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Titel:

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

Dokumenttyp:
Journal Article; Research Support, Non-U.S. Gov't
Autor(en):
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
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 patients but heart transplantation. Here, we show the beneficial potential of reframing titin transcripts by antisense oligonucleotide (AON)-mediated exon skipping in human and murine models of DCM carrying a p...     »
Zeitschriftentitel:
EMBO Mol Med
Jahr:
2015
Band / Volume:
7
Heft / Issue:
5
Seitenangaben Beitrag:
562-76
Sprache:
eng
Volltext / DOI:
doi:10.15252/emmm.201505047
PubMed:
http://view.ncbi.nlm.nih.gov/pubmed/25759365
Print-ISSN:
1757-4676
TUM Einrichtung:
Arbeitsgruppe Tissue Engineering und Regenerative Medizin; I. Medizinische Klinik und Poliklinik (Kardiologie)
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