Author: Dongsheng Duan Publisher: Springer Science & Business Media ISBN: 144191207X Category : Medical Languages : en Pages : 281
Book Description
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: Gisela Gaina Publisher: BoD – Books on Demand ISBN: 1837681554 Category : Medical Languages : en Pages : 116
Book Description
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.
Author: Rudolf Jaenisch Publisher: ISBN: 9781013268564 Category : Medical Languages : en Pages : 128
Book Description
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.
Author: Mingjie Li Publisher: Academic Press ISBN: 012809821X Category : Medical Languages : en Pages : 450
Book Description
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
Author: Jeffrey S. Chamberlain Publisher: CRC Press ISBN: 0849374456 Category : Medical Languages : en Pages : 491
Book Description
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: Basil T. Darras Publisher: Elsevier ISBN: 0124171273 Category : Medical Languages : en Pages : 1156
Book Description
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 Academies of Sciences, Engineering, and Medicine Publisher: National Academies Press ISBN: 0309670950 Category : Medical Languages : en Pages : 319
Book Description
The Social Security Administration (SSA) administers two programs that provide disability benefits: the Social Security Disability Insurance (SSDI) program and the Supplemental Security Income (SSI) program. SSDI provides disability benefits to people (under the full retirement age) who are no longer able to work because of a disabling medical condition. SSI provides income assistance for disabled, blind, and aged people who have limited income and resources regardless of their prior participation in the labor force. Both programs share a common disability determination process administered by SSA and state agencies as well as a common definition of disability for adults: "the inability to engage in any substantial gainful activity by reason of any medically determinable physical or mental impairment which can be expected to result in death or which has lasted or can be expected to last for a continuous period of not less than 12 months." Disabled workers might receive either SSDI benefits or SSI payments, or both, depending on their recent work history and current income and assets. Disabled workers might also receive benefits from other public programs such as workers' compensation, which insures against work-related illness or injuries occurring on the job, but those other programs have their own definitions and eligibility criteria. Selected Health Conditions and Likelihood of Improvement with Treatment identifies and defines the professionally accepted, standard measurements of outcomes improvement for medical conditions. This report also identifies specific, long-lasting medical conditions for adults in the categories of mental health disorders, cancers, and musculoskeletal disorders. Specifically, these conditions are disabling for a length of time, but typically don't result in permanently disabling limitations; are responsive to treatment; and after a specific length of time of treatment, improve to the point at which the conditions are no longer disabling.
Author: Fred F. Ferri Publisher: Elsevier Health Sciences ISBN: 0323679773 Category : Medical Languages : en Pages : 7615
Book Description
Significantly updated with the latest developments in diagnosis and treatment recommendations, Ferri's Clinical Advisor 2020 features the popular "5 books in 1" format to organize vast amounts of information in a clinically relevant, user-friendly manner. This efficient, intuitive format provides quick access to answers on 1,000 common medical conditions, including diseases and disorders, differential diagnoses, and laboratory tests – all reviewed by experts in key clinical fields. Updated algorithms, along with hundreds of new figures, tables, and boxes, ensure that you stay current with today's medical practice. - Contains significant updates throughout, covering all aspects of current diagnosis and treatment. - Features 27 all-new topics including chronic traumatic encephalopathy, medical marijuana, acute respiratory failure, gallbladder carcinoma, shift work disorder, radial tunnel syndrome, fertility preservation in women, fallopian tube cancer, primary chest wall cancer, large-bowel obstruction, inguinal hernia, and bundle branch block, among others. - Includes a new appendix covering Physician Quality Reporting System (PQRS) Measures. - Provides current ICD-10 insurance billing codes to help expedite insurance reimbursements. - Patient Teaching Guides for many of the diseases and disorders are included, most available in both English and Spanish versions, which can be downloaded and printed for patients.
Author: Raymond A. Huml Publisher: Springer ISBN: 3319173626 Category : Medical Languages : en Pages : 206
Book Description
This practical and concise guide offers an overview of muscular dystrophy's complicated features, treatment options and general resources. New treatments and a greater understanding of proteins and structures associated with MD are discussed along with long term patient care. Also included are clinical and developmental challenges within the current regulatory landscape and recent scientific and clinical advances. Muscular Dystrophy offers clinicians, researchers, pharmaceutical executives and patient advocacy groups an easy-to-read reference that provides the necessary perspectives of the care giver and patient.
Author: Dongsheng Duan
Author: Gisela Gaina
Author: Rudolf Jaenisch
Author: Mingjie Li
Author: Jeffrey S. Chamberlain
Author: Basil T. Darras
Author: National Academies of Sciences, Engineering, and Medicine
Author: Fred F. Ferri
Author: George Karpati
Author: Raymond A. Huml