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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Bibliographic Details
Published in:	Nature communications Vol. 14; no. 1; p. 3714
Main Authors:	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.
Format:	Journal Article
Language:	English
Published:	London Nature Publishing Group UK 22-06-2023 Nature Publishing Group Nature Portfolio
Subjects:	13/106 13/51 38/23 42/44 45/100 45/41 45/91 631/208/1516 631/208/4041 631/443/592/75/74 64/60 Animal models Animals Cardiac muscle Cardiomyocytes Cardiomyopathy Cardiomyopathy, Dilated - genetics Cardiomyopathy, Dilated - metabolism Cardiomyopathy, Dilated - therapy Congestive heart failure Coronary artery disease CRISPR Dilated cardiomyopathy Editing Gene Editing Gene sequencing Gene therapy Genomes Heart diseases Heart failure Heart transplantation Humanities and Social Sciences Localization Mice multidisciplinary Muscles Mutation Myocardium - metabolism Myocytes, Cardiac - metabolism RNA-Binding Proteins - genetics RNA-Binding Proteins - metabolism Science Science (multidisciplinary) Skeletal muscle Transplantation Vectors (Biology) Whole genome sequencing
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