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2006, Neurological disease and therapy, ISBN 0824723252, Volume 79., xxii, 461
Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of childhood and currently stands as an incurable condition. This... 
Treatment | Muscular Dystrophy, Duchenne | Duchenne muscular dystrophy | therapy | Medical Genetics | Neurology | Neuroscience | Muscular Dystrophy, Duchenne - therapy
Book
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 09/2018, Volume 115, Issue 39, pp. E9182 - E9191
In gene therapy for Duchenne muscular dystrophy there are two potential immunological obstacles. An individual with Duchenne muscular dystrophy has a genetic... 
Gene replacement therapy | Mdx/mTRG2 mice | DNA plasmid | Duchenne muscular dystrophy | Microdystrophin | STEM-CELLS | microdystrophin | TRANSGENIC MDX MICE | MULTIDISCIPLINARY SCIENCES | MUSCULAR-DYSTROPHY | PYRUVATE-KINASE PK | mdx/mTRG2 mice | SOMATIC REVERSION SUPPRESSION | gene replacement therapy | ADENOASSOCIATED VIRAL VECTORS | MYELIN BASIC-PROTEIN | SKELETAL-MUSCLE | MOUSE MODEL | IN-VIVO | DNA - pharmacokinetics | Dependovirus - genetics | Muscle Strength - genetics | Male | Muscle Strength - immunology | DNA - genetics | Plasmids - pharmacology | Animals | Dystrophin - genetics | Genetic Vectors - pharmacology | Muscular Dystrophy, Duchenne - immunology | Mice, Inbred mdx | Plasmids - genetics | Dystrophin - immunology | Mice | Muscular Dystrophy, Duchenne - metabolism | Muscular Dystrophy, Duchenne - genetics | Muscular Dystrophy, Duchenne - therapy | Dystrophin - metabolism | Disease Models, Animal | Genetic Therapy - methods | Care and treatment | Plasmids | Genetic aspects | Gene therapy | Health aspects | Methods | Multiple sclerosis | Antibodies | Viruses | Diabetes mellitus (insulin dependent) | Immunity | Muscular dystrophy | Proteins | Immunology | Technology | Duchenne's muscular dystrophy | Plasmid DNA | Dystrophin | Deoxyribonucleic acid--DNA | Recombinant | Myelin | Diabetes mellitus | Muscles | Gene expression | Insulin | Immunogenicity | Muscle function | Mutation | Dystrophy | Index Medicus | mdx | Biological Sciences | PNAS Plus | mTRG2 mice
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Molecular Therapy, ISSN 1525-0016, 02/2012, Volume 20, Issue 2, pp. 443 - 455
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Annals of Neurology, ISSN 0364-5134, 06/2009, Volume 65, Issue 6, pp. 667 - 676
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Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 12/2015, Volume 7, Issue 12, pp. 1513 - 1528
Journal Article