Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy

Dilated cardiomyopathy is the second most common cause for heart failure with no cure except a high-risk heart transplantation. Approximately 30% of patients harbor heritable mutations which are amenable to CRISPR-based gene therapy. However, challenges related to delivery of the editing complex and...

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Detalles Bibliográficos
Autores principales: Grosch, Markus, Schraft, Laura, Chan, Adrian, Küchenhoff, Leonie, Rapti, Kleopatra, Ferreira, Anne-Maud, Kornienko, Julia, Li, Shengdi, Radke, Michael H., Krämer, Chiara, Clauder-Münster, Sandra, Perlas, Emerald, Backs, Johannes, Gotthardt, Michael, Dieterich, Christoph, van den Hoogenhof, Maarten M. G., Grimm, Dirk, Steinmetz, Lars M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10287752/
https://www.ncbi.nlm.nih.gov/pubmed/37349314
http://dx.doi.org/10.1038/s41467-023-39352-1

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