Download or read book Myoblast Transfer Therapy written by Robert C. Griggs and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 305 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Myoblast Transfer written by Peter K. Law and published by R G Landes Company. This book was released on 1994-01-01 with total page 164 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Pioneering Human Myoblast Genome Therapy written by Peter K. Law and published by . This book was released on 2009 with total page 140 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Duchenne Muscular Dystrophy written by Byron Arthur Kakulas and published by Raven Press (ID). This book was released on 1992 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Functional Tissue Engineering written by Farshid Guilak and published by Springer Science & Business Media. This book was released on 2003-07-09 with total page 434 pages. Available in PDF, EPUB and Kindle. Book excerpt: -Softcover reprint of a successful hardcover reference (370 copies sold) -Price to be accessible to the rapidly increasing population of students and investigators in the field of tissue engineering -Chapters written by well-known researchers discuss issues in functional tissue engineering as well as provide guidelines and a summary of the current state of technology

Download or read book The Muscular Dystrophies written by Alan E. H. Emery and published by . This book was released on 2001 with total page 338 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Disorders of Voluntary Muscle written by George Karpati and published by Cambridge University Press. This book was released on 2001-07-12 with total page 800 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rewritten and redesigned, this remains the one essential text on the diseases of skeletal muscle.

Download or read book Stem Cell Therapy written by Erik V. Greer and published by Nova Publishers. This book was released on 2006 with total page 248 pages. Available in PDF, EPUB and Kindle. Book excerpt: Among the many applications of stem cell research are nervous system diseases, diabetes, heart disease, auto-immune diseases as well as Parkinson's disease, end-stage kidney disease, liver failure, cancer, spinal cord injury, multiple sclerosis, Parkinson's disease, and Alzheimer's disease. Stem cells are self-renewing, unspecialised cells that can give rise to multiple types all of specialised cells of the body. Stem cell research also involves complex ethical and legal considerations since they involve adult, foetal tissue and embryonic sources. This new book brings together leading research from throughout the world in this frontier field.

Download or read book Handbook of Stem Cell Therapy written by Khawaja Husnain Haider and published by Springer Nature. This book was released on 2022-11-07 with total page 1527 pages. Available in PDF, EPUB and Kindle. Book excerpt: The handbook comprehensively reviews the therapeutic potential of stem cells and stem cell secretome-based cell-free strategies in regenerative medicine. The chapters in section I and section II respectively discuss the diverse applications of mesenchymal stem cells and non- mesenchymal stem cells, including skeletal myoblasts, endothelial progenitor cells, adipose tissue-derived stem cells, induced pluripotent stem cells, and neuronal stem cells in myocardial repair, inflammatory bowel disease, cognitive deficits, wound healing, retinal disorders, and COVID-19. The subsequent chapters in section IIIprimarily focused on the fast-emerging cell-free therapy approach in regenerative medicine for tissue repair and regeneration. These chapters review the impact of stem cell-derived secretome on various biological processes such as angiogenesis, neurogenesis, tissue repair, immunomodulation, musculoskeletal pathologies, wound healing, anti-fibrotic, and anti-tumorigenesis for tissue maintenance and regeneration.Lastly, section IV summarizes miscellaneous aspects of cell-based therapy, includingthe treatment advantages, opportunities, and shortcomings in stem cell-based therapy, potentially helping to refine future studies and translate them from experimental to clinical studies. Moreover, this section also has chapters on cancer stem cells as novel targets in cancer therapeutics. This Major Reference Book (MRW) is a valuable resource for researchers involved in stem cell research to understand the multifaceted therapeutic applications of stem cells and their derivative secretome in regenerative medicine.​

Download or read book Duchenne Muscular Dystrophy written by Alan E. H. Emery and published by OUP Oxford. This book was released on 2015-02-19 with total page 320 pages. Available in PDF, EPUB and Kindle. Book excerpt: Duchenne Muscular Dystrophy, an inherited and progressive muscle wasting disease, is one of the most common single gene disorders found in the developed world. In this fourth edition of the classic monograph on the topic, Alan Emery and Francesco Muntoni are joined by Rosaline Quinlivan, Consultant in Neuromuscular Disorders, to provide a thorough update on all aspects of the disorder. Recent understanding of the nature of the genetic defect responsible for Duchenne Muscular Dystrophy and isolation of the protein dystrophin has led to the development of new theories for the disease's pathogenesis. This new edition incorporates these advances from the field of molecular biology, and describes the resultant opportunities for screening, prenatal diagnosis, genetic counselling and from recent pioneering work with anti-sense oligonucleotides, the possibility of effective RNA therapy. Although there is still no cure for the disorder, there have been significant developments concerning the gene basis, publication of standards of care guidelines, and improvements in management leading to significantly longer survival, particularly with cardio-pulmonary care. The authors also investigate other forms of pharmacological, cellular and gene therapies. Duchenne Muscular Dystrophy will be essential reading not only for scientists and clinicians, but will also appeal to therapists and other professionals involved in the care of patients with muscular dystrophy.

