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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: 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: Scott Thomas Baker
Publisher:
ISBN:
Category : Muscular dystrophy
Languages : en
Pages : 232
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Book Description
Author: Peter K. Law
Publisher:
ISBN: 9781606928172
Category : Gene mapping
Languages : en
Pages : 140
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Book Description
Human Myoblast Genome Therapy (HMGT) is a platform technology of cell transplantation, nuclear transfer, and tissue engineering. Myoblasts are differentiated, immature cells destined to become muscles. Myoblasts cultured from muscle biopsy survive, develop and function to revitalise degenerative muscles upon transplantation. Transplant injury activates regeneration of host myofibers that fuse with the injected myoblasts, sharing their nuclei in a common gene pool of the syncytium. Thus, through nuclear transfer and complementation, human genome can be transferred into muscles of genetically-ill patients to achieve phenotype repair. Myoblasts are safe and efficient universal gene transfer vehicles endogenous to muscles that constitute 50% of the body. Myoblasts fuse among themselves to form new myofibres. Patients take only 2-month cyclosporine to immunosuppress allograft rejection because myofibres do not express MHC-1 antigens. The first correction of human gene defect was published in the Lancet on July 14, 1990 when the therapeutic protein dystrophin was found in the myoblast-injected muscle of a Duchenne muscular dystrophy (DMD) patient. Results over 280 HMGT procedures on MD subjects in the past 15 years demonstrated absolute safety. Myoblast-injected DMD muscles showed improved histology. Strength increase at 18 months post-operatively averaged 123%. FDA-approved clinical trials progressed unto Phase III in USA with direct cost recovery. Heart muscle degeneration is the leading cause of human debilitation and death.
Author: Jason David White
Publisher:
ISBN:
Category : Leukemia inhibitory factor
Languages : en
Pages : 538
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Book Description
Author: Peter Law
Publisher: Eurekah.Com Incorporated
ISBN: 9781587062803
Category : Medical
Languages : en
Pages :
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Book Description
Author: Johnny Huard
Publisher: Springer Science & Business Media
ISBN: 1461221269
Category : Medical
Languages : en
Pages : 294
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Book Description
This book has been written in response to the many physicians and scien tists working on the development of biological approaches to providing therapies for many orthopaedic disorders as well as to improving the healing of many tissues of the musculoskeletal system. The first goal of this book is to make the language compatible between the bench scientist and the clinician working in orthopaedic and sports medicine in order to cover specific areas of the orthopaedic discipline where the treatment can be improved and/or changed by the advancements in molecular medicine. Advancements in molecular biology, which encompass the study of the genetic basis of disease, have produced new diagnostic methods and drug therapies for genetic diseases and acquired disorders. The growth in the understanding of human genetics has also led to the initiation of many human gene therapy experiments. Although many approved therapeutic clinical trials using this new technology have been performed in the last ten years, the first clinical trial using this technology in the area of orthopaedics was performed at the University of Pittsburgh.
Author: R.P. Lanza
Publisher: Springer Science & Business Media
ISBN: 9400901658
Category : Science
Languages : en
Pages : 280
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Book Description
Cell and tissue transplantation is one of the most exciting and rapidly expanding areas in medicine. This first edition of the Yearbook of Cell and Tissue Transplantation summarizes the latest advances in this revolutionary field, including developments in tissue engineering and transplantation of hybrid organs and tissues, while reviewing those data which, while not new, add to the usefulness of this work as a comprehensive reference. The justification for yearbooks is greater than ever as we approach the third millennium, overwhelmed with information. In view of the important and rapid changes occurring in this area, a new edition of this yearbook will appear periodically. The editors' careers at Harvard Medical School guarantee the quality of this book. The authors, too, are uniformly drawn from the highest rank of an unusually dedicated and heterogeneous professional group. from the Foreword by Thomas E. Starzl, Honorary President, The Cell Transplant Society: `No major topic in the global field is left uncovered ... the result will be a feast for those already well informed, and a life raft for those who are not.'
Author: Stefan-M. Pulst
Publisher: Contemporary Neurology
ISBN: 9780195129755
Category : Medical
Languages : en
Pages : 496
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Book Description
Neurogenetics is intended for any physician or scientist who manages patients with inherited diseases of the nervous system. It presents the clinical phenotypes of the most commonly inherited neurologic diseases, and their molecular pathogenesis, followed by a description of the appropriate tests to be used in diagnosis. Two introductory chapters familiarize the nongeneticist with medical genetic terminology and molecular genetic techniques useful in the analysis of genetic disease and genetic testing. Subsequent chapters examine major neurologic disorders caused by single defects, as well as disease phenotypes such as Alzheimer disease or amyotrophic lateral sclerosis which may be caused by defects in single genes, but may also be seen as sporadic diseases. The genetic components of other common neurologic disorders, such as epilepsy, multiple sclerosis, migraine, and stroke are all covered in detail. The final chapter discusses genetic counseling of symptomatic and pre-symptomatic individuals. Throughout, chapters discuss genotype/phenotype correlations and, where appropriate, animal models for inherited human neurologic diseases. Several chapters are devoted to recently discovered diseases caused by unstable DNA repeats. Special emphasis is placed on conveying how DNA testing can be applied to the daily practice of geneticists and neurologists.
