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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BlackWell Publishing Ltd 2015
Materias:
Research Articles
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

Internet

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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