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Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy
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 b...
Autores principales: | 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BlackWell Publishing Ltd
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4492817/ https://www.ncbi.nlm.nih.gov/pubmed/25759365 http://dx.doi.org/10.15252/emmm.201505047 |
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