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| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene 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 |
| duchenne muscular dystrophy gene 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 |
| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene therapy: 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: 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: 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: A Guide to Human Gene Therapy Roland W. Herzog, Sergei Zolotukhin, 2010 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne |
| duchenne muscular dystrophy gene therapy: Occupational Therapy and Duchenne Muscular Dystrophy Kate Stone, Claire Tester, Joy Blakeney, Alex Howarth, Hether McAndrew, Nicola Traynor, Mary McCutcheon, Ruth Johnston, 2007-09-27 This is a practical guide for Occupational Therapists and others who provide services to people who have Duchenne muscular dystrophy. The goal of the book is to deepen the readers understanding of the support that occupational therapists can offer to families who are affected by this condition. Written by experienced occupational therapists with a special interest in Duchenne muscular dystrophy. The book will provide information and support to therapists who may be working in isolation, or who rarely work with this client group. It is for the experienced therapist as well as the student. Each chapter of this book gives a clear and comprehensive description of different aspects of occupational therapy assessments and interventions. |
| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene therapy: 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: 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: 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: 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: Gene Therapy David Cooper, Prof Nick Lemoine, 2020-09-10 Gene Therapy describes the delivery systems now available to target a given tissue with specific gene or oligonucleotide sequences, and explores the utility of animal modules as test systems. In the context of selected disease states, it summarises in vitro and in vivo studies and clinical trials performed to date. |
| duchenne muscular dystrophy gene 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 |
| duchenne muscular dystrophy gene therapy: 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: 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: 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: 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: 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: 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: 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: 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: Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies (Second Edition) Daniel Scherman, 2019-07-16 This unique advanced textbook provides a clear and comprehensive overview of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations. The second edition features new groundbreaking material on genome editing using the recently discovered CRISPR/Cas9 system and on cancer immunotherapy by CAR-T cells. It also presents the historical milestone of gene therapy application in the field of severe combined immunodeficiency, and other fields of gene therapy and molecular medicine.The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of gene therapy, which is already revolutionizing medicine. In this advanced textbook, more than 30 leading scientists come together to explore these topics.This educational introduction provides the background material needed to further explore the subject as well as relevant research literature. It is an invaluable resource to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide or develop a gene therapy strategy. The second edition's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome. |
| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene 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 |
| duchenne muscular dystrophy gene therapy: Frontiers in Human Genetics Eric Yap, 2001 With the completion of human genome sequencing, human genetics is poised for major developments in functional genomics, molecular diagnostics, pathogenesis of complex multifactorial diseases and gene-based therapy. This book was conceived from an international symposium on human genetics and gene therapy that was held in Singapore in 1999. It includes manuscripts of the work presented as well as articles written by a selection of young researchers in the Asia Pacific region who are actively involved in a diverse range of medical problems, including cancer, infections, hypertension and myopia. New technologies being developed in gene therapy, lab-on-chips and bioinformatics are reported. The book provides a snapshot of the diverse approaches and solutions being developed at the frontiers of human genetics. It will be useful to researchers and students in molecular genetics and the life sciences, professionals in the biotechnology and pharmaceutical industries, clinicians who are interested in molecular medicine, etc. |
| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene therapy: Dystrophin Susan C. Brown, Jack A. Lucy, 1997-05-13 The absence of dystrophin is the underlying cause of Duchenne muscular dystrophy. This is the first book to address the structure, function, and distribution of dystrophin and its associated proteins in muscle and brain, rather than concentratiing primarily on the disease. It covers an exciting and rapidly expanding field that promises to have important and widespread implications for many aspects of cell biology. |
| duchenne muscular dystrophy gene therapy: 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: Gene Therapy in Neurological Disorders Mingjie Li, B. Joy Snider, 2018-05-25 Gene therapy has tremendous potential for the treatment of neurological disorders. There has been substantial progress in the development of gene therapy strategies for neurological disorders over the last two decades. Gene Therapy in Neurological Disorders thoroughly reviews currently available gene therapy tools and presents examples of their application in a variety of neurological diseases. The book begins with general reviews of gene therapy strategies with a focus on neurological disorders. The remainder of the chapters present approaches to specific neurological disorders. Each chapter gives an in-depth introduction to the relevant field before diving into the specific tool or application. The book aims to help investigators, students and research staff better understand the principles of gene therapy and its application in the nervous system. - Provides background information and experimental details of gene therapy tools applied for neuroscience research and neurological disorders - Covers a broad range of gene delivery and regulation tools, therapeutic agents, and target cells, including emerging new technologies such as CRISPR/Cas9 genome editing - Discusses applications of gene therapy tools to neurological disorders including neurodegeneration, muscular dystrophy, trauma and chronic pain, and neoplastic diseases |
| duchenne muscular dystrophy gene 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. |
| duchenne muscular dystrophy gene therapy: Viral Vectors for Gene Therapy Curtis A. Machida, 2008-02-02 Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdoctoral training. The “Notes” section contained in each chapter provides valuable troublesho- ing guides to help develop working protocols for your laboratory. With Viral Vectors for Gene Therapy: Methods and Protocols, it has been my intent to develop a comprehensive collection of modern molecular methods for the construction, development, and use of viral vectors for gene transfer and gene therapy. I would like to thank the many chapter authors for their contributions. They are all experts in various aspects of viral vectors, and I appreciate their efforts and hard work in developing comprehensive chapters. As editor, it has been a privilege to preview the development of Viral Vectors for Gene Therapy: Methods and Protocols, and to acquire insight into the various methodological approaches from the many different contri- tors. |
| duchenne muscular dystrophy gene therapy: 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: Muscle Gene Therapy Dongsheng Duan, 2011-01-10 Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies; however, it faces a number of unique challenges, including the need to deliver a therapeutic vector to all muscles throughout the body. In Muscle Gene Therapy: Methods and Protocols, expert researchers in the field present a collection of techniques aimed at bridging the translational gap in muscle gene therapy between the prevalent rodent models and vitally important larger animal models. Divided into three sections, this volume examines basic protocols for optimizing the muscle gene expression cassette and for evaluating the therapeutic outcomes, new developments in muscle gene therapy technology such as adeno-associated viral vector (AAV), oligonucleotide-mediated exon-skipping, and novel RNA-based strategies, and step-by-step guidance on muscle gene delivery in swine, ovine, canine, and non-human primates. Written in the highly successful Methods in Molecular BiologyTM series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, detailed, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Muscle Gene Therapy: Methods and Protocols serves as an invaluable resource for graduate students, post-doctoral fellows, and principle investigators pursuing the crucial advancement of muscle disease gene therapy in the hope of someday curing these debilitating disorders. |
| duchenne muscular dystrophy gene therapy: Brain and Heart Dynamics Stefano Govoni, Pierluigi Politi, Emilio Vanoli, 2020-07-31 This ambitious and comprehensive handbook represents an essential contribution to our current understanding of interactions between heart and brain, a research topic generating growing interest. Despite the increasing awareness that neural mechanisms are the primary cause of cardiac disease and its progression, therapy continues to focus on end-organ protection and does not approach the neural core of the problem. Growing public health problems such as heart failure are still treated with autonomic drugs that are 30-40 years old and simply act on cardiac receptors. However, it has now been shown that the progression of ischemic heart disease to heart failure is mainly due to abnormal central responses to incipient cardiac disease, with neural activation the primary cause rather than the consequence of cardiac remodeling. Written by leading international experts in their respective research areas, the book presents a variety of perspectives on the core topic: from social and philosophical to gender-related aspects. It is designed for a broad readership and includes dedicated sections for cardiologists, psychiatrists, neurologists and psychotherapists looking for a more insightful and targeted approach to neuro-cardiovascular disease. |
