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| dmd exon skipping therapy: Muscle Gene Therapy Dongsheng Duan, 2009-11-26 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. |
| dmd exon skipping therapy: Duchenne Muscular Dystrophy Camilla Bernardini, 2017-10-25 This volume explores experimental approaches used to study Duchenne muscular dystrophy (DMD), an X-linked degenerative skeletal muscle disease caused by mutations in the dystrophin gene. Including the latest progress and scientific achievements, the book covers recent discoveries achieved through in vivo gene editing which have proven to be promising in restoring dystrophin expression, at least in ameliorating skeletal muscle symptoms, and the contents focus on “Omics” techniques in gene expression, protein expression, miRNAs, and long non-coding RNA analysis, as well as experimental studies of the structural/functional changes affecting the skeletal and cardiac muscles and ongoing preclinical studies and clinical trials. 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 practical, Duchenne Muscular Dystrophy: Methods and Protocols serves as a guide for researchers exploring the complicated nature of dystrophin in the hope of helping the victims of this disorder. |
| dmd exon skipping therapy: Nonsense Mutation Correction in Human Diseases Fabrice Lejeune, Hana Benhabiles, Jieshuang Jia, 2016-02-26 Nonsense Mutation Correction in Human Diseases: An Approach for Targeted Medicine provides an introduction on genetic diseases, discusses the prevalence of nonsense mutations, the consequences of a nonsense mutation for the expression of the mutant gene, and the presentation of the nonsense-mediated mRNA decay (NMD). It presents the mechanism of action and rationale associated with each strategy to correct nonsense mutations with the results of clinical trials to further support this basis. In addition, the book shows how it may be possible to combine several of these strategies to ultimately improve the efficiency of correction, also suggesting the future goals and objectives to improve treatment modalities in this evolving sphere of personalized medicine. - Features basic biological and clinical constructs that inform the application of genomic data to clinical decision-making - Includes theories and methods that can be used to link bio-molecular and clinical phenotypes so as to enable integrative hypothesis discovery, testing, and downstream evidence-based practice - Provides design patterns and use cases that contextualize the clinical decision-making and evidence-based practice relative to real world requirements and stakeholders |
| dmd exon skipping therapy: Genome Editing in Neurosciences Rudolf Jaenisch, Feng Zhang, Fred Gage, 2020-10-08 Innovations in molecular biology are allowing neuroscientists to study the brain with unprecedented resolution, from the level of single molecules to integrated gene circuits. Chief among these innovations is the CRISPR-Cas genome editing technology, which has the precision and scalability to tackle the complexity of the brain. This Colloque Médecine et Recherche has brought together experts from around the world that are applying genome editing to address important challenges in neuroscience, including basic biology in model organisms that has the power to reveal systems-level insight into how the nervous system develops and functions as well as research focused on understanding and treating human neurological disorders. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors. |
| dmd exon skipping therapy: Neuromuscular Disorders of Infancy, Childhood, and Adolescence Basil T. Darras, H. Royden Jones Jr., Monique M. Ryan, Darryl C. De Vivo, 2014-12-03 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 |
| dmd exon skipping therapy: Exon Skipping and Inclusion Therapies Toshifumi Yokota, Rika Maruyama, 2018-09-01 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. |
| dmd exon skipping therapy: Duchenne Muscular Dystrophy Jeffrey S. Chamberlain, Thomas A. Rando, 2006-02-27 Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of childhood and currently stands as an incurable condition. This authoritative guide provides a clear overview of the latest current and experimental approaches to the treatment of DMD and examines the clinical, genetic, and pathophysiological aspects of the disease i |
| dmd exon skipping therapy: Neuromuscular Disorders: Management and Treatment E-Book Tulio E. Bertorini, 2010-09-08 Neuromuscular Disorders presents a multi-disciplinary approach to the management and therapeutic treatment of the full range of neuromuscular disorders and resulting complications. Dr. Tulio Bertorini and a contributing team of the world’s leading authorities in the field provide the latest tools and strategies for minimizing disability and maximizing quality of life. Effectively treat your patients using the latest management tools and targeted therapeutic strategies. Manage all neuromuscular disorders as well as resulting complications through comprehensive coverage of diagnosis and evaluations, treatments, and outcomes. Apply the multi-disciplinary approach of an expert in clinical neuromuscular care and a team of world-renown contributors. Easily refer to tools for diagnosis, treatment algorithms, and drug tables included throughout the text. |
