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Muscular Dystrophy Therapeutics


Muscular Dystrophy Therapeutics
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Muscular Dystrophy Therapeutics


Muscular Dystrophy Therapeutics
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Author : Rika Maruyama
language : en
Publisher: Springer Nature
Release Date : 2022-11-18

Muscular Dystrophy Therapeutics written by Rika Maruyama and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-11-18 with Medical categories.


This detailed book presents a comprehensive collection of state-of-the-art protocols on muscular dystrophy therapeutics, covering therapeutics using antisense oligonucleotides, gene replacement, genome editing, small molecules, stem cells, and antibodies. Written by leaders in the field, the volume explores techniques that are currently in use and are starting an exciting therapeutic revolution in muscular dystrophy. As a part of 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 and readily reproducible laboratory protocols, as well as tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Muscular Dystrophy Therapeutics: Methods and Protocols serves as an ideal resource to inspire readers and provide tips, strategies, and advice to develop new therapeutic technologies for this group of diseases.



Duchenne Muscular Dystrophy


Duchenne Muscular Dystrophy
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Author : Jeffrey S. Chamberlain
language : en
Publisher: CRC Press
Release Date : 2006-02-27

Duchenne Muscular Dystrophy written by Jeffrey S. Chamberlain and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2006-02-27 with Medical categories.


Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of childhood for which there is no cure. This authoritative guide provides a clear overview of the clinical, genetic, and pathophysiological aspects of the disease in the context of emerging therapeutic modalities. The only available source on the subject, this reference emphasizes the importance of accurate diagnosis, carrier detection, and genetic counseling, and supplies state-of-the-art information on pharmacological interventions, regenerative medicine, and gene therapy.



Muscular Dystrophy


Muscular Dystrophy
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Author : Gisela Gaina
language : en
Publisher: BoD – Books on Demand
Release Date : 2020-12-23

Muscular Dystrophy written by Gisela Gaina and has been published by BoD – Books on Demand this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-12-23 with Technology & Engineering categories.


Muscular Dystrophy - Research Update and Therapeutic Strategies is for students, researchers, and clinicians interested in muscular dystrophies who want to improve their knowledge of these complex genetic diseases. The book includes information about the genetics of various types of muscular dystrophies as well as explores new and current therapeutic strategies that aim to ameliorate symptoms and improve patients’ quality of life and life expectancy. In addition, this book reviews information on current clinical trials for muscular dystrophies and presents a framework for what to consider during the design of these trials.



Duchenne Muscular Dystrophy


Duchenne Muscular Dystrophy
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Author : Jeffrey S. Chamberlain
language : en
Publisher: CRC Press
Release Date : 2006-02-27

Duchenne Muscular Dystrophy written by Jeffrey S. Chamberlain and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2006-02-27 with Medical categories.


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



Muscle Gene Therapy


Muscle Gene Therapy
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Author : Dongsheng Duan
language : en
Publisher: Springer
Release Date : 2019-03-30

Muscle Gene Therapy written by Dongsheng Duan and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-03-30 with Medical categories.


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


Duchenne Muscular Dystrophy
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Author : Radenka Kuzmanić Šamija
language : en
Publisher:
Release Date : 2018

Duchenne Muscular Dystrophy written by Radenka Kuzmanić Šamija and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018 with Electronic books categories.


Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are dystrophinopathies, a group of muscular dystrophies caused by mutations in the dystrophin gene. Duchenne muscular dystrophy is the most common muscular dystrophy that occurs in children. A mutation in the DMD gene leads to a loss of expression of the dystrophin protein, a subsarcolemmal protein that provides strength, stability, and functionality to the myofibrils. Patients with dystrophinopathies with basic progressive weakness of the musculoskeletal system develop complications of many organ systems that significantly contribute to the deterioration of the clinical condition and shorter life expectancy. Multidisciplinary care has extended the patients,Äô life expectancy and the development of subspecialist branches has enabled the improvement of diagnostic methods and treatment. Recently, therapeutic options in the treatment of DMD have advanced significantly, and new genetic and molecular therapies are emerging. The advent of gene therapy as a causal therapy for DMD has placed additional emphasis on diagnosing and treating the disease as early as possible. This achieves an additional prolongation of life expectancy, increases the quality of life in patients with DMD, and provides hope for patients and their families.



Muscle Gene Therapy


Muscle Gene Therapy
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Author : Dongsheng Duan
language : en
Publisher: Humana Press
Release Date : 2011-01-10

Muscle Gene Therapy written by Dongsheng Duan and has been published by Humana Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011-01-10 with Medical categories.


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.



Potential Therapeutic Strategies For Muscular Dystrophy


Potential Therapeutic Strategies For Muscular Dystrophy
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Author : Gisela Gaina
language : en
Publisher: BoD – Books on Demand
Release Date : 2023-11-02

Potential Therapeutic Strategies For Muscular Dystrophy written by Gisela Gaina and has been published by BoD – Books on Demand this book supported file pdf, txt, epub, kindle and other format this book has been release on 2023-11-02 with Medical categories.


The book provides a comprehensive overview of the different forms of muscular dystrophy (MD), including potential therapeutic interventions and the advantages associated with repurposing pharmaceuticals for treating these conditions. In addition, the book examines the effects of physical training on improving symptoms as well as patient quality of life and life span. The target audience of this book comprises students, researchers, and doctors with an interest in the field of muscular dystrophy.



Regenerative Medicine For Degenerative Muscle Diseases


Regenerative Medicine For Degenerative Muscle Diseases
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Author : Martin K. Childers
language : en
Publisher: Humana Press
Release Date : 2015-12-19

Regenerative Medicine For Degenerative Muscle Diseases written by Martin K. Childers and has been published by Humana Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2015-12-19 with Science categories.


This book compiles and explores cutting-edge research in degenerative skeletal disorders, such as Duchenne muscular dystrophy and congenital myopathy, and new stem-cell based therapies and gene replacement therapy. Twelve expertly-authored chapters navigate the nuances of these treatments in an array of contexts and biological systems. The topics covered include: How are urine cells from a patient with Duchenne muscular dystrophy transformed into beating heart cells? What can reprogrammed cells tell us about heart muscle failure? What do gene mutations mean for those born with a muscle disease? How are manufacturing methods applied to human stem cells? Does therapeutic exercise benefit those patients who receive engineered limb muscle? Is there practical advice about nutrition to enhance muscle function for the Duchenne patient? Can microRNAs be useful to regenerate diseased muscle? Regenerative Medicine for Degenerative Muscle Diseases is ideal for scientists and clinicians from varying disciplines in genetics, cell biology, virology, cell-based manufacturing, rehabilitation medicine, nutrition, veterinary medicine and neurosurgery. The reader will see how transformative changes occur in medicine that can powerfully impact the future for patients suffering from inherited disorders affecting muscles of the body, including the heart.



Delineation Of Duchenne Muscular Dystrophy Gene Therapy Using Genetically Engineered Mice


Delineation Of Duchenne Muscular Dystrophy Gene Therapy Using Genetically Engineered Mice
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Author : Nalinda B. Wasala
language : en
Publisher:
Release Date : 2016

Delineation Of Duchenne Muscular Dystrophy Gene Therapy Using Genetically Engineered Mice written by Nalinda B. Wasala and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2016 with categories.