Retinal --- Macular degeneration --- Retina --- Retinal dystrophy --- Degeneration (Pathology) --- Degeneration --- Diseases --- Ophthalmology. --- Neurosciences. --- Geriatrics. --- Human genetics. --- Gene therapy. --- Geriatrics/Gerontology. --- Human Genetics. --- Gene Therapy. --- Medicine --- Eye --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Genetics --- Heredity, Human --- Human biology --- Physical anthropology --- Gerontology --- Older people --- Neural sciences --- Neurological sciences --- Neuroscience --- Medical sciences --- Nervous system --- Health and hygiene
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This Brief describes the concept and realization of gene therapy for HIV from the unique historic perspective and insight of two pioneers of the clinical applications of stem cell gene therapy for HIV. Gerhard Bauer applied ribozyme-anti-HIV and other vectors to manufacture clinical grade, HIV-resistant hematopoietic stem cells for the first patients that received stem cell gene therapy for HIV, including the first child in the world and the first fully marrow-ablated HIV infected patient. Joseph Anderson developed the most recent and most potent combination anti-HIV lentiviral vectors and pluripotent stem cell applications for HIV gene therapy and tested these in the appropriate in vitro and vivo models, paving the way for novel HIV gene therapy approaches to possibly cure patients. In Gene Therapy for HIV, Bauer and Anderson discuss the unique aspects of this therapy, including its limitations and proper safety precautions and outline a path for a possible functional cure for HIV using stem cell gene therapy based on a cure already achieved with a bone marrow stem cell transplantation performed in Germany using donor stem cells with a naturally arising CCR5 mutation. In addition, the Brief provides a thorough and methodical explanation of the basics of gene therapy, gene therapy vector development, in vitro and in vivo models for HIV gene therapy and clinical applications of HIV gene therapy, including Good Manufacturing Practices.
AIDS (Disease) -- Gene therapy. --- Gene therapy. --- Immunogenetics. --- HIV infections --- AIDS (Disease) --- Lentivirus Infections --- Sexually Transmitted Diseases, Viral --- Immunologic Deficiency Syndromes --- Immune System Diseases --- Retroviridae Infections --- Sexually Transmitted Diseases --- RNA Virus Infections --- Diseases --- Virus Diseases --- HIV Infections --- Public Health --- Health & Biological Sciences --- Communicable Diseases --- Gene therapy --- HIV infections. --- Genetic aspects. --- Acquired immune deficiency syndrome --- Acquired immunodeficiency syndrome --- Acquired immunological deficiency syndrome --- HIV (Viruses) infections --- HTLV-III infections --- HTLV-III-LAV infections --- Human T-lymphotropic virus III infections --- Therapy, Gene --- Medicine. --- Immunology. --- Vaccines. --- Virology. --- Biomedicine. --- Gene Therapy. --- Vaccine. --- Genetic engineering --- Therapeutics --- Microbiology --- Biologicals --- Immunobiology --- Life sciences --- Serology --- Clinical sciences --- Medical profession --- Human biology --- Medical sciences --- Pathology --- Physicians --- Lentivirus infections --- Sexually transmitted diseases --- Immunological deficiency syndromes --- Virus-induced immunosuppression --- Medical virology. --- Medical microbiology --- Virology --- Virus diseases
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The research described in this book represents important steps toward understanding the development of inner ear medicine and new perspectives in regenerative medicine, including efficacy in cochlear implants and various other treatments. The book depicts the mechanisms that underlie inner ear diseases, their experimental models, and proposals for new strategies to treat their symptoms. As well, the exciting future prospects for dealing with the very common problem of inner ear diseases are explained. These disorders occur among many people and include sensorineural hearing loss (SNHL), sudden deafness, senile deafness, noise-induced deafness, tinnitus, dizziness–vertigo, and Ménière’s disease. In Japan alone, there are more than 6 million deaf patients including those with middle-range deafness. There is currently no effective treatment, and regardless of the underlying cause, the damage has been considered irreversible. However, the results of recent research show that these patients actually can recover. The study of hair cells, spiral ganglion neurons, and stem cells for inner ear diseases such as SNHL, tinnitus, dizziness, and vertigo is at the forefront of regenerative medicine and may provide solutions to some of these problems. The information presented here makes this book a valuable professional reference work for all doctors and researchers in the field of otolaryngology who focus on regenerative treatments for inner ear diseases.
