Muscle Gene Therapy

Author : Dongsheng Duan
Publisher : Springer Science & Business Media
Page : 281 pages
File Size : 45,5 Mb
Release : 2009-11-26
Category : Medical
ISBN : 9781441912077

Get Book

Muscle Gene Therapy by Dongsheng Duan Pdf

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.

Author : Dongsheng Duan
Publisher : Humana Press
Page : 382 pages
File Size : 45,8 Mb
Release : 2016-08-23
Category : Medical
ISBN : 1493961829

Get Book

Muscle Gene Therapy by Dongsheng Duan Pdf

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.

Author : Rudolf Jaenisch,Feng Zhang,Fred Gage
Publisher : Springer
Page : 0 pages
File Size : 48,8 Mb
Release : 2018-08-11
Category : Medical
ISBN : 3319868012

Get Book

Genome Editing in Neurosciences by Rudolf Jaenisch,Feng Zhang,Fred Gage Pdf

This book is open access under a CC BY 4.0 license. CRISPR-Cas9 is a rapid, efficient, versatile and relatively cheap method for dissecting the molecular pathways that are the basis of life, as well as for investigating and potentially rectifying faults in these pathways that result in disease. This book reviews how CRISPR-Cas9 and other genome editing techniques are advancing our understanding of development and function in the nervous system, uncovering the molecular causes of neurological disorders and providing tools for gene therapy.

Author : Jeffrey S. Chamberlain,Thomas A. Rando
Publisher : CRC Press
Page : 491 pages
File Size : 45,6 Mb
Release : 2006-02-27
Category : Medical
ISBN : 9780849374456

Get Book

Duchenne Muscular Dystrophy by Jeffrey S. Chamberlain,Thomas A. Rando Pdf

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

Author : Alan E. H. Emery
Publisher : Unknown
Page : 338 pages
File Size : 51,5 Mb
Release : 2001
Category : Medical
ISBN : UOM:39015054284123

Get Book

The Muscular Dystrophies by Alan E. H. Emery Pdf

The muscular dystrophies are an important group of inherited disorders. They are characterized by muscle wasting and weakness, but vary considerably in their clinical manifestations and severity. This text reviews our understanding of the most important of these disorders. In many instances, the genes and protein products responsible for the dystrophies have been identified and it is now possible to establish a precise diagnosis, detect preclinical cases, identify carriers and offer prenatal dianostic testing. The book goes on to describe the opportunities for management of the symptoms through respiratory care, physiotherapy and surgical correction of contracture, and examines the potential, in the future, for effective treatment utilizing the new techniques of gene and cell therapy. Professor Emery has invited chapters from the leading international experts in the field providing a unique insight into the current situation and the hopes for the future.

Author : Paula Johanson
Publisher : The Rosen Publishing Group, Inc
Page : 66 pages
File Size : 55,8 Mb
Release : 2008-08-15
Category : Self-Help
ISBN : 9781435848245

Get Book

Muscular Dystrophy by Paula Johanson Pdf

Muscular dystrophy (MD) is a genetic disorder that gradually weakens a person’s muscles. It is caused by missing or incorrect genetic information in the body’s cells. The person’s body is unable to make the proteins needed to build and maintain healthy muscles. Although there is no cure for muscular dystrophy, researchers are learning about how to prevent and treat the condition. Doctors have learned a great deal about how to improve muscle and joint function and how to slow the deterioration of muscles. Their goal is to help children, teenagers, and adults with muscular dystrophy live active and independent lives for as long as possible. This book delves into the history of the disorder, the genetic basis of MD, the main types of MD, and living with MD and suggests some coping strategies. It also explores diagnostic tests for MD, genetic research, and the promising effects of gene therapy.

Author : Gisela Gaina
Publisher : BoD – Books on Demand
Page : 117 pages
File Size : 53,9 Mb
Release : 2020-12-23
Category : Technology & Engineering
ISBN : 9781839684746

Get Book

Muscular Dystrophy by Gisela Gaina Pdf

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.