| duchenne muscular dystrophy gene therapy: Muscle Biopsy Victor Dubowitz, 1985 |
Gene Therapy for Duchenne Muscular Dystrophy - PMC
So far, there is no effective treatment for deteriorating muscle function in DMD patients. A promising approach for treating this life-threatening disease is gene transfer to restore …
FDA Approves First Gene Therapy for Treatment of Certain …
Today, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy …
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, …
New Gene Therapy for Duchenne Muscular Dystrophy
Oct 23, 2024 · A new single-dose intravenous gene therapy, delandistrogene moxeparvovec, creates “micro-dystrophin” — a scaled-down version of the large gene that encodes dystrophin …
AAV gene therapy for Duchenne muscular dystrophy: the …
Oct 9, 2024 · Delandistrogene moxeparvovec is a single-administration recombinant adeno-associated virus rhesus isolate serotype 74 (rAAVrh74) vector-based gene transfer therapy, …
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 …
Gene Therapy for Duchenne Muscular Dystrophy | Children's …
On Thursday, June 22, the U.S. Food and Drug Administration (FDA) approved Elevidys, the first gene therapy for Duchenne muscular dystrophy (DMD), for patients with the disease between …
The FDA and Gene Therapy for Duchenne Muscular Dystrophy
May 1, 2024 · The US Food and Drug Administration (FDA) grants accelerated approval that includes the standard of a surrogate outcome that is “reasonably likely” to predict clinical …
Duchenne muscular dystrophy gene therapy | ScienceDaily
May 14, 2025 · Delandistrogene moxeparvovec is a one-time gene therapy infusion approved by the Food and Drug Administration in June 2024 for use in those age four and older. The …
Life-Changing New Gene Therapy For Duchenne Muscular Dystrophy
May 21, 2025 · Elevidys Ⓡ (delandistrogene moxeparvovec) is the only FDA approved gene therapy for any muscular dystrophy and can only be used for children age 4 years and older …
HIGHLIGHTS OF PRESCRIBING INFORMATION
ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated in individuals at least 4 years of age: during administration and for at least 3 hours after end of infusion. • For …
June 15, 2025 Sarepta Community Letter: Safety Update …
Dear Duchenne Community, At Sarepta, our mission is rooted in a deep commitment to the Duchenne community and to the safety and well-being of every individual we serve. Today, we …
HIGHLIGHTS OF PRESCRIBING INFORMATION - U.S. Food and …
ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated in individuals at least 4 years of age: • For the treatment of Duchenne muscular dystrophy (DMD) in
Gene Therapy for Duchenne Muscular Dystrophy - SAGE …
Gene Therapy for Duchenne Muscular Dystrophy Nertiyan Elangkovan and George Dickson∗ Centres for Gene & Cell Therapy and Biomedical Sciences, Department of Biological …
What is Duchenne Muscular Dystrophy? A DMD Fact Sheet
MUSCULAR . DYSTROPHY. Duchenne muscular dystrophy (DMD) is the . most common form of muscular dystrophy. It is ... Elevidys* is the first FDA approved gene therapy . to treat DMD. …
2024 New Directions in Biology and Disease of Skeletal …
Apr 5, 2024 · SGT-003, a Next Generation Microdystrophin Gene Therapy for Treatment of Duchenne ... The Role of Utrophin in the Therapy of Duchenne Muscular Dystrophy . 6:15 – …
What is Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the ... In Duchenne, a mutation in the DMD gene interferes with the production of the dystrophin protein, which is needed to form and maintain …
DONGSHENG DUAN’S PUBLICATIONS Books - MU School of …
dystrophin expression as a surrogate endpoint for Duchenne muscular dystrophy clinical trials. Human Gene Therapy 2023. In-press. • Wasala LP, Watkin TB, Wasala NB, Burke MJ, Yue Y, …
Practical considerations for delandistrogene moxeparvovec …
Practical considerations for delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy JR Mendell,1,2 C Proud,3* C Zaidman,4 S Mason,5 E Darton,5 …
Assessment of systemic AAV-microdystrophin gene therapy …
Jan 4, 2023 · Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystro- phin, a membrane-stabilizing protein encoded by the DMD …
Systemicdeliveryoffull-lengthdystrophinin Duchenne …
Duchenne muscular dystrophy mice Yuan Zhou 1,2, Chen Zhang1, Weidong Xiao 1,RolandW.Herzog1 & ... independent FL-dystrophin gene therapy for DMD, warranting further …
High hopes and high costs: Exploring advances in Duchenne …
advances in Duchenne muscular dystrophy gene therapy May 14 2025 Credit: Pixabay/CC0 Public Domain To help neurologists, clinicians and families understand the current
FDA expands approval for Duchenne muscular dystrophy …
muscular dystrophy eligible for this therapy, helping to address the ongoing, urgent treatment need for patients with this devastating and life- threatening disease," Peter Marks, M.D., Ph.D ...
