Duchenne Muscular Dystrophy: Current Knowledge, Treatment, and Future Prospects
| Autor: | Paula C. Demacio, Peter N. Ray, W. Biggar, Daniel J. Stevens, Henry J. Klamut |
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| Rok vydání: | 2002 |
| Předmět: |
Male
musculoskeletal diseases congenital hereditary and neonatal diseases and abnormalities mdx mouse Cell Transplantation Duchenne muscular dystrophy Bioinformatics Germline Dystrophin Glycoprotein complex medicine Animals Humans Orthopedics and Sports Medicine Muscular dystrophy Muscle Skeletal Gene biology business.industry Genetic Therapy General Medicine medicine.disease Muscular Dystrophy Duchenne Cytoskeletal Proteins biology.protein Surgery business Minigene |
| Zdroj: | Clinical Orthopaedics and Related Research. 401:88-106 |
| ISSN: | 0009-921X |
| Popis: | The cloning of the dystrophin gene has led to major advances in the understanding of the molecular genetic basis of Duchenne, Becker, and other muscular dystrophies associated with mutations in genes encoding members of the dystrophin-associated glycoprotein complex. The recent introduction of pharmaceutical agents such as prednisone has shown great promise in delaying the progression of Duchenne muscular dystrophy but there remains a need to develop more long-term therapeutic interventions. Knowledge of the nature of the dystrophin gene and the glycoprotein complex has led many researchers to think that somatic gene replacement represents the most promising approach to treatment. The potential use of this strategy has been shown in the mdx mouse model of Duchenne muscular dystrophy, where germ line gene transfer of either a full-length or a smaller Becker-type dystrophin minigene prevents necrosis and restores normal muscle function. |
| Databáze: | OpenAIRE |
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