Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathy. Issue 5 (10th March 2015)

• Record Type: Journal Article
• Authors: Gramlich, Michael; Pane, Luna Simona; Zhou, Qifeng; Chen, Zhifen; Murgia, Marta; Schötterl, Sonja; Goedel, Alexander; Metzger, Katja; Brade, Thomas; Parrotta, Elvira; Schaller, Martin; Gerull, Brenda; Thierfelder, Ludwig; Aartsma‐Rus, Annemieke; Labeit, Siegfried; Atherton, John J; McGaughran, Julie; Harvey, Richard P; Sinnecker, Daniel; Mann, Matthias; Laugwitz, Karl‐Ludwig; Gawaz, Meinrad Paul; Moretti, Alessandra
• Is Part Of: EMBO molecular medicine. Volume 7:Issue 5(2015:May)
• Page Start: 562
• Page End: 576
• Publication Date: 2015-03-10
• Subjects: Molecular biology -- Periodicals; Medical genetics -- Periodicals; Pathology, Molecular -- Periodicals; 616.04205
• DOI: 10.15252/emmm.201505047
• Languages: English
• ISSNs: 1757-4676

Abstract

<abstract abstract-type="graphical" xml:lang="en" id="emmm201505047-abs-0003"> <title> <x xml:space="preserve">Abstract</x> </title> <p>Truncating mutations in the giant sarcomeric protein titin (TTN) are a major cause for inherited forms of dilated cardiomyopathy (DCM). Antisense oligonucleotides (AON)‐mediated exon skipping is shown here to be a promising strategy as potential treatment option for DCM. <graphic position="anchor" mimetype="image" xlink:href="ark:/27927/pgjk6vsbbd" orientation="portrait" xlink:type="simple" xmlns:xlink="http://www.w3.org/1999/xlink" /></p> </abstract>