Dilated cardiomyopathy is the second most common cause for heart failure with an inherited form due to mutations of genes such as RBM20. The project team proposes a new avenue for correcting disease-causing mutations in RBM20, by using base editors enabling the efficient repair of mutations in non-dividing cells. The team wants to optimize crucial parameters in mice to attain the highest editing efficiency and specificity in heart muscle cells. The goal is to avoid deleterious off-target mutations in other cell types. Therefore, the team will establish a new method to quantify the base editing efficacy in single cells of the heart and evaluate the benefits of base editing on disease progression. This sets new grounds for the use of base editors in clinical trials as genetic tools to treat inherited heart conditions.

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