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Exploration Of The Mitochondria As A Potential Therapeutic Target In Duchenne Muscular Dystrophy
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Book Synopsis Exploration of the Mitochondria as a Potential Therapeutic Target in Duchenne Muscular Dystrophy by : Meghan Clare Hughes
Download or read book Exploration of the Mitochondria as a Potential Therapeutic Target in Duchenne Muscular Dystrophy written by Meghan Clare Hughes and published by . This book was released on 2019 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease resulting from a mutation in the X-linked gene encoding the protein dystrophin. DMD is characterized by profound muscle weakness as degenerating muscle is replaced by fat and connective tissue. Early loss of ambulation followed by premature death due to cardiac and/or respiratory failure characterize the most debilitating aspects of DMD, a disease for which there is currently no cure. Limited success has been reported when treating DMD with gene based therapies. Current standard of care involves glucocorticoids, which target a secondary cellular myopathy; inflammation. While this line of treatment has provided promising benefits, these drugs present a variety of negative side effects for patients. As such, extensive research has been focused on identifying both therapeutic targets and corresponding novel therapies for the treatment of the DMD myopathy. The focus of this dissertation was to first determine the degree and precise mechanism of mitochondrial dysfunction in DMD followed by the evaluation of SBT-20, a mitochondrial-targeted peptide, as a therapeutic candidate for the treatment and prevention of DMD pathophysiology. In order to address these questions, we first comprehensively evaluated mitochondrial bioenergetics across a spectrum of oxidative and glycolytic muscles in the D2.B10-DMDmdx/2J mouse (D2.mdx) in early and late stages of disease progression. We then tested the efficacy of SBT-20 in improving both DMD myopathy in respiratory and skeletal muscles, as well as the underlying mechanism of mitochondrial dysfunction in dystrophic muscle. Our findings reveal that the mitochondrial H2O2 emission is elevated during impaired oxidative phosphorylation in cardiac, respiratory, oxidative and glycolytic muscle in young (4-week) and aged (52-week) D2.mdx mice and furthermore, that there is a specific defect in mtCK functionality in oxidative muscle. SBT-20 was effective in improving mitochondrial bioenergetics following short (4 weeks) and long (12 weeks) term treatment. This was associated with improved pathophysiology following short-term treatment. Taken together, this thesis identifies impairments in mitochondrial bioenergetics as a contributing factor to the pathophysiology in dystrophic muscle and further highlights SBT-20 as a promising therapy for the improvement of myopathy in DMD.
Book Synopsis Studies of Pseudohypertrophic Muscular Dystrophy by : Charles A. Bonsett
Download or read book Studies of Pseudohypertrophic Muscular Dystrophy written by Charles A. Bonsett and published by . This book was released on 1969 with total page 184 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Heritable Human Genome Editing by : The Royal Society
Download or read book Heritable Human Genome Editing written by The Royal Society and published by National Academies Press. This book was released on 2021-01-16 with total page 239 pages. Available in PDF, EPUB and Kindle. Book excerpt: Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight.
Book Synopsis A Treatise on Localized Electrization, and Its Applications to Pathology and Therapeutics by : Guillaume-Benjamin Duchenne
Download or read book A Treatise on Localized Electrization, and Its Applications to Pathology and Therapeutics written by Guillaume-Benjamin Duchenne and published by . This book was released on 1871 with total page 388 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Exon Skipping and Inclusion Therapies by : Toshifumi Yokota
Download or read book Exon Skipping and Inclusion Therapies written by Toshifumi Yokota and published by Humana Press. This book was released on 2018-09-01 with total page 569 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents a comprehensive collection of detailed state-of-the-art exon skipping and splices modulation protocols. Chapters detail 14 genetic diseases, AON-mediated therapies, and CRISPR/Cas9-mediated gene editing therapies. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help researchers initiate the development of next-generation therapies.
Book Synopsis Disorders of Voluntary Muscle by : George Karpati
Download or read book Disorders of Voluntary Muscle written by George Karpati and published by Cambridge University Press. This book was released on 2001-07-12 with total page 800 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rewritten and redesigned, this remains the one essential text on the diseases of skeletal muscle.
Book Synopsis Calcium Signalling and Disease by : Ernesto Carafoli
Download or read book Calcium Signalling and Disease written by Ernesto Carafoli and published by Springer Science & Business Media. This book was released on 2007-09-29 with total page 591 pages. Available in PDF, EPUB and Kindle. Book excerpt: Authors highlight several promising discoveries in the field of calcium signaling that provide new information about both genetic and acquired pathologies. Their discussions will give you new insights into the underlying causes of congenital and acquired diseases and point the way to new, even more promising research and therapies.
Book Synopsis Myology: Scientific basis of myology by : Andrew Engel
Download or read book Myology: Scientific basis of myology written by Andrew Engel and published by . This book was released on 1994 with total page 1937 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Neurogenetics, Part II written by and published by Elsevier. This book was released on 2018-01-29 with total page 480 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neurogenetics, Part II, Volume 148, the latest release in the Handbook of Clinical Neurology, provides the latest information on the genetic methodologies that are having a significant impact on the study of neurological and psychiatric disorders. Using genetic science, researchers have identified over 200 genes that cause or contribute to neurological disorders. Still an evolving field of study, defining the relationship between genes and neurological and psychiatric disorders is expected to dramatically grow in scope. Part II builds on the foundation of Part I, expanding the coverage to dementias, paroxysmal disorders, neuromuscular disorders, white matter and demyelination diseases, cerebrovascular diseases, adult psychiatric disorders and cancer and phacomatoses. Contains comprehensive coverage of neurogenetics Details the latest science and its impact on our understanding of neurological, psychiatric disorders Presents a focused reference for clinical practitioners and the neuroscience/neurogenetics research community
Book Synopsis Regenerative Pharmacology by : George J. Christ
Download or read book Regenerative Pharmacology written by George J. Christ and published by Cambridge University Press. This book was released on 2013-04-15 with total page 361 pages. Available in PDF, EPUB and Kindle. Book excerpt: A state-of-the-art primer on the role of pharmacological sciences in regenerative medicine, for advanced students, postdoctoral fellows, and researchers.
Book Synopsis Muscle Gene Therapy by : Dongsheng Duan
Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.
Book Synopsis Molecular Mechanisms of Muscular Dystrophies by : Steve J. Winder
Download or read book Molecular Mechanisms of Muscular Dystrophies written by Steve J. Winder and published by CRC Press. This book was released on 2006-01-26 with total page 264 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents a collection of chapters covering a spectrum of muscular dystrophies and one myopathy that represent most of the major muscular dystrophies, and, in particular, those where a molecular understanding of the underlying mechanisms is most advanced.
Book Synopsis Homeostatic Control of Brain Function by : Detlev Boison
Download or read book Homeostatic Control of Brain Function written by Detlev Boison and published by Oxford University Press. This book was released on 2016 with total page 657 pages. Available in PDF, EPUB and Kindle. Book excerpt: Homeostatic Control of Brain Function offers a broad view of brain health and diverse perspectives for potential treatments, targeting key areas such as mitochondria, the immune system, epigenetic changes, and regulatory molecules such as ions, neuropeptides, and neuromodulators. Loss of homeostasis becomes expressed as a diverse array of neurological disorders. Each disorder has multiple comorbidities - with some crossing over several conditions - and often disease-specific treatments remain elusive. When current pharmacological therapies result in ineffective and inadequate outcomes, therapies to restore and maintain homeostatic functions can help improve brain health, no matter the diagnosis. Employing homeostatic therapies may lead to future cures or treatments that address multiple comorbidities. In an age where brain diseases such as Alzheimer's or Parkinson's are ever present, the incorporation of homeostatic techniques could successfully promote better overall brain health. Key Features include · A focus on the homeostatic controls that significantly depend on the way one lives, eats, and drinks. · Highlights from emerging research in non-pharmaceutical therapies including botanical medications, meditation, diet, and exercise. · Incorporation of homeostatic therapies into existing basic and clinical research paradigms. · Extensive scientific basic and clinical research ranging from molecules to disorders. · Emerging practical information for improving homeostasis. · Examples of homeostatic therapies in preventing and delaying dysfunction. Both editors, Detlev Boison and Susan Masino, bring their unique expertise in homeostatic research to the overall scope of this work. This book is accessible to all with an interest in brain health; scientist, clinician, student, and lay reader alike.
Book Synopsis Drosophila Models for Human Diseases by : Masamitsu Yamaguchi
Download or read book Drosophila Models for Human Diseases written by Masamitsu Yamaguchi and published by Springer. This book was released on 2018-06-27 with total page 314 pages. Available in PDF, EPUB and Kindle. Book excerpt: Most biological pathways, physical and neurological properties are highly conserved between humans and Drosophila and nearly 75% of human disease-causing genes have a functional homologue in Drosophila. This volume provides recent advances in Drosophila models for various human diseases, with each chapter providing a review of studies involving Drosophila models, as well as detailed protocols commonly used in laboratories. Starting with a review of Drosophila’s value as a highly tractable model organism for studying human diseases, subsequent chapters present Drosophila models for specific human diseases. The book provides a useful resource for all scientists who are starting to use the Drosophila model in their studies, and for researchers working in the pharmaceutical industry and using new screening models to develop new medicines for various diseases.
Author :National Academies of Sciences, Engineering, and Medicine Publisher :National Academies Press ISBN 13 :0309452880 Total Pages :329 pages Book Rating :4.3/5 (94 download)
Book Synopsis Human Genome Editing by : National Academies of Sciences, Engineering, and Medicine
Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.
Book Synopsis Neuromuscular Disorders of Infancy, Childhood, and Adolescence by : Basil T. Darras
Download or read book Neuromuscular Disorders of Infancy, Childhood, and Adolescence written by Basil T. Darras and published by Elsevier. This book was released on 2014-12-03 with total page 1156 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuromuscular disorders are diagnosed across the lifespan and create many challenges especially with infants, children and adolescents. This new edition of the definitive reference, edited by the established world renowned authorities on the science, diagnosis and treatment of neuromuscular disorders in childhood is a timely and needed resource for all clinicians and researchers studying neuromuscular disorders, especially in childhood. The Second Edition is completely revised to remain current with advances in the field and to insure this remains the standard reference for clinical neurologists and clinical research neurologists. The Second Edition retains comprehensive coverage while shortening the total chapter count to be an even more manageable and effective reference. Carefully revised new edition of the classic reference on neuromuscular disorders in infancy, childhood and adolescence. Definitive coverage of the basic science of neuromuscular disease and the latest diagnosis and treatment best practices. Includes coverage of clinical phenomenology, electrophysiology, histopathology, molecular genetics and protein chemistry
Book Synopsis Systemic Autoimmunity by : P. E. Bigazzi
Download or read book Systemic Autoimmunity written by P. E. Bigazzi and published by CRC Press. This book was released on 1991-08-30 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: Surveys the biotechnologically influenced advances in the understanding of systemic autoimmune disorders, highlighting recent research using cell biology and biochemistry, the cloning of immune cells, recombinant DNA, and molecular genetics. Among the topics are the role of complement in inflammatio
