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Journal Article
Journal Article
Nature, ISSN 0028-0836, 08/2012, Volume 488, Issue 7409, pp. 111 - 115
Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases that involve RNA or protein gain-of-function effects. In the hereditary... 
CTG REPEAT | 3' END | PROTEIN | MESSENGER-RNA | MULTIDISCIPLINARY SCIENCES | GENE-EXPRESSION | TOXIC RNA | MODIFIED ANTISENSE OLIGONUCLEOTIDE | PHOSPHOROTHIOATE OLIGONUCLEOTIDES | MOUSE MODELS | TRINUCLEOTIDE REPEAT | Humans | Muscle, Skeletal - metabolism | RNA, Messenger - metabolism | RNA, Untranslated - genetics | Gene Knockdown Techniques | RNA - genetics | Myotonic Dystrophy - pathology | Base Sequence | Muscle, Skeletal - drug effects | Oligonucleotides, Antisense - therapeutic use | RNA, Messenger - antagonists & inhibitors | Mice, Inbred mdx | Myotonic Dystrophy - genetics | Disease Models, Animal | RNA - metabolism | Oligonucleotides, Antisense - pharmacology | Myotonin-Protein Kinase | RNA - antagonists & inhibitors | Mice, Inbred C57BL | RNA, Messenger - genetics | Gene Silencing | Protein-Serine-Threonine Kinases - genetics | Mice, Transgenic | Myotonic Dystrophy - therapy | Transcriptome - drug effects | Transcriptome - genetics | Myotonic Dystrophy - physiopathology | Animals | Oligonucleotides, Antisense - genetics | Cell Nucleus - genetics | RNA, Long Noncoding | Alleles | Trinucleotide Repeat Expansion - genetics | Mice | Mice, Inbred BALB C | Cell Nucleus - drug effects | Ribonuclease H - metabolism | Myotonic dystrophy | RNA | Oligonucleotides | Physiological aspects | Development and progression | Genetic aspects | Health aspects
Journal Article
Drug Discovery Today, ISSN 1359-6446, 12/2018, Volume 23, Issue 12, pp. 2013 - 2022
Journal Article
Journal Article
Neuromuscular Disorders, ISSN 0960-8966, 2010, Volume 20, Issue 8, pp. 479 - 492
Summary Cardiac disease is a common clinical manifestation of neuromuscular disorders, particularly of muscular dystrophies. Heart muscle cells as well as... 
Neurology | Treatment | Arrhythmia | Cardiomyopathy | Sudden death | Muscular dystrophy | CONDUCTION-SYSTEM | PROXIMAL MYOTONIC MYOPATHY | CLINICAL SPECTRUM | LINKED DILATED CARDIOMYOPATHY | NEUROSCIENCES | NUCLEAR-MEMBRANE | CLINICAL NEUROLOGY | CAUSE AUTOSOMAL-DOMINANT | LEFT-VENTRICULAR DYSFUNCTION | SUDDEN CARDIAC DEATH | LAMIN A/C GENE | SARCOGLYCAN GENE | Cardiomyopathy, Dilated - pathology | Muscular Dystrophies - therapy | Myofibrils - pathology | Humans | Muscular Dystrophy, Facioscapulohumeral - genetics | Muscular Dystrophies - genetics | Myotonic Dystrophy - pathology | Muscular Dystrophies, Limb-Girdle - genetics | Myotonic Dystrophy - genetics | Genetic Diseases, X-Linked - genetics | Muscular Dystrophies, Limb-Girdle - pathology | Cardiomyopathy, Dilated - genetics | Muscular Dystrophy, Emery-Dreifuss - genetics | Muscular Dystrophies - congenital | Muscular Dystrophy, Emery-Dreifuss - pathology | Muscular Dystrophy, Duchenne - pathology | Myocardium - pathology | Muscular Dystrophies - complications | Muscular Dystrophies - pathology | Muscular Dystrophy, Duchenne - complications | Heart Diseases - etiology | Genetic Diseases, X-Linked - pathology | Muscular Dystrophy, Facioscapulohumeral - pathology | Heterozygote | Muscular Dystrophy, Duchenne - genetics | Heart Diseases - pathology | Heart failure | Cardiology | Genetic aspects
Journal Article