Labyrinth (Ear) --- Regenerative medicine --- Diseases --- Treatment. --- Medicine --- Regeneration (Biology) --- Inner ear --- Internal ear --- Ear --- Hearing --- Otorhinolaryngology. --- Head --- Gene therapy. --- Neurosciences. --- Regenerative Medicine/Tissue Engineering. --- Head and Neck Surgery. --- Gene Therapy. --- Surgery. --- Neural sciences --- Neurological sciences --- Neuroscience --- Medical sciences --- Nervous system --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Ear, nose, and throat diseases --- ENT diseases --- Otorhinolaryngology --- Regenerative medicine. --- Tissue engineering. --- Otolaryngologic surgery. --- Operative otolaryngology --- Otolaryngologic surgery --- Surgery, Operative --- Surgery, Orificial --- Biomedical engineering --- Tissue culture
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"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.
Biomedical engineering. --- Gene therapy. --- Genetic engineering. --- Stem cells. --- Tissue engineering --- Bones --- Cartilage --- Connective Tissue --- Culture Techniques --- Skeleton --- Musculoskeletal System --- Investigative Techniques --- Tissues --- Anatomy --- Clinical Laboratory Techniques --- Analytical, Diagnostic and Therapeutic Techniques and Equipment --- Methods --- Bone and Bones --- Tissue Engineering --- Health & Biological Sciences --- Biomedical Engineering --- Gene therapy --- Diseases --- Tissue engineering. --- Bone regeneration. --- Methodology. --- Mechanical properties. --- Bone --- Bone biomechanics --- Bone mechanics --- Mechanical properties of bones --- Gristle --- Therapy, Gene --- Regeneration --- Engineering. --- Regenerative medicine. --- Biomedical Engineering. --- Genetic Engineering. --- Gene Therapy. --- Regenerative Medicine/Tissue Engineering. --- Stem Cells. --- Biomedical engineering --- Regenerative medicine --- Tissue culture --- Clinical engineering --- Medical engineering --- Bioengineering --- Biophysics --- Engineering --- Medicine --- Regeneration (Biology) --- Colony-forming units (Cells) --- Mother cells --- Progenitor cells --- Cells --- Designed genetic change --- Engineering, Genetic --- Gene splicing --- Genetic intervention --- Genetic surgery --- Genetic recombination --- Biotechnology --- Transgenic organisms --- Genetic engineering --- Therapeutics --- Construction --- Industrial arts --- Technology --- Bone remodeling --- Connective tissues --- Ligaments --- Biomedical Engineering and Bioengineering.
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This book discusses the emergence of a new class of genes with a specific anticancer activity. These genes, recently defined as “Anticancer Genes”, are reviewed in individual chapters on their mode of action, the specific cell death signals they induce, and the status of attempts to translate them into clinical application. Anticancer Genes provides an overview of this nascent field, its genesis, current state, and prospect. It discusses how Anticancer Genes might lead to the identification of a repertoire of signaling pathways directed against cellular alterations that are specific for tumor cells. With contributions from experts worldwide, Anticancer Genes is an essential guide to this dynamic topic for researchers and students in cancer research, molecular medicine, pharmacology and toxicology and genetics as well as clinicians and clinical researchers interested in the therapeutic potential of this exciting new field.
