Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathy

Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathy

Abstract Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated cardiomyopathy (DCM), a heart disease characterized by ventricular dilatation, systolic dysfunction, and progressive heart failure. To date, there are no specific treatment options for DCM p...

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Bibliographic Details
Main Authors: Michael Gramlich, Luna Simona Pane, Qifeng Zhou, Zhifen Chen, Marta Murgia, Sonja Schötterl, Alexander Goedel, Katja Metzger, Thomas Brade, Elvira Parrotta, Martin Schaller, Brenda Gerull, Ludwig Thierfelder, Annemieke Aartsma‐Rus, Siegfried Labeit, John J Atherton, Julie McGaughran, Richard P Harvey, Daniel Sinnecker, Matthias Mann, Karl‐Ludwig Laugwitz, Meinrad Paul Gawaz, Alessandra Moretti
Format: Article
Language:English
Published: Springer Nature 2015-03-01
Series:EMBO Molecular Medicine
Subjects:
dilated cardiomyopathy
exon skipping
induced pluripotent stem cells
titin
Online Access:https://doi.org/10.15252/emmm.201505047

Internet

https://doi.org/10.15252/emmm.201505047

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