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Science Translational Medicine, ISSN 1946-6234, 04/2018, Volume 10, Issue 437, p. eaan0713
Duchenne muscular dystrophy (DMD) is a lethal hereditary muscle disease caused by mutations in the gene encoding the muscle protein dystrophin. These mutations... 
MEDICINE, RESEARCH & EXPERIMENTAL | EFFICACY | PRO051 | BODYWIDE | MOUSE | RESTORATION | PHASE-2 | MUTATIONS | PATHOLOGY | DELIVERY | CELL BIOLOGY
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
Science Translational Medicine, ISSN 1946-6234, 04/2014, Volume 6, Issue 230, pp. 230fs14 - 230fs14
Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage... 
MEDICINE, RESEARCH & EXPERIMENTAL | BODYWIDE | DUCHENNE MUSCULAR-DYSTROPHY | MICE | EXPRESSION | DELIVERY | CELL BIOLOGY | Genetic Therapy | Dystrophin - genetics | Dystrophin - therapeutic use | Muscular Dystrophies - therapy | Humans | Exons - genetics | Clinical Trials as Topic | Muscular Dystrophies - genetics | Index Medicus
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