Gene Therapy Strategies for Duchenne Muscular Dystrophy …
Gene Therapy Strategies for Duchenne Muscular Dystrophy Utilizing Recombinant Adeno-associated Virus Vectors Michael J. Blankinship,1 Paul Gregorevic,1 and Jeffrey S. …
RGX-202, an investigational gene therapy for the treatment …
RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy: interim clinical data Aravindhan Veerapandiyan, M.D. Arkansas Children’s Hospital RGX-202 …
Selected Oral Abstracts - asgct.org
Duchenne Muscular Dystrophy Niclas Bengtsson1, Stephen Hauschka2 1University of Washington, Seattle, Washington, 2University of Washington, Seattle, WA Our longtime …
Gene therapy: Microdystrophin restores muscle strength in …
therapy in the treatment of Duchenne muscular dystrophy. Indeed, after injecting microdystrophin (a “shortened” version of the dystrophin gene) via a drug vector, the researchers managed to …
Potential limitations of microdystrophin gene therapy for …
gene therapy for Duchenne muscular dystrophy Cora C. Hart,1,2 Young il Lee,1,2 Jun Xie, 3 Guangping Gao, Brian L. Lin,4 David W. Hammers,1,2 and H. Lee Sweeney1,2 1Department …
Gene therapeutic strategies and relevant clinical trials in ...
Neuromuscular disorder is a diverse group of genetic disease, among which Duchenne muscular dystrophy and Spinal ... critical development of gene therapy in this field, which might be …
CRITERIA FOR PRIOR AUTHORIZATION - kdhe.ks.gov
See the Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD) ClinicalTrials.gov Identifier: NCT01603407.10 References: 1. Gloss D, Moxley RT, Ashwal S, …
New gene therapy for muscular dystrophy offers hope
A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but …
Genethon Presents Positive Initial Results from a Phase 1/2/3 …
of its Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy at ASGCT Breakthroughs in Muscular Dystrophy in Chicago • Based on positive safety and efficacy data in the Phase 1/2 …
Effective Date: 08/08/2024 - BCBSM
- Duchenne muscular dystrophy (DMD) is a rare, life-limiting, progressive childhood disease that affects 1 in 3,500 – ... - Elevidys is an adeno-associated virus vector-based gene therapy …
Assessment of systemic AAV-microdystrophin gene therapy …
Jan 4, 2023 · Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystro- phin, a membrane-stabilizing protein encoded by the DMD …
Muscular Dystrophy – Gene Therapy – Elevidys - Cigna
Muscular Dystrophy – Gene Therapy – Elevidys ... Duchenne Muscular Dystrophy (DMD). Approval is not recommended due to the unclear clinical benefit of Elevidys. Elevidys clinical …
Duchenne Muscular Dystrophy
Gene Therapy and its Application for Duchenne Muscular Dystrophy Gene therapy is a medical intervention that treats or prevents disease by correcting, modifying, or replacing genes, as …
DigitalCommons@KCU - Kansas City University
gene is 2.3 Mb in humans with 79 exons, producing a 14 kb RNA and 427 kDa protein [5,7,8]. One-third of DMD cases are due to a . de novo. mutation, and two-thirds of cases are of …
Japan approves Sarepta's gene therapy to treat Duchenne …
May 14, 2025 · Japan approves Sarepta's gene therapy to treat Duchenne Muscular Dystrophy 14 May 2025 | News Company is eligible to receive up to $103.5 M in near-term regulatory and …
Duchenne muscular dystrophy: pathogenesis and promising …
Keywords Duchenne muscular dystrophy · Dystrophin · Muscle atrophy · Therapies Introduction Duchenne muscular dystrophy (DMD) is an X-linked reces-sive disorder characterized by …
RGX-202, an Investigational Gene Therapy for the Treatment …
RGX-202 is the only gene therapy designed to deliver a transgene for a microdystrophin with the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin …
Dystrophin Immunity after Gene Therapy for Duchenne’s …
The new england journal of medicine n engl j med 388;24 nejm.org June 15, 2023 Correspondence Dystrophin Immunity after Gene Therapy for Duchenne’s Muscular Dystrophy …
Duchenne Muscular Dystrophy: Experimental models on …
