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This book contains the contribution to the 37th Hemophilia Symposium, Hamburg 2006. The main topics are epidemiolgy, treatment of inhibitors in hemophiliacs, hemophilic arthropathy and synovitis, relevant hemophilia treatment 2006, and pediatric hemostasiology. The volume is rounded off by numerous free papers and posters on hemophilia, casuistics, and diagnostics.

Medicine & Public Health. --- Hematology. --- Orthopedics. --- Pediatrics. --- Medicine. --- Médecine --- Hématologie --- Orthopédie --- Pédiatrie --- Hemofilie --- Hemophilia -- Congresses. --- Hemophilia A -- Congresses. --- Hemophilia B -- Congresses. --- von Willebrand Diseases -- Congresses. --- Hemophilia --- Blood Coagulation Disorders, Inherited --- Genetic Diseases, X-Linked --- Hemorrhagic Disorders --- Coagulation Protein Disorders --- Blood Coagulation Disorders --- Genetic Diseases, Inborn --- Hematologic Diseases --- Hemic and Lymphatic Diseases --- Congenital, Hereditary, and Neonatal Diseases and Abnormalities --- Diseases --- Hemophilia B --- Hemophilia A --- Medicine --- Health & Biological Sciences --- Pathology --- Paediatrics --- Pediatric medicine --- Children --- Orthopaedics --- Orthopedia --- Surgery --- Haematology --- Internal medicine --- Blood --- Health and hygiene

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chromosomen --- genitaal stelsel --- Gynaecology. Obstetrics --- Urology. Andrology --- pediatrie --- aangeboren afwijkingen --- Paediatrics --- Adolescent. --- Sexual Dysfunction, Physiological. --- Sex Chromosome Aberrations. --- 176.4 --- #GROL:MEDO-616.6 --- #GBIB:IDGP --- kinderchirurgie --- Abnormalities, Sex Chromosome --- Chromosome Abnormalities, Sex --- Sex Chromosome Abnormalities --- Aberration, Sex Chromosome --- Aberrations, Sex Chromosome --- Abnormality, Sex Chromosome --- Chromosome Aberration, Sex --- Chromosome Aberrations, Sex --- Chromosome Abnormality, Sex --- Sex Chromosome Aberration --- Sex Chromosome Abnormality --- Sex Chromosomes --- Sex Chromosome Disorders --- Genetic Diseases, X-Linked --- Sex Chromosome Disorders of Sex Development --- Sex Disorders --- Sexual Disorders, Physiological --- Sexual Dysfunctions, Physiological --- Physiological Sexual Disorder --- Physiological Sexual Disorders --- Physiological Sexual Dysfunction --- Physiological Sexual Dysfunctions --- Sexual Disorder, Physiological --- Reproductive Health --- Adolescents --- Adolescents, Female --- Adolescents, Male --- Teenagers --- Teens --- Adolescence --- Youth --- Adolescent, Female --- Adolescent, Male --- Female Adolescent --- Female Adolescents --- Male Adolescent --- Male Adolescents --- Teen --- Teenager --- Youths --- Minors --- Seksuele perversies --- abnormalities --- 176.4 Seksuele perversies --- Sexual Health --- Adolescent --- Sexual Dysfunction, Physiological --- Sex Chromosome Aberrations

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Muscle disease represents an important health threat to the general population. For thousands of years, a cure has been deemed extremely remote, if not impossible, for many relentless muscle diseases such as Duchenne muscular dystrophy. The cloning of muscle disease genes and the prospect of introducing the normal gene to diseased muscle by gene therapy finally bring the hope of a cure. Tremendous progress has been made chasing the muscle gene therapy dream over the past two decades. Valuable information is scattered in the literature. This book represents the first compilation specifically dedicated to issues related to muscle gene therapy. It offers a much needed, up-to-date overview and perspective on the current strategies and status of muscle gene therapy. It covers the cutting edge development of a variety of fascinating strategies such as exon-skipping, AAV gene therapy, RNAi and stem cell approaches. With contributions from prominent investigators in the field, it provides a framework to translate bench science to clinical practice in the upcoming years. This book is a must-have for anyone who is interested in muscle gene therapy including established investigators, clinicians, post-doctoral fellows, graduate students, funding agencies, patients and their families and friends.

Muscles -- Disease. --- Muscles -- Diseases -- Gene therapy -- Laboratory manuals. --- Muscles -- Diseases -- Gene therapy. --- Muscles --- Biological Therapy --- Muscular Dystrophies --- Genetic Engineering --- Investigative Techniques --- Biology --- Genetic Diseases, X-Linked --- Muscular Disorders, Atrophic --- Analytical, Diagnostic and Therapeutic Techniques and Equipment --- Genetic Diseases, Inborn --- Biological Science Disciplines --- Genetic Techniques --- Therapeutics --- Muscular Dystrophy, Duchenne --- Gene Therapy --- Methods --- Genetics --- Natural Science Disciplines --- Congenital, Hereditary, and Neonatal Diseases and Abnormalities --- Muscular Diseases --- Diseases --- Disciplines and Occupations --- Musculoskeletal Diseases --- Neuromuscular Diseases --- Nervous System Diseases --- Medicine --- Health & Biological Sciences --- Pathology --- Musculoskeletal System Diseases --- Gene therapy --- Gene therapy. --- Diseases. --- Myopathy --- Therapy, Gene --- Medicine. --- Human genetics. --- Immunology. --- Virology. --- Developmental biology. --- Biomedicine. --- Human Genetics. --- Developmental Biology. --- Genetic engineering --- Medical virology. --- Medical microbiology --- Virology --- Virus diseases --- Immunobiology --- Life sciences --- Serology --- Development (Biology) --- Growth --- Ontogeny --- Heredity, Human --- Human biology --- Physical anthropology --- Microbiology

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The present book is an in-depth synopsis of recent advances in the fragile X field. The fragile X syndrome (FXS) is the most common form of inherited mental retardation, and the molecular-genetic basis of this syndrome has been the subject of extensive experimental scrutiny over the last two decades. This book covers the spectrum of current FXS research, ranging from Drosophila model systems via mouse models to clinical and psychiatric aspects. The volume also provides updates on FXS-related diseases such as the fragile X-associated tremor/ataxia syndrome. Significant progress in recent years, as highlighted here by some of the most eminent researchers in the field, are grounds for optimism that successful therapeutical approaches may be feasible in the not too distant future.

Fragile X syndrome. --- Fragile X syndrome -- Animal models. --- Fragile X syndrome --- Mental Retardation, X-Linked --- Sex Chromosome Disorders --- Investigative Techniques --- Chromosome Disorders --- Mental Retardation --- Analytical, Diagnostic and Therapeutic Techniques and Equipment --- Genetic Diseases, X-Linked --- Heredodegenerative Disorders, Nervous System --- Neurobehavioral Manifestations --- Genetic Diseases, Inborn --- Congenital Abnormalities --- Congenital, Hereditary, and Neonatal Diseases and Abnormalities --- Neurologic Manifestations --- Nervous System Diseases --- Diseases --- Fragile X Syndrome --- Models, Animal --- Medicine --- Biology --- Health & Biological Sciences --- Pediatrics --- Cytology --- Animal models --- X-linked mental retardation. --- XLMR (Disease) --- fra(X) syndrome --- Life sciences. --- Human genetics. --- Gene therapy. --- Molecular biology. --- Cell biology. --- Stem cells. --- Neurobiology. --- Life Sciences. --- Cell Biology. --- Human Genetics. --- Molecular Medicine. --- Stem Cells. --- Gene Therapy. --- Intellectual disability --- X chromosome --- Syndromes --- X-linked mental retardation --- Abnormalities --- Cytology. --- Medicine. --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Colony-forming units (Cells) --- Mother cells --- Progenitor cells --- Cells --- Neurosciences --- Clinical sciences --- Medical profession --- Human biology --- Life sciences --- Medical sciences --- Pathology --- Physicians --- Genetics --- Heredity, Human --- Physical anthropology --- Cell biology --- Cellular biology --- Cytologists --- Health Workforce --- Molecular biochemistry --- Molecular biophysics --- Biochemistry --- Biophysics --- Biomolecules --- Systems biology --- Medical genetics. --- Neurosciences. --- Medical Genetics. --- Biomedical Research. --- Neuroscience. --- Stem Cell Biology. --- Clinical Genetics. --- Research. --- Neural sciences --- Neurological sciences --- Neuroscience --- Nervous system --- Biological research --- Biomedical research --- Clinical genetics --- Heredity of disease --- Human genetics --- Genetic disorders --- Genetic aspects