| dmd exon skipping therapy: Rosenberg's Molecular and Genetic Basis of Neurological and Psychiatric Disease Roger N. Rosenberg, Juan M. Pascual, 2014-10-28 Rosenberg’s Molecular and Genetic Basis of Neurologic and Psychiatric Disease, Fifth Edition provides a comprehensive introduction and reference to the foundations and key practical aspects relevant to the majority of neurologic and psychiatric disease. A favorite of over three generations of students, clinicians and scholars, this new edition retains and expands the informative, concise and critical tone of the first edition. This is an essential reference for general medical practitioners, neurologists, psychiatrists, geneticists, and related professionals, and for the neuroscience and neurology research community. The content covers all aspects essential to the practice of neurogenetics to inform clinical diagnosis, treatment and genetic counseling. Every chapter has been thoroughly revised or newly commissioned to reflect the latest scientific and medical advances by an international team of leading scientists and clinicians. The contents have been expanded to include disorders for which a genetic basis has been recently identified, together with abundant original illustrations that convey and clarify the key points of the text in an attractive, didactic format. Previous editions have established this book as the leading tutorial reference on neurogenetics. Researchers will find great value in the coverage of genomics, animal models and diagnostic methods along with a better understanding of the clinical implications. Clinicians will rely on the coverage of the basic science of neurogenetics and the methods for evaluating patients with biochemical abnormalities or gene mutations, including links to genetic testing for specific diseases. Comprehensive coverage of the neurogenetic foundation of neurological and psychiatric disease Detailed introduction to both clinical and basic research implications of molecular and genetic understanding of the brain Detailed coverage of genomics, animal models and diagnostic methods with new coverage of evaluating patients with biochemical abnormalities or gene mutations |
| dmd exon skipping therapy: Curing Genetic Diseases through Genome Reprogramming , 2021-06-24 Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome. - Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing - Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects - Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations |
| dmd exon skipping therapy: Molecular Biology of RNA David Elliott, Michael Ladomery, 2017-01-31 RNA plays a central, and until recently, somewhat underestimated role in the genetics underlying all forms of life on earth. This versatile molecule not only plays a crucial part in the synthesis of proteins from a DNA template, but is also intrinsically involved in the regulation of gene expression, and can even act as a catalyst in the form of a ribozyme. This latter property has led to the hypothesis that RNA - rather than DNA - could have played an essential part in the origin of life itself. This landmark text provides a systematic overview of the exciting and rapidly moving field of RNA biology. Key pioneering experiments, which provided the underlying evidence for what we now know, are described throughout, while the relevance of the subject to human disease is highlighted via frequent boxes. For the second edition of Molecular Biology of RNA, more introductory material has been incorporated at the beginning of the text, to aid students studying the subject for the first time. Throughout the text, new material has been included - particularly in relation to RNA binding domains, non-coding RNAs, and the connection between RNA biology and epigenetics. Finally, a new closing chapter discusses how exciting new technologies are being used to explore current topical areas of research. |
| dmd exon skipping therapy: Antisense RNA Design, Delivery, and Analysis Virginia Arechavala-Gomeza, Alejandro Garanto, 2022-02-26 This open access volume gathers a variety of models, delivery systems, and approaches that can be used to assess RNA technology for exploiting antisense as a therapeutic intervention. Beginning with a section on the design of antisense technology and their delivery, the book continues by covering model systems developed to evaluate efficacy, both in vivo and in vitro, as well as methods to evaluate preclinically the toxicity associated with these new potential drugs, and intellectual property considerations. Written for the highly successful Methods in Molecular Biology series, 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 practical, Antisense RNA Design, Delivery, and Analysis provides basic knowledge and a large collection of methods to facilitate the work of newcomers to this vibrant and expanding field. This book was conceived thanks to the network DARTER (Delivery of Antisense RNA Therapeutics). DARTER is funded by the EU Cooperation of Science and Technology (COST), which aims to enhance interaction and collaborations between researchers in Europe and other countries. |