Duchenne Muscular Dystrophy: ... Physical therapy (PT) has been used to reduce muscular, cardiac and vascular abnormalities ... cell or gene therapy to replace the defective dystrophin …
SGT-001 Microdystrophin Gene Therapy for Duchenne …
SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy Patrick Gonzalez, PhD. Associate Director, R&D, Solid Biosciences
A Roadmap to Newborn Screening for Duchenne Muscular …
Duchenne; newborn screening; gene therapy; muscular dystrophy This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC …
Gene therapy progress and prospects: Duchenne muscular …
Duchenne muscular dystrophy gene therapy K Foster et al 1678 Gene Therapy. associated damage. Levels of up to 60% transfection can be achieved with a reporter gene in the mouse …
Evaluation of rAAVrh74 gene therapy vector seroprevalence …
and is being investigated for use in gene therapy for Duchenne muscular dystrophy (DMD). Objective: To evaluate prevalence of total binding antibodies (neutralizing and non …
What is Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy. It is a genetic disorder ... In Duchenne, a mutation in the DMD gene interferes with the production of …
Reassessing the acceptability of risk of death associated with …
In 2017/2018, Parent Project Muscular Dystrophy (PPMD) & RTI International conducted a study on risk tolerance for gene therapy (GT) for Duchenne muscular dystrophy (Duchenne). We …
The new challenge of exercise + muscular dystrophy
duchenne muscular dystrophy, DMD, exercise, combined therapy, mdx 1 Duchenne muscular dystrophy overview DMD is an incurable fatal muscular disease with X-linked recessive …
Delandistrogene Moxeparvovec Gene Therapy in Individuals …
Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus Report of the AAN Guidelines Subcommittee Maryam Oskoui,1 Tracie …
Pharmacologic Treatment of Duchenne Muscular Dystrophy
(golodirsen) are new drugs used for Duchenne Muscular Dystrophy (DMD), which is a rare genetic disorder characterized by progressive muscle degeneration and weakness. It is …
Genethon’s Scientists Featured in Multiple Presentations
model of Duchenne muscular dystrophy" – Anne Galy, Ph.D., will introduce the design of tools that generate fibrosis-specific killer CAR T cells to reduce fibrosis of the muscle tissues and …
Gene therapy as a potential therapeutic option for …
active Duchenne muscular dystrophy gene therapy trial; Katherine Beaverson is an employee of Pfizer, Inc; Sharon Hesterlee was an employee of Pfizer Inc, at the time of the research and is …
Pfizer Provides Update on Phase 3 Study of Investigational …
Jun 12, 2024 · Boys with Duchenne Muscular Dystrophy Wednesday, June 12, 2024 - 04:30pm NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) today announced that CIFFREO, …
Clinician Perspectives of Gene Therapy Treatment Option for …
Background: Duchenne muscular dystrophy (DMD) is a progressive, life-limiting, neuromuscular disorder. Clinicians play Clinicians play an important role in informing families about therapy …
Systemic AAV Micro-dystrophin Gene Therapy for …
laboratory.1,15,16 Thefull-lengthdystrophin geneis2.6mb.Itencodes 79 exons. The 11.5-kb coding sequence translates into a 427-kD pro-tein. Dystrophin can be divided into four major …
A guide to genetic testing in Duchenne muscular dystrophy
Duchenne muscular dystrophy ©2017 Sarepta Therapeutics, Inc. 215 First Street, Cambridge, MA 02142. ... The goal of a genetic test is to identify changes in a gene that may cause a disease. …
Potential limitations of micro-dystrophin gene therapy for …
3 53 INTRODUCTION 54 Duchenne muscular dystrophy (DMD) is an X-linked disorder that affects approximately 1 55 in 5000 newborn males (1). It is the most common of the childhood …
Pfizer Presents Initial Clinical Data on Phase 1b Gene Therapy …
investigational gene therapy. Other objectives of the clinical study include measurement of dystrophin expression and distribution, as well as assessments of muscle strength, quality and …
