Article
Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy.
Nature communications - 22 Jun 2023
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
Abstract excerpt
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 off-target concerns hamper the broad applicability of CRISPR agents in the heart. We employ a combination of the...
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