Cancer --- Medical genetics. --- Biomedicine. --- Genetic aspects. --- Clinical genetics --- Diseases --- Heredity of disease --- Human genetics --- Medical sciences --- Pathology --- Genetic disorders --- Cancer genetics --- Cancer genes --- Genetic aspects --- Medicine. --- Oncology. --- Gene therapy. --- Human genetics. --- Toxicology. --- Molecular Medicine. --- Cancer Research. --- Gene Therapy. --- Human Genetics. --- Pharmacology/Toxicology. --- Genetics --- Heredity, Human --- Human biology --- Physical anthropology --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Tumors --- Clinical sciences --- Medical profession --- Life sciences --- Physicians --- Chemicals --- Medicine --- Pharmacology --- Poisoning --- Poisons --- Toxicology --- Health Workforce --- Molecular biology. --- Cancer research. --- Pharmacology. --- Drug effects --- Medical pharmacology --- Chemotherapy --- Drugs --- Pharmacy --- Cancer research --- Molecular biochemistry --- Molecular biophysics --- Biochemistry --- Biophysics --- Biomolecules --- Systems biology --- Physiological effect
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This volume describes our current understanding of the biological role of visual and non-visual arrestins in different cells and tissues, focusing on the mechanisms of arrestin-mediated regulation of GPCRs and non-receptor signaling proteins in health and disease. The book covers wide range of arrestin functions, emphasizing therapeutic potential of targeting arrestin interactions with individual partners.
Arrestins. --- Membrane proteins. --- Membranes (Biology) --- Proteins --- Membrane proteins --- Toxicology. --- Biochemistry. --- Gene therapy. --- Cell physiology. --- Cell receptors. --- Pharmacology/Toxicology. --- Biochemistry, general. --- Protein Structure. --- Gene Therapy. --- Cell Physiology. --- Receptors. --- Biological chemistry --- Chemical composition of organisms --- Organisms --- Physiological chemistry --- Biology --- Chemistry --- Medical sciences --- Chemicals --- Medicine --- Pharmacology --- Poisoning --- Poisons --- Cell membrane receptors --- Cell surface receptors --- Receptors, Cell --- Binding sites (Biochemistry) --- Cell membranes --- Cell function --- Cytology --- Physiology --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Composition --- Toxicology --- Pharmacology. --- Proteins . --- Proteids --- Biomolecules --- Polypeptides --- Proteomics --- Drug effects --- Medical pharmacology --- Chemotherapy --- Drugs --- Pharmacy --- Physiological effect
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This book provides comprehensive coverage of smart biomaterials and their potential applications, a field that is developing at a very rapid pace. Because smart biomaterials are an emerging class of biomaterials that respond to small changes in external stimuli with large discontinuous changes in their physical properties, they have been designed to act as an “on–off” switch for, among others, bioseparation, immunoanalysis, drug delivery technologies, gene therapy, diagnostics, biosensors, and artificial muscles. After an introduction to the topic and the history of smart biomaterials, the author gives the reader an in-depth look at the properties, mechanics, and characterization of smart biomaterials including hydrogels, particles, assemblies, surfaces, fibers, and conjugates. Information on the wide range of applications for these materials follows, including drug delivery, tissue engineering, diagnostics, biosensors, bioseparation, and actuators. In addition, recent advances in shape memory biomaterials as active components of medical devices are also presented.
Smart materials. --- Biomedical materials. --- Materials science. --- Material science --- Physical sciences --- Biocompatible materials --- Biomaterials --- Medical materials --- Medicine --- Biomedical engineering --- Materials --- Biocompatibility --- Prosthesis --- Adaptive materials --- Intelligent materials --- Sense-able materials --- Biomaterials. --- Gene therapy. --- Polymers. --- Nanotechnology. --- Gene Therapy. --- Polymer Sciences. --- Soft and Granular Matter, Complex Fluids and Microfluidics. --- Polymere --- Polymeride --- Polymers and polymerization --- Macromolecules --- Molecular technology --- Nanoscale technology --- High technology --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Polymers . --- Amorphous substances. --- Complex fluids. --- Complex liquids --- Fluids, Complex --- Amorphous substances --- Liquids --- Soft condensed matter --- Bioartificial materials --- Hemocompatible materials --- Biomaterials (Biomedical materials)
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