| dmd exon skipping therapy: RNA Splicing and Backsplicing: Disease and Therapy Rosanna Asselta, Stefano Duga, Emanuele Buratti, Eladio Andrés Velasco, 2020-12-24 This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact. |
| dmd exon skipping therapy: Recent Advances in Nutrigenetics and Nutrigenomics C Bouchard, J. M. Ordovas, 2012-06-19 Recent Advances in Nutrigenetics and Nutrigenomics. |
| dmd exon skipping therapy: Translating Regenerative Medicine to the Clinic Jeffrey Laurence, Pedro Baptista, Anthony Atala, 2015-11-18 Translating Regenerative Medicine to the Clinic reviews the current methodological tools and experimental approaches used by leading translational researchers, discussing the uses of regenerative medicine for different disease treatment areas, including cardiovascular disease, muscle regeneration, and regeneration of the bone and skin. Pedagogically, the book concentrates on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field. It promotes cross-disciplinary communication between the sub-specialties of medicine, but remains unified in theme by emphasizing recent innovations, critical barriers to progress, the new tools that are being used to overcome them, and specific areas of research that require additional study to advance the field as a whole. Volumes in the series include Translating Gene Therapy to the Clinic, Translating Regenerative Medicine to the Clinic, Translating MicroRNAs to the Clinic, Translating Biomarkers to the Clinic, and Translating Epigenetics to the Clinic. - Encompasses the latest innovations and tools being used to develop regenerative medicine in the lab and clinic - Covers the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field - Contains extensive pedagogical updates aiming to improve the education of translational researchers in this field - Provides a transdisciplinary approach that supports cross-fertilization between different sub-specialties of medicine |
| dmd exon skipping therapy: The Human Genome Julia E. Richards, R. Scott Hawley, 2010-12-12 Significant advances in our knowledge of genetics were made during the twentieth century but in the most recent decades, genetic research has dramatically increased its impact throughout society. Genetic issues are now playing a large role in health and public policy, and new knowledge in this field will continue to have significant implications for individuals and society. Written for the non-majors human genetics course, Human Genetics, Third Edition will increase the genetics knowledge of students who are learning about human genetics for the first time. This thorough revision of the best-selling Human Genome, Second Edition includes entirely new chapters on forensics, stem cell biology, bioinformatics, and societal/ethical issues associated with the field. New special features boxes make connections between human genetics and human health and disease. Carefully crafted pedagogy includes chapter-opening case studies that set the stage for each chapter; concept statements interspersed throughout the chapter that keep first-time students focused on key concepts; and end-of-chapter questions and critical thinking activities. This new edition will contribute to creating a genetically literate student population that understands basic biological research, understands elements of the personal and health implications of genetics, and participates effectively in public policy issues involving genetic information. - Includes topical material on forensics, disease studies, and the human genome project to engage non-specialist students - Full, 4-color illustration program enhances and reinforces key concepts and themes - Uniform organization of chapters includes interest boxes that focus on human health and disease, chapter-opening case studies, and concept statements to engage non-specialist readers |
| dmd exon skipping therapy: Disorders of Voluntary Muscle George Karpati, David Hilton-Jones, Robert C. Griggs, 2001-07-12 Rewritten and redesigned, this remains the one essential text on the diseases of skeletal muscle. |
