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Author: Robert C. Griggs
Publisher: Springer Science & Business Media
ISBN: 1468458655
Category : Medical
Languages : en
Pages : 305
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Book Description
I am pleased to introduce this volume on Myoblast Transfer Therapy on behalf of the Muscular Dystrophy Association and all of its Advisory Committees. The international conference which led to this volume brought together leading basic scientists and clinical investigators for the purpose of coordinating the development of this new field in the fight against muscular dystrophy. The Muscular Dystrophy Association is the nation's most rapidly growing voluntary health agency in terms of its programs of patient care, research, and professional and public education. Success is attributable to its National Chairman, Jerry Lewis, to its effective corporate membership, and to the many physicians and scientists who give their time freely to advise on policies, to review grant applications, and to participate in meetings such as this. I should like to acknowledge a large number of other individuals to whom we are indebted: the broad segment of the American public which continually and generously supports our spectrum of services. The Muscular Dystrophy Association, next year, should raise in excess of $115,000,000. These contributions are derived from more than 10 million American families. These families are not only pledging their money but expressing their hopes that we will find answers to the tragic problem of neuromuscular disease. We are confident that the fruits of this meeting will move the frontier of research forward toward that goal.
Author: Robert C. Griggs
Publisher: Springer Science & Business Media
ISBN: 1468458655
Category : Medical
Languages : en
Pages : 305
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Book Description
I am pleased to introduce this volume on Myoblast Transfer Therapy on behalf of the Muscular Dystrophy Association and all of its Advisory Committees. The international conference which led to this volume brought together leading basic scientists and clinical investigators for the purpose of coordinating the development of this new field in the fight against muscular dystrophy. The Muscular Dystrophy Association is the nation's most rapidly growing voluntary health agency in terms of its programs of patient care, research, and professional and public education. Success is attributable to its National Chairman, Jerry Lewis, to its effective corporate membership, and to the many physicians and scientists who give their time freely to advise on policies, to review grant applications, and to participate in meetings such as this. I should like to acknowledge a large number of other individuals to whom we are indebted: the broad segment of the American public which continually and generously supports our spectrum of services. The Muscular Dystrophy Association, next year, should raise in excess of $115,000,000. These contributions are derived from more than 10 million American families. These families are not only pledging their money but expressing their hopes that we will find answers to the tragic problem of neuromuscular disease. We are confident that the fruits of this meeting will move the frontier of research forward toward that goal.
Author: Peter K. Law
Publisher: R G Landes Company
ISBN: 9781879702769
Category : Medical
Languages : en
Pages : 164
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Book Description
Author: Scott Thomas Baker
Publisher:
ISBN:
Category : Muscular dystrophy
Languages : en
Pages : 232
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Book Description
Author: Gisela Gaina
Publisher: BoD – Books on Demand
ISBN: 1839684747
Category : Technology & Engineering
Languages : en
Pages : 117
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Book Description
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: Alan E. H. Emery
Publisher:
ISBN:
Category : Medical
Languages : en
Pages : 338
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Book Description
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: Byron Arthur Kakulas
Publisher: Raven Press (ID)
ISBN:
Category : Medical
Languages : en
Pages : 328
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Book Description
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: Dongsheng Duan
Publisher: Springer Science & Business Media
ISBN: 144191207X
Category : Medical
Languages : en
Pages : 281
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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: Clayton Travis Fragall
Publisher:
ISBN:
Category : Duchenne muscular dystrophy
Languages : en
Pages :
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Book Description
[Truncated abstract] Of the 700 neuromuscular diseases currently described in man Duchenne Muscular Dystrophy (DMD) is both the most common and most severe of those diseases affecting primary muscle function. Mutations in the dystrophin gene result in the loss of dystrophin or production of a non-functional dystrophin protein, an essential element in the membrane stabilising machinery of muscle fibres. DMD is an X-linked recessive disease that affects about 1 in 3500 live male births. Males suffer almost exclusively from the crippling pathology of this disease due to their lack of a second X chromosome with a competent dystrophin gene. The dystrophin gene is one of the largest known human genes making it particularly susceptible to random mutations. In fact approximately one third of cases arise with no prior family history of the disease. There is no cure for DMD and despite decades of research only palliative treatment is available to sufferers. Myoblast Transfer Therapy (MTT) is aimed at utilising the muscles natural repair mechanism via the direct injection of donor myoblasts (muscle precursor cells), which express functional dystrophin, into the damaged, dystrophic, tissue in order to provide a cell-based gene rescue. The objective of this procedure is for the donor myoblasts, upon injection into dystrophic muscle, to migrate throughout the tissue and fuse with host myoblasts to form what is referred to as mosaic muscle fibres. Whilst numerous animal model studies and some human clinical trials have indicated the MTT approach to be feasible in principle, myoblast death and immune rejection appears to limit the practicality of this potential therapy. The rapid disappearance of donor myoblasts from transplanted muscles after MTT is one of the most controversial and significant obstacles facing research in this area. A commonly used method to detect cell survival is quantitation of the Y chromosome following transplantation of male donor cells into female hosts. This thesis presents a direct comparison between real time quantitative polymerase chain reaction (Q-PCR) and the DNA hybridisation (slot-blot) technique for quantitation of Y chromosome DNA. Results show that Q-PCR has a significantly greater linear quantitation range and is up to 40-fold more sensitive at low concentrations of male DNA, detecting as little as 1 ng of male DNA in each female Tibialis Anterior (TA) muscle. At high male DNA concentrations, accurate quantitation by Q-PCR is 2.5 times higher than the maximum possible with slot-blot. Thus Q-PCR has a greater dynamic range and is more sensitive than slot-blot analysis for the detection of donor cell engraftment in a trans-sexual transplantation model. Subsequent to these improvements in transplant quantitation experiments were conducted to examine claims in the literature that a significant loss of donor myoblasts occur in the first hour following MTT. These experiments defined the appropriate reference standard for the quantitation of donor cell survival and clearly showed that there is no significant loss of donor cells during the first hour post-transplantation ...
Author: Jeffrey S. Chamberlain
Publisher: CRC Press
ISBN: 0849374456
Category : Medical
Languages : en
Pages : 491
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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: Rika Maruyama
Publisher: Springer Nature
ISBN: 1071627724
Category : Medical
Languages : en
Pages : 568
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Book Description
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.
