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Molecular Therapy, ISSN 1525-0016, 02/2012, Volume 20, Issue 2, pp. 443 - 455
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Journal Article
Journal Article
Journal of Biological Chemistry, ISSN 0021-9258, 07/2015, Volume 290, Issue 29, pp. 17784 - 17795
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
Journal Article
Human Gene Therapy, ISSN 1043-0342, 09/2017, Volume 28, Issue 9, pp. 737 - 746
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Molecular Therapy, ISSN 1525-0016, 06/2007, Volume 15, Issue 6, pp. 1160 - 1166
Journal Article