Download or read book Somatic Gene Therapy written by P.L. Chang and published by CRC Press. This book was released on 2018-01-17 with total page 313 pages. Available in PDF, EPUB and Kindle. Book excerpt: As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.

Download or read book Muscle Transplantation written by G. Freilinger and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 431 pages. Available in PDF, EPUB and Kindle. Book excerpt: It was a long and troublesome history but an inspiring and excltmg one too-from the time of Zielonko, a Russian pathologist who in 1874 first transplanted muscles, to the present day microneurovascular muscle graft. Today we know that for functional recovery of muscle tissue vascularisation and neurotisation are of equal importance. With the establishment of microsurgery of small vessels and nerves, its application for myopias tic operations has gained increasing interest. It was my intention to bring together leading authorities from all over the world in the field of muscle transplantation. Open questions and unsolved problems had to be discussed between research workers in the basic medical sciences and our clinical colleagues; in this way progress can best be accomplished. This publication is the result of the "Vienna Muscle Symposium" (June 13-14, 1980). It gives an up-to-date viewpoint and should act as a stimulus to all who are interested in this particular field. I wish to express my sincere thanks to those who have contributed to this publication, to the two co-editors J. Holle and B. M. Carlson for their effective cooperation, and to Springer-Verlag Wien for their prompt and most efficient work.

Download or read book Gene Transfer in the Cardiovascular System written by Keith L. March and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 552 pages. Available in PDF, EPUB and Kindle. Book excerpt: The goal of gene transfer is protein expression. a process brought about by the insertion of a gene coding for a foreign protein into target cells resulting in the synthesis of the foreign protein For gene therapy, a tmnsferred therapeutic gene must be expressed at a level beneficial for the patient. This chapter provides an introductory overview of the rapidly evolving field of non-viral approaches for gene delivery to rnarnrnalian cells. Although currently there are fewer ongoing clinical trials using non-viral approaches than those using viral based systems, the number of non-viral trials is increasing. The long range goal of some research groups is the development of a genetically engineered artificial virus targeted to specific cells in the human body. An arurual conference, organized by Cambridge Healthtech Institute entitled "Artificial Self-Assembling Systems for Gene Transfer", brings together researchers interested in this field [1]. Assembly of an artificial virus is very complex; other research groups aim to develop simpler delivery systems consisting of a plasmid combined with delivery agents. Viral-based systems are very successful for gene delivery, but despite their successes, viral-based systems have some geneml limitations and system-specific limitations. When employing a viml-based system, the following limitations should be considered: • size limitation of the inserted gene due to packaging constraints (e. g. adenovirus, retrovirus) . • potential tumorigenesis (e. g. retrovirus) • potential for insertional mutagenesis (greater than plasmid based systems) • potential imrnunogenicity (e. g.

Download or read book Dystrophin written by Susan C. Brown and published by Cambridge University Press. This book was released on 1997-05-13 with total page 360 pages. Available in PDF, EPUB and Kindle. Book excerpt: The absence of dystrophin is the underlying cause of Duchenne muscular dystrophy. This is the first book to address the structure, function, and distribution of dystrophin and its associated proteins in muscle and brain, rather than concentratiing primarily on the disease. It covers an exciting and rapidly expanding field that promises to have important and widespread implications for many aspects of cell biology.

Download or read book IPSC Derived Progenitors written by Alexander Birbrair and published by Elsevier. This book was released on 2021-11-12 with total page 318 pages. Available in PDF, EPUB and Kindle. Book excerpt: "[A]ddresses how induced pluripotent stems cells can be differentiated into distinct progenitors. Progenitors are often the first step to making more differentiating cell types. This volume addresses a variety of iPSC-derived progenitors, such as neural stem cells, craniofacial mesenchymal progenitors, astrocyte progenitors, mesothelial progenitors, keratinocyte progenitors, bone progenitors, chondrocyte progenitors, dental pulp stem cells, nephron progenitors, mesenchymal stem cells, hematopoietic stem cells, and cancer stem cells. The volume is written for researchers and scientists in stem cell therapy, cellular and molecular biology, and regenerative medicine and is contributed by world-renowned authors in the field"--Page 4 of cover.

Download or read book Muscle Regeneration written by Alexander Mauro and published by Raven Press (ID). This book was released on 1979 with total page 582 pages. Available in PDF, EPUB and Kindle. Book excerpt: