Abstract
Experiments in mice have supported the idea of treating Duchenne muscular dystrophy (DMD) by implanting normal muscle precursor cells into dystrophin-deficient muscles. However, similar experiments on DMD patients have had little success. Gene therapy for DMD, by introducing dystrophin constructs via retroviral or adenoviral vectors, has been shown to be possible in the mouse, but the efficiency and safety aspects of this technique will have to be carefully examined before similar experiments can be attempted in man. Direct injection of dystrophin cDNA constructs into mdx muscles has given rise to very low levels of dystrophin and this may be a possibility for the treatment of heart muscle.
Publication types
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Animals
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Cell Transplantation*
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Child
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Combined Modality Therapy
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DNA, Complementary / administration & dosage
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DNA, Complementary / genetics
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DNA, Complementary / therapeutic use
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Dystrophin / biosynthesis
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Dystrophin / genetics*
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Forecasting
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Genetic Therapy*
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Genetic Vectors
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Humans
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Male
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Mice
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Mice, Mutant Strains
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Mice, Transgenic
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Muscles / cytology*
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Muscular Dystrophies / genetics
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Muscular Dystrophies / therapy*
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Muscular Dystrophy, Animal / genetics
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Muscular Dystrophy, Animal / therapy
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RNA / administration & dosage
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RNA / therapeutic use
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Recombinant Fusion Proteins / biosynthesis
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Recombinant Fusion Proteins / genetics
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Stem Cell Transplantation*
Substances
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DNA, Complementary
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Dystrophin
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Recombinant Fusion Proteins
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RNA