| dmd exon skipping therapy: Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy Vijai Singh, 2021-06-15 Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy, presents the collation of chapters written by eminent scientists worldwide. CRISPR-Cas9 is a key technology for targeted genome editing and regulation in a number of organisms including mammalian cells. It is a rapid, simple, and cost-effective solution. CRISPR-Cas system has recently gained much scientific and public attention. This volume covers CRISPR-Cas9 based mammalian genome editing, creating disease models, cancer therapy, neurological, heredity, blood disorders, defective gene correction, stem cells therapy, epigenetic modifications, patents, ethics, biosafety and regulatory issues challenges and opportunities. This book is a key source of information on mammalian genome editing available in a single volume. This book will be useful for beginners in mammalian genome editing and also students, researchers, scientists, policymakers, clinicians and stakeholders interested in genome editing in several areas. Offers basic understanding and a clear picture of mammalian genome editing through CRISPR-Cas systems Discusses how to create mammalian disease models, stem cell modification, epigenetic modifications, correction of defective gene in blood disorders, heredity, neurological disorders and many more Discusses the application of CRISPR-Cas9 systems in basic sciences, biomedicine, molecular biology, translational sciences, neurobiology, neurology, cancer, stem cells, and many more |
| dmd exon skipping therapy: Pediatric Pharmacotherapy Wieland Kiess, Matthias Schwab, Johannes van den Anker, 2020-07-15 This volume provides readers with the most updated scientific information on the efficacy and safety of medicines for children and adolescents. The book enriches the understanding of pediatric pharmacotherapy for health professionals, regulatory agencies, pharmaceutical companies and learned societies. It contains important information on the pharmacodynamics and pharmacokinetics of drugs. It summarizes the latest investigations on the effects of pharmacological treatments in relation to and dependent on age, gender, fat mass and disease status. Therefore and importantly, this volume reviews the latest data on how pharmacotherapy has to be adjusted and personalized in regards to stages of development and during the pediatric lifespan from neonate through adolescence. In addition, the topic of rare diseases and special challenges for pharmacotherapy will be included and will provide readers with the necessary knowledge to handle complex diseases and treatment strategies especially in relation to pharmacotherapy of rare and orphan diseases. |
| dmd exon skipping therapy: Animal Models in Medicine and Biology Eva Tvrdá, Sarat Chandra Yenisetti, 2020-04-08 Thanks to animal models, our knowledge of biology and medicine has increased enormously over the past decades, leading to significant breakthroughs that have had a direct impact on the prevention, management and treatment of a wide array of diseases.This book presents a comprehensive reference that reflects the latest scientific research being done in a variety of medical and biological fields utilizing animal models. Chapters on Drosophila, rat, pig, rabbit, and other animal models reflect frontier research in neurology, psychiatry, cardiology, musculoskeletal disorders, reproduction, chronic diseases, epidemiology, and pain and inflammation management. Animal Models in Medicine and Biology offers scientists, clinicians, researchers and students invaluable insights into a wide range of issues at the forefront of medical and biological progress. |
| dmd exon skipping therapy: A Guide to Duchenne Muscular Dystrophy Janet Hoskin, 2017-12-14 The prognosis for individuals with Duchenne Muscular Dystrophy (DMD) is improving, with some men with DMD living into their 30s and 40s. More vital than ever, this book helps teachers and parents to support children and young people with DMD with their education and transition into adulthood. Leading experts on DMD explain Duchenne and its impact in easy-to-understand terms. Going beyond physical management, particular focus is put on learning and behavioural issues, including speech delay and difficulty learning to read, as well as common comorbid conditions, such as ADHD, autism and OCD. Raising aspirations, the book gives guidance on effective support in the classroom and advice on the transition to adulthood, employment and independent living. |
| dmd exon skipping therapy: Oligonucleotide-Based Therapies Olof Gissberg, Rula Zain, Karin E. Lundin, 2020-08-15 This book provides a collection of comprehensive, up-to-date, and broadly applicable guides to the research and development fields of oligonucleotide (ON) therapeutics. Covering topics from the study of antisense and anti-gene effects to oligonucleotides in the context of drug discovery and development, the volume explores a wide-ranging and useful spectrum of methods and protocols needed to take full advantage of therapeutic applications involving ONs. Written for the highly successful Methods in Molecular Biology series, 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 practical, Oligonucleotide-Based Therapies: Methods and Protocols aims to be a great aid in the laboratory as well as an ideal reference guide when designing antisense and anti-gene oligonucleotides for therapeutic applications. |
| dmd exon skipping therapy: Muscle Biopsy Victor Dubowitz, 1985 |
