Muscle Gene Therapy PDF Download
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Author | : Dongsheng Duan |
Publisher | : Springer Science & Business Media |
Total Pages | : 281 |
Release | : 2009-11-26 |
Genre | : Medical |
ISBN | : 144191207X |
Download Muscle Gene Therapy Book in PDF, ePub and Kindle
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 |
Total Pages | : 382 |
Release | : 2011-08-25 |
Genre | : Medical |
ISBN | : 9781617379833 |
Download Muscle Gene Therapy Book in PDF, ePub and Kindle
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 | : Alan E. H. Emery |
Publisher | : |
Total Pages | : 338 |
Release | : 2001 |
Genre | : Medical |
ISBN | : |
Download The Muscular Dystrophies Book in PDF, ePub and Kindle
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 | : Rudolf Jaenisch |
Publisher | : |
Total Pages | : 128 |
Release | : 2020-10-08 |
Genre | : Medical |
ISBN | : 9781013268564 |
Download Genome Editing in Neurosciences Book in PDF, ePub and Kindle
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 | : Jeffrey S. Chamberlain |
Publisher | : CRC Press |
Total Pages | : 351 |
Release | : 2006-02-27 |
Genre | : Medical |
ISBN | : 1040073670 |
Download Duchenne Muscular Dystrophy Book in PDF, ePub and Kindle
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.
Author | : |
Publisher | : |
Total Pages | : |
Release | : 1997 |
Genre | : |
ISBN | : |
Download Gene Therapy for Muscular Dystrophy, Evaluation of a Muscle-specific Promoter for Adenovirus-mediated Gene Transfer Book in PDF, ePub and Kindle
Author | : Paula Johanson |
Publisher | : The Rosen Publishing Group, Inc |
Total Pages | : 66 |
Release | : 2008-08-15 |
Genre | : Self-Help |
ISBN | : 1435848241 |
Download Muscular Dystrophy Book in PDF, ePub and Kindle
Muscular dystrophy (MD) is a genetic disorder that gradually weakens a persons muscles. It is caused by missing or incorrect genetic information in the bodys cells. The persons 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 | : Ariberto Fassati |
Publisher | : |
Total Pages | : 240 |
Release | : 1997 |
Genre | : |
ISBN | : |
Download Studies on Retroviral-mediated Gene Transfer in Skeletal Muscle for Gene Therapy of Duchenne Muscular Dystrophy Book in PDF, ePub and Kindle
Author | : George Karpati |
Publisher | : Cambridge University Press |
Total Pages | : 800 |
Release | : 2001-07-12 |
Genre | : Medical |
ISBN | : 9780521650625 |
Download Disorders of Voluntary Muscle Book in PDF, ePub and Kindle
Rewritten and redesigned, this remains the one essential text on the diseases of skeletal muscle.
Author | : Jeffrey S. Chamberlain |
Publisher | : CRC Press |
Total Pages | : 491 |
Release | : 2006-02-27 |
Genre | : Medical |
ISBN | : 0849374456 |
Download Duchenne Muscular Dystrophy Book in PDF, ePub and Kindle
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