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International Journal of Cancer, ISSN 0020-7136, 10/2016, Volume 139, Issue 7, pp. 1648 - 1657
Journal Article
Journal Article
Annals of Neurology, ISSN 0364-5134, 01/2015, Volume 77, Issue 1, pp. 75 - 99
Journal Article
Molecular Cancer Research, ISSN 1541-7786, 06/2015, Volume 13, Issue 6, pp. 1009 - 1021
Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 07/2013, Volume 5, Issue 7, pp. 1128 - 1145
Exon skipping mediated by antisense oligoribonucleotides (AON) is a promising therapeutic approach for genetic disorders, but has not yet been evaluated for... 
cardiac myosin‐binding protein‐C | hypertrophic cardiomyopathy | alternative splicing | exon skipping | antisense oligoribonucleotide | Hypertrophic cardiomyopathy | Alternative splicing | Antisense oligoribonucleotide | Cardiac myosin-binding protein-C | Exon skipping | MEDICINE, RESEARCH & EXPERIMENTAL | BINDING-PROTEIN-C | RESTORES DYSTROPHIN EXPRESSION | UBIQUITIN-PROTEASOME SYSTEM | cardiac myosin-binding protein-C | SPLICE DONOR SITE | DILATED CARDIOMYOPATHY | CARDIAC GENE-TRANSFER | DUCHENNE MUSCULAR-DYSTROPHY | MUTATIONS | ANTISENSE OLIGONUCLEOTIDES | Genetic Therapy | Alternative Splicing | Exons | Humans | Oligoribonucleotides, Antisense - administration & dosage | RNA, Small Nuclear - administration & dosage | RNA, Small Nuclear - genetics | Myocardium - metabolism | HEK293 Cells | Adenoviridae - genetics | Cardiomyopathy, Hypertrophic - therapy | Heart - physiopathology | RNA, Small Nuclear - therapeutic use | Cardiomyopathy, Hypertrophic - genetics | Oligoribonucleotides, Antisense - therapeutic use | Transduction, Genetic | Hypertrophy, Left Ventricular - prevention & control | RNA, Messenger - genetics | Myocardium - pathology | Gene Knock-In Techniques | Carrier Proteins - genetics | Animals | Cardiomyopathy, Hypertrophic - physiopathology | Oligoribonucleotides, Antisense - genetics | Mice | Mutation | Cardiomyopathy, Hypertrophic - pathology | Protein Isoforms - genetics | Cardiomyopathy | Health aspects | Heart diseases | Analysis | Life Sciences | Human health and pathology | Cardiology and cardiovascular system
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
Journal Article
Journal Article
Cell Reports, ISSN 2211-1247, 09/2016, Volume 17, Issue 1, pp. 261 - 274
Journal Article