| dmd exon skipping therapy: Cardiomyopathy Gustav Mattsson, Peter Magnusson, 2021 Cardiomyopathies are diseases of the heart muscle with diverse etiologies ranging from myocarditis to gene mutations. They are classified according to morphology and function, and then further categorized based on whether they are familial or non-familial and based on specific etiologies. This book examines the various cardiomyopathies, including arrhythmogenic cardiomyopathy, hypertrophic cardiomyopathy, and dilated cardiomyopathy, as well as their genetic basis. |
| dmd exon skipping therapy: Gene Correction Francesca Storici, 2016-09-03 Gene correction is a technology that gives us the tools for both repairing and mutating DNA, for discovering gene functions and for engineering new genetic variants. Gene Correction: Methods and Protocols provides a user friendly, detailed and up-to-date collection of strategies and methodologies utilized for generating specific sequence changes in the DNA of cells in the laboratory, while also tackling the major problems that the field of gene correction faces. This volume brings together many experts in the field of gene correction to disclose a wide and varied array of specific gene correction protocols for engineering mutations in DNA, for delivering correcting DNA to target cells, and for improving the accuracy and safety of the gene correction process. Written in the 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 protocols, and notes on troubleshooting and avoiding known pitfalls. Authoritative and easily accessible, Gene Correction: Methods and Protocols seeks to serve scientists of all backgrounds interested in the area of gene targeting/recombination/therapy. |
| dmd exon skipping therapy: Muscular Dystrophy Corrado Angelini, 2013 Muscular dystrophies include a heterogeneous series of diseases that range from childhood to adult onset cases, of difficult diagnosis and treatment. This book presents the state of the art in muscular dystrophies, after the molecular revolution. |
| dmd exon skipping therapy: Principles of Cloning Jose Cibelli, Ian Wilmut, Rudolf Jaenisch, John Gurdon, Robert Lanza, Michael West, Keith H.S. Campbell, 2013-09-24 Principles of Cloning, Second Edition is the fully revised edition of the authoritative book on the science of cloning. The book presents the basic biological mechanisms of how cloning works and progresses to discuss current and potential applications in basic biology, agriculture, biotechnology, and medicine. Beginning with the history and theory behind cloning, the book goes on to examine methods of micromanipulation, nuclear transfer, genetic modification, and pregnancy and neonatal care of cloned animals. The cloning of various species—including mice, sheep, cattle, and non-mammals—is considered as well. The Editors have been involved in a number of breakthroughs using cloning technique, including the first demonstration that cloning works in differentiated cells done by the Recipient of the 2012 Nobel Prize for Physiology or Medicine – Dr John Gurdon; the cloning of the first mammal from a somatic cell – Drs Keith Campbell and Ian Wilmut; the demonstration that cloning can reset the biological clock - Drs Michael West and Robert Lanza; the demonstration that a terminally differentiated cell can give rise to a whole new individual – Dr Rudolf Jaenisch and the cloning of the first transgenic bovine from a differentiated cell – Dr Jose Cibelli. The majority of the contributing authors are the principal investigators on each of the animal species cloned to date and are expertly qualified to present the state-of-the-art information in their respective areas. - First and most comprehensive book on animal cloning, 100% revised - Describes an in-depth analysis of current limitations of the technology and research areas to explore - Offers cloning applications on basic biology, agriculture, biotechnology, and medicine |
| dmd exon skipping therapy: Rewriting Nature Paul Enríquez, 2021-06-24 Rewriting Nature is a cogent, riveting interdisciplinary exploration of the law, science, and policy of emerging genome-editing technology. |
| dmd exon skipping therapy: Antisense Drug Technology Stanley T. Crooke, 2007-07-25 Extensively revised and updated, Antisense Drug Technology: Principles, Strategies, and Applications, Second Edition reflects the logarithmic progress made in the past four years of oligonucleotide-based therapies, and, in particular, antisense therapeutics and research. Interpreting lessons learned from the clinical trials of first generati |
