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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 | Index Medicus
Journal Article
Journal Article
Nature Medicine, ISSN 1078-8956, 2011, Volume 17, Issue 6, pp. 720 - 725
Myotonic dystrophy is the most common muscular dystrophy in adults and the first recognized example of an RNA-mediated disease. Congenital myotonic dystrophy... 
MEDICINE, RESEARCH & EXPERIMENTAL | CTG REPEAT | CENTRONUCLEAR MYOPATHY | PROTEIN-KINASE | BIOCHEMISTRY & MOLECULAR BIOLOGY | MYOTUBULAR MYOPATHY | CELL BIOLOGY | SKELETAL-MUSCLE | MESSENGER-RNA | GENE | CHLORIDE CHANNEL | AMPHIPHYSIN-2 BIN1 | MUTATIONS | Cell Line | RNA-Binding Proteins - physiology | Humans | Alternative Splicing - physiology | Exons - genetics | Muscle Weakness - genetics | Protein Isoforms - physiology | Tumor Suppressor Proteins - physiology | Adaptor Proteins, Signal Transducing - physiology | Myotonic Dystrophy - physiopathology | Animals | Tumor Suppressor Proteins - genetics | Adaptor Proteins, Signal Transducing - genetics | Myotonic Dystrophy - genetics | Mice | Nuclear Proteins - physiology | Muscle Weakness - physiopathology | Nuclear Proteins - genetics | Muscle Fibers, Skeletal - physiology | RNA-Binding Proteins - metabolism | Protein Isoforms - genetics | Muscle weakness | Myotonic dystrophy | RNA | Physiological aspects | Genetic aspects | Research | Risk factors | Musculoskeletal diseases | Musculoskeletal system | Biosynthesis | Protein synthesis | Muscular dystrophy | Index Medicus | Alternative Splicing | Exons | Myotonic Dystrophy | RNA-Binding Proteins | Nuclear Proteins | Life Sciences | Adaptor Proteins, Signal Transducing | Muscle Fibers, Skeletal | Protein Isoforms | Muscle Weakness | Tumor Suppressor Proteins | Cancer | Naturvetenskap | Natural Sciences
Journal Article
Molecular Therapy, ISSN 1525-0016, 01/2017, Volume 25, Issue 1, pp. 24 - 43
Journal Article
Neuron, ISSN 0896-6273, 08/2012, Volume 75, Issue 3, pp. 437 - 450
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 8/2009, Volume 106, Issue 33, pp. 13915 - 13920
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 11/2009, Volume 106, Issue 44, pp. 18551 - 18556
Journal Article
Journal Article
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