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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
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 1/2001, Volume 98, Issue 1, pp. 42 - 47
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease arising from defects in the dystrophin gene, typically nonsense or frameshift mutations,... 
Oligoribonucleotides | Biological Sciences | Transfection | Splicing | Reverse transcriptase polymerase chain reaction | Exons | Introns | Duchenne muscular dystrophy | Fluorescence | Muscle fibers | Genetic mutation | POSITIVE FIBERS | PROTEIN | RNA | MULTIDISCIPLINARY SCIENCES | MUTATION | MUSCLE | DUCHENNE MUSCULAR-DYSTROPHY | GENE-TRANSFER | CELL | SOMATIC REVERSION SUPPRESSION | OLIGONUCLEOTIDES | Immunohistochemistry | Membrane Glycoproteins - metabolism | Dystrophin - biosynthesis | Oligoribonucleotides, Antisense - administration & dosage | Molecular Sequence Data | Base Sequence | RNA Splicing - genetics | Mice, Inbred mdx | Cytoskeletal Proteins - metabolism | RNA Precursors - metabolism | Muscles - metabolism | Muscular Dystrophy, Duchenne - therapy | Disease Models, Animal | Introns - genetics | Oligoribonucleotides, Antisense - therapeutic use | Mice, Inbred C57BL | Cells, Cultured | Exons - genetics | RNA Precursors - genetics | Injections, Intramuscular | Open Reading Frames - genetics | Reverse Transcriptase Polymerase Chain Reaction | Animals | Dystrophin - genetics | Sarcoglycans | Oligoribonucleotides, Antisense - genetics | Fluorescein | Mice | Muscular Dystrophy, Duchenne - genetics | Phosphatidylethanolamines - metabolism | Microscopy, Fluorescence | Genetic aspects | Muscular dystrophy | Genetics | Biochemistry | Mutation | Index Medicus
Journal Article
PLoS ONE, ISSN 1932-6203, 07/2013, Volume 8, Issue 7, pp. e69194 - e69194
Journal Article
Journal Article
The Journal of Cell Biology, ISSN 0021-9525, 3/2000, Volume 148, Issue 5, pp. 985 - 995
Journal Article
Stem Cells, ISSN 1066-5099, 06/2007, Volume 25, Issue 6, pp. 1364 - 1374
Journal Article