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| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: Exploring Novel Clinical Trial Designs for Gene-Based Therapies National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Forum on Regenerative Medicine, 2020-08-27 Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop. |
| duchenne muscular dystrophy gene therapy clinical trials: 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 |
| duchenne muscular dystrophy gene therapy clinical trials: 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 |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: Rare Disease Drug Development Raymond A. Huml, 2021-11-08 This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers. |
| duchenne muscular dystrophy gene therapy clinical trials: Duchenne Muscular Dystrophy Alan E. H. Emery, Francesco Muntoni, Rosaline C. M. Quinlivan, 2015-02-19 Duchenne Muscular Dystrophy, an inherited and progressive muscle wasting disease, is one of the most common single gene disorders found in the developed world. In this fourth edition of the classic monograph on the topic, Alan Emery and Francesco Muntoni are joined by Rosaline Quinlivan, Consultant in Neuromuscular Disorders, to provide a thorough update on all aspects of the disorder. Recent understanding of the nature of the genetic defect responsible for Duchenne Muscular Dystrophy and isolation of the protein dystrophin has led to the development of new theories for the disease's pathogenesis. This new edition incorporates these advances from the field of molecular biology, and describes the resultant opportunities for screening, prenatal diagnosis, genetic counselling and from recent pioneering work with anti-sense oligonucleotides, the possibility of effective RNA therapy. Although there is still no cure for the disorder, there have been significant developments concerning the gene basis, publication of standards of care guidelines, and improvements in management leading to significantly longer survival, particularly with cardio-pulmonary care. The authors also investigate other forms of pharmacological, cellular and gene therapies. Duchenne Muscular Dystrophy will be essential reading not only for scientists and clinicians, but will also appeal to therapists and other professionals involved in the care of patients with muscular dystrophy. |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: NORD Guide to Rare Disorders National Organization for Rare Disorders, 2003 NORD Guide to Rare Disorders is a comprehensive, practical, authoritative guide to the diagnosis and management of more than 800 rare diseases. The diseases are discussed in a uniform, easy-to-follow format--a brief description, signs and symptoms, etiology, related disorders, epidemiology, standard treatment, investigational treatment, resources, and references.The book includes a complete directory of orphan drugs, a full-color atlas of visual diagnostic signs, and a Master Resource List of support groups and helpful organizations. An index of symptoms and key words offers physicians valuable assistance in finding the information they need quickly. |
| duchenne muscular dystrophy gene therapy clinical trials: Introduction to Basics of Pharmacology and Toxicology Gerard Marshall Raj, Ramasamy Raveendran, 2019-11-16 This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology. |
| duchenne muscular dystrophy gene therapy clinical trials: Heritable Human Genome Editing The Royal Society, National Academy of Sciences, National Academy of Medicine, International Commission on the Clinical Use of Human Germline Genome Editing, 2021-01-16 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. |
| duchenne muscular dystrophy gene therapy clinical trials: Registries for Evaluating Patient Outcomes Agency for Healthcare Research and Quality/AHRQ, 2014-04-01 This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews. |
| duchenne muscular dystrophy gene therapy clinical trials: 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 |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: 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 gene therapy clinical trials: Assessing Genetic Risks Institute of Medicine, Committee on Assessing Genetic Risks, 1994-01-01 Raising hopes for disease treatment and prevention, but also the specter of discrimination and designer genes, genetic testing is potentially one of the most socially explosive developments of our time. This book presents a current assessment of this rapidly evolving field, offering principles for actions and research and recommendations on key issues in genetic testing and screening. Advantages of early genetic knowledge are balanced with issues associated with such knowledge: availability of treatment, privacy and discrimination, personal decision-making, public health objectives, cost, and more. Among the important issues covered: Quality control in genetic testing. Appropriate roles for public agencies, private health practitioners, and laboratories. Value-neutral education and counseling for persons considering testing. Use of test results in insurance, employment, and other settings. |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: Spinal Muscular Atrophy Charlotte J Sumner, Sergey Paushkin, Chien-Ping Ko, 2016-10-24 Spinal Muscular Atrophy: Disease Mechanisms and Therapy provides the latest information on a condition that is characterized by motoneuron loss and muscle atrophy, and is the leading genetic cause of infant mortality. Since the identification of the gene responsible for SMA in 1995, there have been important advances in the basic understanding of disease mechanisms, and in therapeutic development. This book provides a comprehensive accounting of recent advances in basic and clinical research that covers SMA clinical features and standards of care, multifaceted aspects of SMN protein functions and SMA disease pathology, various animal models, and biomarkers, as well as current therapeutic development. This title is ideal for graduate students/postdocs and principal investigators who are already in the SMA field and