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Journal Article
Nature Communications, ISSN 2041-1723, 07/2017, Volume 8, Issue 1, pp. 16105 - 16105
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 04/2017, Volume 114, Issue 16, pp. 4213 - 4218
Duchenne muscular dystrophy (DMD) is a lethal genetic disorder caused by an absence of the dystrophin protein in bodywide muscles, including the heart.... 
Cardiac Purkinje fibers | Dystrophic dog model | Peptide-conjugated morpholinos | Duchenne muscular dystrophy | Exon skipping | MUSCLE PATHOLOGY | MULTIDISCIPLINARY SCIENCES | DMD GENE | CARDIOMYOPATHY | BODYWIDE | dystrophic dog model | peptide-conjugated morpholinos | MDX52 MICE | OLIGONUCLEOTIDES | RESCUE | exon skipping | LABORATORY-ANIMALS | cardiac Purkinje fibers | EXPRESSION | EXONS 45-55 | Genetic Therapy | Muscular Dystrophy, Animal - genetics | Exons | Male | Muscle, Skeletal - metabolism | Muscular Dystrophy, Animal - complications | Muscular Dystrophy, Duchenne - complications | Cardiomyopathies - etiology | Cardiomyopathies - therapy | Cell-Penetrating Peptides - pharmacology | Animals | Dogs | Morpholinos - pharmacology | Female | Muscular Dystrophy, Animal - therapy | Muscle, Skeletal - pathology | Muscular Dystrophy, Duchenne - genetics | Muscular Dystrophy, Duchenne - therapy | Dystrophin - metabolism | Disease Models, Animal | Heart | Physiological aspects | Health aspects | Conduction | Intravenous administration | Peptides | Toxicity | Cardiomyopathy | Effects | Muscular dystrophy | Fibers | Oligomers | Nerve conduction | Arginine | Duchenne's muscular dystrophy | Degeneration | Polymers | Heart diseases | Dystrophin | EKG | Cardiac muscle | Abnormalities | Muscles | Skeletal muscle | Purkinje fibers | Myocardium | Dystrophy | Index Medicus | Biological Sciences
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 06/2014, Volume 124, Issue 6, pp. 2651 - 2667
Duchenne muscular dystrophy (DMD) is caused by mutations in the gene encoding dystrophin, which results in dysfunctional signaling pathways within muscle.... 
MEDICINE, RESEARCH & EXPERIMENTAL | SKELETAL-MUSCLE | MDX MOUSE | MESSENGER-RNAS | CENTRONUCLEAR MYOPATHY | GLYCOPROTEIN COMPLEX | C2C12 MYOBLAST | TUMOR-SUPPRESSOR | CELL-PROLIFERATION | MYOBLAST FUSION | PROTEIN-KINASE B | Muscular Dystrophy, Animal - genetics | Up-Regulation | Humans | Male | MicroRNAs - metabolism | Muscle, Skeletal - metabolism | Muscle Fibers, Skeletal - metabolism | Muscular Dystrophy, Animal - pathology | Guanine Nucleotide Exchange Factors - metabolism | Base Sequence | Mice, Inbred mdx | Muscular Dystrophy, Animal - metabolism | Proto-Oncogene Proteins c-akt - metabolism | Cell Line | Guanine Nucleotide Exchange Factors - genetics | Signal Transduction | Mice, Inbred C57BL | PTEN Phosphohydrolase - metabolism | Mice, Transgenic | Muscular Dystrophy, Duchenne - pathology | Nerve Tissue Proteins - genetics | Sequence Homology, Nucleic Acid | Nerve Tissue Proteins - metabolism | Carrier Proteins - genetics | Animals | Carrier Proteins - metabolism | Muscle Fibers, Skeletal - pathology | Mice | MicroRNAs - genetics | Muscular Dystrophy, Duchenne - metabolism | Muscle, Skeletal - pathology | Muscular Dystrophy, Duchenne - genetics | MicroRNA | Cellular signal transduction | Genetic aspects | Diagnosis | Properties | Genetic regulation | Muscular dystrophy | Muscular system | Mutation | Rodents | Index Medicus | Abridged Index Medicus
Journal Article
Science, ISSN 0036-8075, 12/2004, Volume 306, Issue 5702, pp. 1796 - 1799
Most mutations in the dystrophin gene create a frameshift or a stop in the mRNA and are associated with severe Duchenne muscular dystrophy. Exon skipping that... 