| dmd exon skipping therapy: Plastic and Reconstructive Surgery Maria Z. Siemionow, 2015-01-12 There is a paradigm shift in plastic and reconstructive surgery from the interest of developing new surgical techniques into the application of new technologies via research based studies on stem cells, tissue engineering and new field of reconstructive transplantation such as e.g. face, hand or larynx transplants. This approach is relatively novel and introduced to plastic surgery within past decade. Thus there is an urgent need to facilitate access to this new knowledge which was not traditionally a part of plastic surgery curriculum. The most efficient way of introducing these new approaches is via presentation of pertinent to different fields (stem cell, transplantation, nerve regeneration, tissue engineering) experimental models which can be used as a tool to develop technologies of interest by different groups of surgeons. These surgical specialities which will be interested and benefit from the book include: plastic and reconstructive surgeons, microsurgeons, hand surgeons, orthopaedic surgeons, neurosurgeons and transplant surgeons. |
| dmd exon skipping therapy: Muscular Dystrophy National Institute of Neurological Diseases and Stroke, 1971 |
| dmd exon skipping therapy: Cardiac Diseases David C. Gaze, Aleksandar Kibel, 2021-07-28 This is the latest book in a series of cardiovascular-related texts from IntechOpen Publishing. The present volume considers general aspects of cardiac disease and is divided into three distinct sections covering cardiac risk, cardiorenal pathology, and novel interventional surgical techniques. The chapters offer insight into the current state of the art with respect to the risks of developing cardiovascular diseases, maintenance of patent vascular access in patients with the cardiorenal syndrome, and a plethora of novel interventional technologies all aimed at salvaging damaged tissue and improving prognosis and reducing mortality. This volume of 18 chapters is intended for general medical and biomedical students at both undergraduate and postgraduate level. It also offers insightful updates on recent advances in the understanding of the pathophysiology of cardiac diseases and the new techniques added to the medical armamentarium to improve the outcomes and prevent mortality and would be of interest to those working in academia and healthcare science. |
| dmd exon skipping therapy: Gene Therapy of the Central Nervous System: From Bench to Bedside Michael G. Kaplitt, Matthew During, 2006 Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find insider information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system. |
| dmd exon skipping therapy: Pediatric Neuropsychiatry C. Edward Coffey, Roger A. Brumback, 2006 Pediatric Neuropsychiatry provides the most updated and clinically relevant information on psychiatric disorders in children and adolescents with disturbances of brain function. Bridging the fields of psychiatry and neurology, this landmark work emphasizes the link between developmental brain biology and behavior. Major sections focus on neuropsychiatric aspects of specific psychiatric and neurologic disorders, highlighting the influence of the developing nervous system on these disorders' pathophysiology, manifestations, clinical course, treatment, and prognosis. Other sections discuss all contemporary diagnostic and therapeutic modalities. Chapters include case histories, algorithms, tables, and appendices that explain the rudiments of testing. |
| dmd exon skipping therapy: Therapeutic Oligonucleotides Jens Kurreck, 2008 This book provides a compelling overall update on current status of RNA interference |
| dmd exon skipping therapy: Pulmonary Function Testing in Children: Techniques and Standards George Polgar, Promadhat Varuni, 1971 |
| dmd exon skipping therapy: Mechanisms in Duchenne Muscular Dystrophy Pathophysiology and Treatment Atsushi Asakura, Masatoshi Suzuki, Katsumasa Goto, 2023-09-07 |
| dmd exon skipping therapy: Viral Gene Therapy Ke Xu, 2011 The development of technologies that allow targeting of specific cells has progressed substantially in recent years for several types of vectors, particularly viral vectors, which have been used in 70% of gene therapy clinical trials. Particular viruses have been selected as gene delivery vehicles because of their capacities to carry foreign genes and their ability to efficiently deliver these genes associated with efficient gene expression. This book is designed to present the most recent advances in viral gene therapy. |
| dmd exon skipping therapy: Duchenne Muscular Dystrophy: New Insights for the Healthcare Professional: 2012 Edition , 2012-12-10 Duchenne Muscular Dystrophy: New Insights for the Healthcare Professional / 2012 Edition is a ScholarlyPaper™ that delivers timely, authoritative, and intensively focused information about Duchenne Muscular Dystrophy in a compact format. The editors have built Duchenne Muscular Dystrophy: New Insights for the Healthcare Professional / 2012 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Duchenne Muscular Dystrophy in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Duchenne Muscular Dystrophy: New Insights for the Healthcare Professional / 2012 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/. |