need to keep updated on recent findings and approaches, and for those who are new to, or would like to join, the field. Likewise, users will find an excellent source of reading for biotech/pharma scientists, clinical researchers, and practitioners, regulators, and patients and their advocacy organizations. Furthermore, this book is a handy reference for researchers and clinicians who may want to apply the research strategies and therapeutic approaches in SMA to other rare diseases. - Provides comprehensive, up-to-date reviews by leading investigators on diverse topics of SMA, including clinical features and patient care, SMN genetics and protein functions, animal models, disease pathology and mechanisms, biomarkers, current therapeutic development, and the role of non-profit organizations in therapeutic development - Written to bridge multiple disciplines and promote better communications among basic scientists, clinical researchers, and health care providers on the latest developments in SMA - Includes outstanding questions and perspectives for future investigations and key references for additional detailed study |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: AAV Gene Therapy: Immunology and Immunotherapeutics Jose Martinez-Navio, Nicole K. Paulk, Guangping Gao, 2022-02-09 Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma. The other Topic Editors declare no conflict of interest with regards to the Research Topic theme |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: The Sum of Small Things Elizabeth Currid-Halkett, 2017-05-15 How the leisure class has been replaced by a new elite, and how their consumer habits affect us all In today’s world, the leisure class has been replaced by a new elite. Highly educated and defined by cultural capital rather than income bracket, these individuals earnestly buy organic, carry NPR tote bags, and breast-feed their babies. They care about discreet, inconspicuous consumption—like eating free-range chicken and heirloom tomatoes, wearing organic cotton shirts and TOMS shoes, and listening to the Serial podcast. They use their purchasing power to hire nannies and housekeepers, to cultivate their children’s growth, and to practice yoga and Pilates. In The Sum of Small Things, Elizabeth Currid-Halkett dubs this segment of society “the aspirational class” and discusses how, through deft decisions about education, health, parenting, and retirement, the aspirational class reproduces wealth and upward mobility, deepening the ever-wider class divide. Exploring the rise of the aspirational class, Currid-Halkett considers how much has changed since the 1899 publication of Thorstein Veblen’s Theory of the Leisure Class. In that inflammatory classic, which coined the phrase “conspicuous consumption,” Veblen described upper-class frivolities: men who used walking sticks for show, and women who bought silver flatware despite the effectiveness of cheaper aluminum utensils. Now, Currid-Halkett argues, the power of material goods as symbols of social position has diminished due to their accessibility. As a result, the aspirational class has altered its consumer habits away from overt materialism to more subtle expenditures that reveal status and knowledge. And these transformations influence how we all make choices. With a rich narrative and extensive interviews and research, The Sum of Small Things illustrates how cultural capital leads to lifestyle shifts and what this forecasts, not just for the aspirational class but for everyone. |
| duchenne muscular dystrophy gene therapy clinical trials: 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 |
| duchenne muscular dystrophy gene therapy clinical trials: 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 |
| duchenne muscular dystrophy gene therapy clinical trials: 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 |
| duchenne muscular dystrophy gene therapy clinical trials: Principles of Good Clinical Practice Michael J. McGraw, 2010 Part of RPS Pharmacy Business Administration Series, this book offers good clinical practice guidelines. It includes standards on how clinical trials should be conducted, provide assurance of safety and efficacy of various drugs and protect human rights. |
| duchenne muscular dystrophy gene therapy clinical trials: Muscular Dystrophy National Institute of Neurological Diseases and Stroke, 1971 |
| duchenne muscular dystrophy gene therapy clinical trials: Muscle Disorders in Childhood Victor Dubowitz, 1995 This reference focuses on the clinical assessment, diagnosis, management, and prognosis of all forms of muscle diseases that affect children. Includes a readable account of relevant genetics, biochemistry, and molecular biology, in addition to numerous case histories. |
| duchenne muscular dystrophy gene therapy clinical trials: Fundamental Virology Peter M. Howley, Diane E. Griffin, 2001 Designed for graduate students and researchers in all biological and biomedical sciences, this volume brings together the basic science chapters from the two-volume Fourth Edition of Fields Virology. These 37 chapters comprise a comprehensive text and reference on the concepts and research techniques of contemporary virology and the biochemistry, molecular biology, and replication of all viruses. The first part of the book covers basic concepts of general virology and the second part focuses on specific virus families. |
| duchenne muscular dystrophy gene therapy clinical trials: Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function William Duddy, Stephanie Duguez, 2021 This compilation focuses on recent advances in the molecular and cellular understandingof neuromuscular biology, and the treatment of neuromuscular disease.These advances are at the forefront of modern molecular methodologies, oftenintegrating across wet-lab cell and tissue models, dry-lab computational approaches,and clinical studies. The continuing development and application ofmultiomics methods offer particular challenges and opportunities in the field,not least in the potential for personalized medicine. |
| duchenne muscular dystrophy gene therapy clinical trials: Muscle Biopsy Victor Dubowitz, 1985 |
