Muscle Gene Therapy
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Muscle Gene Therapy
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Author : Dongsheng Duan
language : en
Publisher: Springer Science & Business Media
Release Date : 2009-11-26
Muscle Gene Therapy written by Dongsheng Duan and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2009-11-26 with Medical categories.
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.
Muscle Gene Therapy
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Author : Dongsheng Duan
language : en
Publisher: Humana Press
Release Date : 2011-08-25
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 2011-08-25 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.
Studies On Retroviral Mediated Gene Transfer In Skeletal Muscle For Gene Therapy Of Duchenne Muscular Dystrophy
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Author : Ariberto Fassati
language : en
Publisher:
Release Date : 1997
Studies On Retroviral Mediated Gene Transfer In Skeletal Muscle For Gene Therapy Of Duchenne Muscular Dystrophy written by Ariberto Fassati and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1997 with categories.
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.
Improved Plasmid Based Muscle Gene Therapy
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Author : Yasamine Modarresi
language : en
Publisher:
Release Date : 2016
Improved Plasmid Based Muscle Gene Therapy written by Yasamine Modarresi 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.
Muscular dystrophies are a group of inherited disorders characterized by progressive muscle weakness, and degeneration. One approach to treatment would be the replacement of the deficient protein via gene therapy. For effective gene therapy, both efficiency of gene delivery and stable expression of the transferred gene are important factors. Our goal was to determine which of the following mammalian expression vectors would be more useful (more stable) for muscle gene therapy; pAcGFP1-C1 and pEPito. We were also interested in switching/substituting both vectors' CMV promoter/enhancer region with the muscle specific promoter, Desmin (DES), to increase their stability for muscle gene therapy. This was accomplished by transfecting C2C12 myotubes with the aforementioned vectors. Both vectors showed relatively continuous GFP expression. Myotubes transfected with pEPito continued to express GFP till day 8. Cells transfected with pACGFP1-C1 also showed continuous GFP expression till day 6. Our results show that both vectors are promising candidates for gene therapy in muscle cells as they maintained stable gene expression of the GFP reporter gene for at least 6 days. Further studies should be done in order to determine the maximum duration that the myotubes would be able to maintain the plasmids and show continuous expression. Future studies could be done to assess stability of these vectors in mice which may lead to future gene therapy trials for muscle disorders and improving gene therapy strategies for other disorders.
Muscular Dystrophy
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Author : Paula Johanson
language : en
Publisher: The Rosen Publishing Group, Inc
Release Date : 2008-08-15
Muscular Dystrophy written by Paula Johanson and has been published by The Rosen Publishing Group, Inc this book supported file pdf, txt, epub, kindle and other format this book has been release on 2008-08-15 with Self-Help categories.
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.
Gene Therapy For Muscular Dystrophy Evaluation Of A Muscle Specific Promoter For Adenovirus Mediated Gene Transfer
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Author :
language : en
Publisher:
Release Date : 1997
Gene Therapy For Muscular Dystrophy Evaluation Of A Muscle Specific Promoter For Adenovirus Mediated Gene Transfer written by and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1997 with categories.
Genome Editing In Neurosciences
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Author : Rudolf Jaenisch
language : en
Publisher: Springer
Release Date : 2018-08-11
Genome Editing In Neurosciences written by Rudolf Jaenisch and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-08-11 with Medical categories.
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.
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.
Duchenne Muscular Dystrophy
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Author : Byron Arthur Kakulas
language : en
Publisher: Raven Press (ID)
Release Date : 1992
Duchenne Muscular Dystrophy written by Byron Arthur Kakulas and has been published by Raven Press (ID) this book supported file pdf, txt, epub, kindle and other format this book has been release on 1992 with Medical categories.
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.