Antisense-mediated exon skipping: A therapeutic strategy for titin-based dilated cardiomyopathy

EMBO Molecular Medicine

Volumn 7, Issue 5, 2015, Pages 562-576

  Gramlich, Michael ^a,b   Pane, Luna Simona ^c   Zhou, Qifeng ^a   Chen, Zhifen ^c   Murgia, Marta ^d,e   Schötterl, Sonja ^a   Goedel, Alexander ^c   Metzger, Katja ^a   Brade, Thomas ^c   Parrotta, Elvira ^c,f   Schaller, Martin ^a   Gerull, Brenda ^g   Thierfelder, Ludwig ^h   Aartsma Rus, Annemieke ⁱ   Labeit, Siegfried ^j   Atherton, John J ^k   Mcgaughran, Julie ^l   Harvey, Richard P ^b,m   Sinnecker, Daniel ^c   Mann, Matthias ^d   Laugwitz, Karl Ludwig ^c,n   Gawaz, Meinrad Paul ^a   Moretti, Alessandra ^c,n   more..

^a UNIVERSITY OF TÜBINGEN   (Germany)

^b VICTOR CHANG CARDIAC RESEARCH INSTITUTE   (Australia)

^c TECHNICAL UNIVERSITY OF MUNICH   (Germany)

^d MAX PLANCK INSTITUTE OF BIOCHEMISTRY   (Germany)

^e UNIVERSITY OF PADOVA   (Italy)

^f UNIVERSITY MAGNA GRAECIA OF CATANZARO   (Italy)

^g UNIVERSITY OF CALGARY   (Canada)

^h MAX DELBRÜCK CENTER FOR MOLECULAR MEDICINE   (Germany)

ⁱ LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^j UNIVERSITY OF HEIDELBERG   (Germany)

^k UNIVERSITY OF QUEENSLAND   (Australia)

^l ROYAL BRISBANE AND WOMEN'S HOSPITAL   (Australia)

^m UNIVERSITY OF NEW SOUTH WALES   (Australia)

ⁿ DZHK GERMAN CENTRE FOR CARDIOVASCULAR RESEARCH   (Germany)

more..

Author keywords

Dilated cardiomyopathy; Exon skipping; Induced pluripotent stem cells; Titin

Indexed keywords

ALPHA ACTININ; ANGIOTENSIN II; ANTISENSE OLIGONUCLEOTIDE; CONNECTIN; ISOPRENALINE; MYOSIN HEAVY CHAIN; MYOSIN HEAVY CHAIN ALPHA; MYOSIN HEAVY CHAIN BETA; SMALL NUCLEAR RNA; TROPONIN T;

ADULT; ARTICLE; AUTOSOMAL DOMINANT INHERITANCE; CASE REPORT; CELLULAR DISTRIBUTION; CONGESTIVE CARDIOMYOPATHY; CONTROLLED STUDY; CYTOPLASM; DOWN REGULATION; ECHOCARDIOGRAPHY; ELECTRON MICROSCOPY; EMBRYO; ENHANCER REGION; EXON; EXON SKIPPING; FEMALE; FLUORESCENCE IN SITU HYBRIDIZATION; FRAMESHIFT MUTATION; HEART LEFT VENTRICLE EJECTION FRACTION; HEART MUSCLE CELL; HUMAN; HUMAN CELL; IMMUNOCYTOCHEMISTRY; IMMUNOFLUORESCENCE; LENTIVIRUS INFECTION; MASS SPECTROMETRY; MIDDLE AGED; MUSCLE FIBRIL; NONHUMAN; OPEN READING FRAME; PHENOTYPE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEOMICS; QUANTITATIVE ANALYSIS; REAL TIME POLYMERASE CHAIN REACTION; SARCOMERE; SIGNAL TRANSDUCTION; TRANSIENT TRANSFECTION; UPREGULATION; WILD TYPE; ANIMAL; CARDIAC MUSCLE CELL; CARDIOMYOPATHY, DILATED; CELL CULTURE; CYTOLOGY; DISEASE MODEL; DRUG EFFECTS; GENE EXPRESSION REGULATION; GENE THERAPY; GENETICS; METABOLISM; MOUSE; PATHOPHYSIOLOGY; PHYSIOLOGY; PROCEDURES; TRANSGENIC MOUSE;

ANIMALS; CARDIOMYOPATHY, DILATED; CELLS, CULTURED; CONNECTIN; CYTOLOGICAL TECHNIQUES; DISEASE MODELS, ANIMAL; EXONS; FRAMESHIFT MUTATION; GENE EXPRESSION REGULATION; GENETIC THERAPY; HUMANS; MICE; MICE, TRANSGENIC; MYOCYTES, CARDIAC; MYOFIBRILS; OLIGONUCLEOTIDES, ANTISENSE;

EID: 84929043720     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.15252/emmm.201505047     Document Type: Article

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