| duchenne muscular dystrophy gene therapy clinical trials: Muscle Gene Therapy Dongsheng Duan, Jerry R. Mendell, 2019-03-30 About 7 million people worldwide are suffering from various inherited neuromuscular diseases. Gene therapy brings the hope of treating these diseases at their genetic roots. Muscle Gene Therapy is the only book dedicated to this topic. The first edition was published in 2010 when the field was just about to enter its prime time. The progress made since then has been unprecedented. The number of diseases that have been targeted by gene therapy has increased tremendously. The gene therapy toolbox is expanded greatly with many creative novel strategies (such as genome editing and therapy with disease-modifying genes). Most importantly, clinical benefits have begun to emerge in human patients. To reflect rapid advances in the field, we have compiled the second edition of Muscle Gene Therapy with contributions from experts that have conducted gene therapy studies either in animal models and/or in human patients. The new edition offers a much needed, up-to-date overview and perspective on the foundation and current status of neuromuscular disease gene therapy. It provides a framework to the development and regulatory approval of muscle gene therapy drugs in the upcoming years. This book is a must-have for anyone who is interested in neuromuscular disease gene therapy including those in the research arena (established investigators and trainees in the fields of clinical practice, veterinary medicine and basic biomedical sciences), funding and regulatory agencies, and patient community. |
| duchenne muscular dystrophy gene therapy clinical trials: Pulmonary Function Testing in Children: Techniques and Standards George Polgar, Promadhat Varuni, 1971 |
| duchenne muscular dystrophy gene therapy clinical trials: Treating Spasticity C. David Marsden, International Medical Society of Motor Disturbances. Congress, 1989 |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
| duchenne muscular dystrophy gene therapy clinical trials: Immunology, Infection, and Immunity Jeffrey B. Lyczak, Lee M. Wetzler, 2004 |
| duchenne muscular dystrophy gene therapy clinical trials: 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. |
AAV gene therapy for Duchenne muscular dystrophy: the …
Oct 9, 2024 · Here we report results from Part 1 (52 weeks) of EMBARK (ClinicalTrials.gov: NCT05096221), a large, phase 3, two-part, multinational, randomized, double-blind, placebo …
Duchenne muscular dystrophy: Five trials to watch - Clinical Trials …
Mar 26, 2025 · Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five DMD trials to keep an eye on in 2025. Research by …
FDA Approves First Gene Therapy for Treatment of Certain …
FDA Approves Elevidys, a gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene …
AAV gene therapy for Duchenne muscular dystrophy: the …
Delandistrogene moxeparvovec, an adeno-associated rh74-viral vector-based gene therapy, addresses absent functional dystrophin in DMD. Here the phase 3 EMBARK study aimed to …
[Ad hoc announcement pursuant to Art. 53 LR] Roche provides
1 day ago · A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is …
Duchenne muscular dystrophy gene therapy | ScienceDaily
May 14, 2025 · To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, …
Duchenne Muscular Dystrophy Clinical Trials - Rare Disease …
Aug 24, 2020 · A gene transfer therapy study to evaluate the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) following imlifidase infusion in participants with …
REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM …
Jun 5, 2025 · RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or …
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
Aug 21, 2023 · This newly approved gene therapy delivers a copy of a gene that encodes a shortened, functional form of dystrophin, the gene that is mutated in DMD patients. Dystrophin …
The Triumphs And Trials Of Duchenne Gene Therapy - Forbes
3 days ago · Earlier this year, Sarepta Therapeutics reported the first death linked to its gene therapy, Elevidys, used to treat Duchenne muscular dystrophy. The patient, a 16-year-old boy, …
AAV gene therapy for Duchenne muscular dystrophy: the …
Oct 9, 2024 · Here we report results from Part 1 (52 weeks) of EMBARK (ClinicalTrials.gov: NCT05096221), a large, phase 3, two-part, multinational, randomized, double-blind, placebo …
Duchenne muscular dystrophy: Five trials to watch - Clinical Trials …
Mar 26, 2025 · Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five DMD trials to keep an eye on in 2025. Research by …
FDA Approves First Gene Therapy for Treatment of Certain …
FDA Approves Elevidys, a gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene …
AAV gene therapy for Duchenne muscular dystrophy: the …
Delandistrogene moxeparvovec, an adeno-associated rh74-viral vector-based gene therapy, addresses absent functional dystrophin in DMD. Here the phase 3 EMBARK study aimed to …
[Ad hoc announcement pursuant to Art. 53 LR] Roche provides
1 day ago · A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is …
Duchenne muscular dystrophy gene therapy | ScienceDaily
May 14, 2025 · To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, …
Duchenne Muscular Dystrophy Clinical Trials - Rare Disease …
Aug 24, 2020 · A gene transfer therapy study to evaluate the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) following imlifidase infusion in participants with …
REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA …
Jun 5, 2025 · RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or …
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
Aug 21, 2023 · This newly approved gene therapy delivers a copy of a gene that encodes a shortened, functional form of dystrophin, the gene that is mutated in DMD patients. Dystrophin …
The Triumphs And Trials Of Duchenne Gene Therapy - Forbes
3 days ago · Earlier this year, Sarepta Therapeutics reported the first death linked to its gene therapy, Elevidys, used to treat Duchenne muscular dystrophy. The patient, a 16-year-old boy, …