Proteins | Legs | Messenger RNA | Reverse transcriptase polymerase chain reaction | Exons | Duchenne muscular dystrophy | Muscles | Small nuclear RNA | Reports | Muscle fibers | Genetic mutation | SKELETAL-MUSCLE | HEMOPHILIA-B | MULTIDISCIPLINARY SCIENCES | MDX MOUSE MUSCLE | MUTATION | DUCHENNE MUSCULAR-DYSTROPHY | GENE-TRANSFER | EXPRESSION | PRE-MESSENGER-RNA | DELIVERY | VECTOR | Dependovirus - genetics | Genetic Therapy | Muscular Dystrophy, Animal - genetics | Muscular Dystrophy, Animal - physiopathology | Muscle, Skeletal - metabolism | RNA, Messenger - metabolism | Muscular Dystrophy, Animal - pathology | RNA Splicing | Transfection | Muscular Dystrophy, Duchenne - physiopathology | RNA, Small Nuclear - genetics | Mice, Inbred mdx | Muscular Dystrophy, Animal - therapy | Muscular Dystrophy, Duchenne - therapy | Dystrophin - metabolism | Oligonucleotides, Antisense - pharmacology | Introns | RNA, Messenger - genetics | Muscular Dystrophy, Duchenne - pathology | Muscle, Skeletal - physiology | Muscle Fibers, Skeletal - immunology | Animals | Muscle Contraction | Dystrophin - genetics | Muscle Fibers, Skeletal - pathology | Mice | Muscle, Skeletal - pathology | Muscular Dystrophy, Duchenne - genetics | Mutation | RNA, Small Nuclear - metabolism | Genetic Vectors | Usage | Care and treatment | Genetic aspects | Gene therapy | Exon (Molecular genetics) | Ribonucleic acid--RNA | Genes | Rodents | Muscular dystrophy | Index Medicus
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 12/2012, Volume 109, Issue 50, pp. 20614 - 20619
Duchenne muscular dystrophy (DMD) is a devastating genetic muscular disorder of childhood marked by progressive debilitating muscle weakness and wasting, and... 
Artificial satellites | Plasmids | Duchenne muscular dystrophy | Stem cells | Muscles | Electroporation | Muscle fibers | Skeletal muscle | Hypertrophy | Myoblasts | Noncanonical Wnt signaling | REGENERATION | MULTIDISCIPLINARY SCIENCES | MOUSE | SUSCEPTIBILITY | INJURY | SATELLITE STEM-CELLS | MDX MICE | DAMAGE | noncanonical Wnt signaling | SKELETAL-MUSCLE | CONTRACTION | PATHWAY | skeletal muscle | Genetic Therapy | Muscular Dystrophy, Animal - genetics | Muscular Dystrophy, Animal - drug therapy | Muscular Dystrophy, Animal - physiopathology | Humans | Male | RNA, Messenger - metabolism | Muscular Dystrophy, Animal - pathology | Gene Knockdown Techniques | Wnt Proteins - genetics | Mice, Inbred mdx | Plasmids - genetics | Electrochemotherapy | MEF2 Transcription Factors | Myogenic Regulatory Factors - genetics | Signal Transduction | Mice, Inbred C57BL | RNA, Messenger - genetics | Myogenic Regulatory Factors - metabolism | Wnt Proteins - therapeutic use | Regeneration - physiology | Animals | Muscle Contraction - physiology | Muscle, Skeletal - physiopathology | Mice | Wnt Proteins - physiology | Muscle, Skeletal - pathology | Plasmids - administration & dosage | Physiological aspects | Care and treatment | Rapamycin | Health aspects | Muscular dystrophy | Signal transduction | Genetic disorders | Rodents | Cells | Index Medicus | Biological Sciences
Journal Article
Journal Article