Myoblast Transfer Therapy - Robert C. Griggs

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Résumé :
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 dollars115,000,000. 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.

Sommaire:
1 Molecular Biology of Myogenesis and Regeneration.- MyoD: A Regulatory Gene of Skeletal Myogenesis.- Myoblasts, Satellite Cells, and Myoblast Transfer.- Regeneration of Skeletal Muscle Fibers after Necrosis.- Detection of Truncated Dystrophin in Fetal DMD Myotubes.- General Discussion.- 2 Myoblast/Satellite Cell Antigenicity.- Immunological Aspects of Histoincompatible Myoblast Transfer into Non-tolerant Hosts.- A Comparison of Long-term Survival of Muscle Precursor Cell Suspensions and Minced Muscle Allografts in the Non-tolerant Mouse.- N-CAM is a Target Cell Surface Antigen for the Purification of Muscle Cells for Myoblast Transfer Therapy.- Immunochemical Analyses of the Myoblast Membrane and Lineage.- General Discussion.- 3 Practical Aspects of Myoblast Implantation.- The Principles and Practice of Myoblast Transfer.- Myoblast Transfer Improves Muscle Genetics/Structure/ Function and Normalizes the Behavior and Life Span of Dystrophic Mice.- Practical Aspects of Myoblast Implantation: Investigations on Two Inherited Myopathies in Animals.- Purification and Proliferation of Human Myoblasts Isolated with Fluorescence Activated Cell Sorting.- The Proliferation and Fusion of Myoblasts In Vivo.- The DMD Gene Promoter: A Potential Role in Gene Therapy.- General Discussion.- 4 In Situ Fusion: Nuclear Domains and mRNA/Protein Migration.- Fibroblasts Fuse with Myotubes Developing in Culture.- Phenotypic and Functional Reversion of Muscular Dysgenesis by Heterotypic Fibroblast-Myotube Fusion In Vitro.- Control of Satellite Cell Proliferation.- Regeneration of Skeletal Muscle Induced by Satellite Cell Grafts.- Localization of Muscle Gene Products in Nuclear Domains: Does this Constitute a Problem for Myoblast Therapy?.- Mouse Chimeras and Genetic Rescue of Mosaic Muscle.- General Discussion.- 5 Development of Myogenic Cell Cultures.- Improved Media for Rapid Clonal Growth of Normal Human Skeletal Muscle Satellite Cells.- Retroviral Lineage Markers for Assessing Myoblast Fate In Vivo.- Myogenic Conversion of Human Non-muscle Cells for the Diagnosis and Therapy of Neuromuscular Diseases.- General Discussion.- 6 Monitoring Clinical Success: Phenotypic Transformation.- Genetic and Biochemical Determinations in the Pre-transplant Workup and in the Post-transplant Assessment Period.- Use of Quantitative Myometry in the Evaluation of Myoblast Transfer Therapy.- Clinical Issues in Myoblast Transfer.- Quantitation of Muscle Mass and Muscle Protein Synthesis Rate: Documenting a Response to Myoblast Transfer.- Plausible Structural/Functional/Behavioral/Biochemical Transformations Following Myoblast Transfer Therapy.- Mdx Mouse as Therapeutic Model System: Development and Implementation of Phenotypic Monitoring.- Golden Retriever Muscular Dystrophy: Monitoring for Success.- Molecular Markers for Myoblast Transplantation in GRMD.- The Role of the Xmd Dog in the Assessment of Myoblast Transfer Therapy.- The Dilemma of Manifesting Carriers in the Context of Myoblast Transplantation.- Immunosuppressive Therapy in Duchenne Muscular Dystrophy: Considerations for Myoblast Transfer Studies.- General Discussion.- 7 Implementation of Human Trials.- Synopsis of Proceedings of the Workshop on the Implementation of Myoblast Transfer in Humans.- Participants.