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Muscular Dystrophies. --- Myotonia Congenita.

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Adult stem cells are responsible for tissue regeneration and repair throughout life. Their quiescence or activation are tightly regulated by common signalling pathways that often recapitulate those happening during embryonic development, and thus it is important to understand their regulation not only in postnatal life, but also during foetal development. In this regard, skeletal muscle is an interesting tissue since it accounts for a large percentage of body mass (about 40%), it is highly amenable to intervention through exercise and it is also key in metabolic and physiological changes underlying frailty susceptibility in the elderly. While muscle-resident satellite cells are responsible for all myogenic activity in physiological conditions and become senescent in old age, other progenitor cells such as mesoangioblasts do seem to contribute to muscle regeneration and repair after tissue damage. Similarly, fibro-adipogenic precursor cells seem to be key in the aberrant response that fills up the space left from atrophied muscle mass and which ends up with a dysfunctional muscle having vast areas of fatty infiltration and fibrosis. The complex interplay between these stem/progenitor cell types and their niches in normal and pathological conditions throughout life are the subjects of intense investigation. This eBook highlights recent developments on the role of stem cells in skeletal muscle function, both in prenatal and postnatal life, and their regulation by transcriptional, post-transcriptional and epigenetic mechanisms. Additionally, it includes articles on interventions associated with exercise, pathological changes in neuromuscular diseases, and stem cell aging.

ageing --- satellite cell --- pericytes --- fibrosis --- myogenesis --- muscular dystrophies --- rejuvenation --- epigenetics --- muscle wasting --- sarcopenia

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This practical and concise guide offers an overview of muscular dystrophy's (MD) complicated features, treatment options and general resources.  New treatments and a greater understanding of proteins and structures associated with MD are discussed along with long-term patient care.  Also included are clinical and developmental challenges within the current regulatory landscape and recent scientific and clinical advances.  Muscular Dystrophy: A Concise Guide offers clinicians, researchers, pharmaceutical executives and patient advocacy groups an easy-to-read reference that provides the necessary perspectives of the care giver and patient.

Medicine & Public Health. --- Neurology. --- Pharmacotherapy. --- Medicine/Public Health, general. --- Medicine. --- Médecine --- Neurologie --- Muscular dystrophy. --- Medicine --- Health & Biological Sciences --- Neurology --- Muscular dystrophies --- Dystrophy --- Genetic disorders --- Neuromuscular diseases --- Clinical sciences --- Medical profession --- Human biology --- Life sciences --- Medical sciences --- Pathology --- Physicians --- Nervous system --- Neuropsychiatry --- Diseases --- Health Workforce --- Neurology . --- Drug therapy --- Pharmacotherapy --- Therapeutics --- Drugs --- Pharmacology

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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