Author : Byron Arthur Kakulas,John McC. Howell,Allen D. Roses
Publisher : Raven Press (ID)
Page : 328 pages
File Size : 48,5 Mb
Release : 1992
Category : Medical
ISBN : UOM:39015025287692

Get Book

Duchenne Muscular Dystrophy by Byron Arthur Kakulas,John McC. Howell,Allen D. Roses Pdf

This timely volume assesses recent progress in the search for a curative treatment for Duchenne muscular dystrophy (DMD). Leading international experts highlight important advances in our understanding of dystrophinopathies, discuss the use of animal models in developing therapies for DMD, and analyze experiments in humans and animals on myoblast transfer and direct gene transfer therapy. The papers presented and the critical discussions among the contributing authors define the major problems that need to be addressed in future research. The opening chapters review the latest studies on the pathological features of Duchenne and Becker muscular dystrophy. the ultrastructural localization and functions of dystrophin, and normal and abnormal dystrophin gene expression. The contributors then describe and compare two different animal models of Duchenne muscular dystrophy: the xmd dog, which exhibits skeletal muscular dystrophy similar to human DMD, and the mdx mouse, which shares the genetic dystrophin defect underlying the disease, but does not develop severe progressive skeletal myopathy. Full consideration is given to the relevance of both animal models in defining the mechanisms of muscular dystrophy and evaluating therapeutic strategies. A major portion of the book focuses on experiments with myoblast transfer in DMD patients and in animals. Noted investigators detail methods for determining the extent to which transplanted non-dystrophic myoblasts survive within the host, fuse with dystrophic muscle, correct the dystrophin defect, arrest the pathological changes in the host muscle, and prevent or slow the progressive loss of muscle function. The contributors also explore newapproaches to direct gene transfer in DMD and assess the relative feasibility of donor myoblast transfer, direct gene transfer, and patient myoblast-mediated gene transfer. This volume offers much-needed direction to researchers developing therapies for Duchenne and Becker muscular dystrophy. It will also be a valuable stimulus to scientists investigating other muscular dystrophies and genetic diseases.

Author : Basil T. Darras,H. Royden Jones Jr.,Monique M. Ryan,Darryl C. De Vivo
Publisher : Elsevier
Page : 1156 pages
File Size : 52,8 Mb
Release : 2014-12-03
Category : Medical
ISBN : 9780124171275

Get Book

Neuromuscular Disorders of Infancy, Childhood, and Adolescence by Basil T. Darras,H. Royden Jones Jr.,Monique M. Ryan,Darryl C. De Vivo Pdf

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

Author : National Institute of Neurological Diseases and Stroke
Publisher : Unknown
Page : 26 pages
File Size : 50,5 Mb
Release : 1971
Category : Muscular dystrophy
ISBN : MINN:31951D03894033U

Get Book

Muscular Dystrophy by National Institute of Neurological Diseases and Stroke Pdf

Author : Peter K. Law
Publisher : R G Landes Company
Page : 164 pages
File Size : 44,5 Mb
Release : 1994-01-01
Category : Medical
ISBN : 1879702762

Get Book

Myoblast Transfer by Peter K. Law Pdf

Author : Charlotte J Sumner,Sergey Paushkin,Chien-Ping Ko
Publisher : Academic Press
Page : 506 pages
File Size : 54,9 Mb
Release : 2016-10-24
Category : Medical
ISBN : 9780128036860

Get Book

Spinal Muscular Atrophy by Charlotte J Sumner,Sergey Paushkin,Chien-Ping Ko Pdf

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

Author : George Karpati,David Hilton-Jones,Robert C. Griggs
Publisher : Cambridge University Press
Page : 800 pages
File Size : 51,9 Mb
Release : 2001-07-12
Category : Medical
ISBN : 0521650623

Get Book

Disorders of Voluntary Muscle by George Karpati,David Hilton-Jones,Robert C. Griggs Pdf

Rewritten and redesigned, this remains the one essential text on the diseases of skeletal muscle.

Author : Rika Maruyama,Toshifumi Yokota
Publisher : Springer Nature
Page : 568 pages
File Size : 42,6 Mb
Release : 2022-11-18
Category : Medical
ISBN : 9781071627723

Get Book

Muscular Dystrophy Therapeutics by Rika Maruyama,Toshifumi Yokota Pdf

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.

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Publisher : National Academies Press
Page : 93 pages
File Size : 49,9 Mb
Release : 2019-12-01
Category : Medical
ISBN : 9780309495844

Get Book

Advancing Gene-Targeted Therapies for Central Nervous System Disorders by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders Pdf

On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.