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

Autor: Grosch, M., Schraft, L., Chan, A., Küchenhoff, L., Rapti, K., Ferreira, A.M., Kornienko, J., Li, S., Radke, M.H., Krämer, C., Clauder-Münster, S., Perlas, E., Backs, J., Gotthardt, M., Dieterich, C., van den Hoogenhof, M.M.G., Grimm, D., Steinmetz, L.M.

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, challen

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=mdc______med::9320cb63ada0ccde084f07108e87431d
http://edoc.mdc-berlin.de/23544/1/23544oa.pdf

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

Autor: Grosch M; European Molecular Biology Laboratory (EMBL), Genome Biology Unit, Heidelberg, Germany.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA.; DZHK (German Center for Cardiovascular Research), Partner Site Heidelberg/Mannheim, Heidelberg, Germany., Schraft L; European Molecular Biology Laboratory (EMBL), Genome Biology Unit, Heidelberg, Germany., Chan A; Klaus Tschira Institute for Integrative Computational Cardiology, University of Heidelberg, Heidelberg, Germany., Küchenhoff L; European Molecular Biology Laboratory (EMBL), Genome Biology Unit, Heidelberg, Germany., Rapti K; Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Medical Faculty, BioQuant, University of Heidelberg, Heidelberg, Germany., Ferreira AM; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Kornienko J; European Molecular Biology Laboratory (EMBL), Genome Biology Unit, Heidelberg, Germany.; DZHK (German Center for Cardiovascular Research), Partner Site Heidelberg/Mannheim, Heidelberg, Germany., Li S; European Molecular Biology Laboratory (EMBL), Genome Biology Unit, Heidelberg, Germany., Radke MH; Translational Cardiology and Functional Genomics, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, Berlin, Germany.; German Center for Cardiovascular Research (DZHK), Partner Site Berlin, Berlin, Germany., Krämer C; Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Medical Faculty, BioQuant, University of Heidelberg, Heidelberg, Germany., Clauder-Münster S; European Molecular Biology Laboratory (EMBL), Genome Biology Unit, Heidelberg, Germany., Perlas E; Epigenetics and Neurobiology Unit, EMBL Rome, Monterotondo, Italy., Backs J; DZHK (German Center for Cardiovascular Research), Partner Site Heidelberg/Mannheim, Heidelberg, Germany.; Institute of Experimental Cardiology, University Hospital Heidelberg, Heidelberg, Germany., Gotthardt M; Translational Cardiology and Functional Genomics, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, Berlin, Germany.; German Center for Cardiovascular Research (DZHK), Partner Site Berlin, Berlin, Germany.; Department of Cardiology, Charité Universitätsmedizin Berlin, Berlin, Germany., Dieterich C; DZHK (German Center for Cardiovascular Research), Partner Site Heidelberg/Mannheim, Heidelberg, Germany.; Klaus Tschira Institute for Integrative Computational Cardiology, University of Heidelberg, Heidelberg, Germany., van den Hoogenhof MMG; DZHK (German Center for Cardiovascular Research), Partner Site Heidelberg/Mannheim, Heidelberg, Germany.; Institute of Experimental Cardiology, University Hospital Heidelberg, Heidelberg, Germany., Grimm D; DZHK (German Center for Cardiovascular Research), Partner Site Heidelberg/Mannheim, Heidelberg, Germany.; Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Medical Faculty, BioQuant, University of Heidelberg, Heidelberg, Germany.; German Center for Infection Research (DZIF), Partner Site Heidelberg, Heidelberg, Germany., Steinmetz LM; European Molecular Biology Laboratory (EMBL), Genome Biology Unit, Heidelberg, Germany. Lars.Steinmetz@stanford.edu.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA. Lars.Steinmetz@stanford.edu.; DZHK (German Center for Cardiovascular Research), Partner Site Heidelberg/Mannheim, Heidelberg, Germany. Lars.Steinmetz@stanford.edu.; Stanford Genome Technology Center, Palo Alto, CA, USA. Lars.Steinmetz@stanford.edu.

Publikováno v: Nature communications [Nat Commun] 2023 Jun 22; Vol. 14 (1), pp. 3714. Date of Electronic Publication: 2023 Jun 22.