| dmd exon skipping therapy: A Small If: The Inspiring Story of a 17-Year-Old with a Fatal Disease-and a Mission to Cure It Elijah Stacy, 2021-09-28 How hard would you fight if you only had a sliver of hope? Elijah Stacy suffers from Duchenne muscular dystrophy, a fatal muscle wasting disease. At the age of sixteen, to avoid agonizing surgery, he had to endure enough physical therapy to change the shape of his own spine. It had never been done before, but still, his doctor gave him a small if. In A Small If, Elijah shares thirteen lessons he's learned throughout his life's journey of losing his ability to walk, getting bullied in school, losing his brother Max to Duchenne, and learning that his other brother has the same disease. Elijah explains how to overcome extreme suffering by developing an adapter's mindset, understanding what it means to control something rather than influencing it, and using other people's negative energy as the ultimate motivation. Today, Elijah's mission to minimize human suffering and propel human prosperity is fueling his ambition to cure his disease. He's been told he has less than ten years to live, but he's faced long odds before. Sometimes, all you need is A Small If. |
Duchenne Muscular Dystrophy (DMD): Symptoms & Treatment - Cleveland Clinic
Jul 25, 2022 · Duchenne muscular dystrophy (DMD) is a condition that causes skeletal and heart muscle weakness that quickly gets worse with time. Symptoms usually begin by the age of 6 …
Duchenne muscular dystrophy - Wikipedia
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. [3] [7] [8] The onset of muscle weakness typically begins around age four, with …
Duchenne Muscular Dystrophy - StatPearls - NCBI Bookshelf
Jul 10, 2023 · Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. It is the most common hereditary neuromuscular disease and does not …
About Duchenne Muscular Dystrophy - National Human …
Apr 18, 2013 · DMD is a rapidly progressive form of muscular dystrophy that occurs primarily in boys. It is caused by an alteration (mutation) in a gene, called the DMD gene that can be …
Duchenne Muscular Dystrophy - Johns Hopkins Medicine
Duchenne muscular dystrophy, or DMD, is associated with the most severe clinical symptoms of all the muscular dystrophies. It is caused by a genetic mutation on one of the mother’s X …
Duchenne Muscular Dystrophy: Symptoms, Treatment, and More
Jun 5, 2024 · Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disease that affects the muscles. Caused by a defective gene and first appearing in childhood, …
What To Know About Duchenne Muscular Dystrophy (DMD)
Feb 20, 2025 · Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscular weakness and mild intellectual disability.
Duchenne muscular dystrophy | About the Disease | GARD
Duchenne muscular dystrophy (DMD) affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily in males, though in rare cases may affect females. The …
Ashburn, VA Dentist | Joanna Claustro DMD
Joanna Claustro DMD specializes in dentist services such as dental exams, dental crowns, clear braces, and tmj therapy in Ashburn, VA and nearby areas.
Understanding Duchenne Muscular Dystrophy (DMD)
Learn about Duchenne muscular dystrophy (DMD), including its signs and symptoms, the stages of disease progression, and the important role of dystrophin.
Duchenne Muscular Dystrophy (DMD): Symptoms & Treatment - Cleveland Clinic
Jul 25, 2022 · Duchenne muscular dystrophy (DMD) is a condition that causes skeletal and heart muscle weakness that quickly gets worse with time. Symptoms usually begin by the age of 6 …
Duchenne muscular dystrophy - Wikipedia
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. [3] [7] [8] The onset of muscle weakness typically begins around age four, with …
Duchenne Muscular Dystrophy - StatPearls - NCBI Bookshelf
Jul 10, 2023 · Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. It is the most common hereditary neuromuscular disease and does not …
About Duchenne Muscular Dystrophy - National Human Genome …
Apr 18, 2013 · DMD is a rapidly progressive form of muscular dystrophy that occurs primarily in boys. It is caused by an alteration (mutation) in a gene, called the DMD gene that can be …
Duchenne Muscular Dystrophy - Johns Hopkins Medicine
Duchenne muscular dystrophy, or DMD, is associated with the most severe clinical symptoms of all the muscular dystrophies. It is caused by a genetic mutation on one of the mother’s X …
Duchenne Muscular Dystrophy: Symptoms, Treatment, and More
Jun 5, 2024 · Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disease that affects the muscles. Caused by a defective gene and first appearing in childhood, …
What To Know About Duchenne Muscular Dystrophy (DMD)
Feb 20, 2025 · Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscular weakness and mild intellectual disability.
Duchenne muscular dystrophy | About the Disease | GARD
Duchenne muscular dystrophy (DMD) affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily in males, though in rare cases may affect females. The …
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Understanding Duchenne Muscular Dystrophy (DMD)
Learn about Duchenne muscular dystrophy (DMD), including its signs and symptoms, the stages of disease progression, and the important role of dystrophin.
