Article

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

2022-12-15

Abstract excerpt

Dilated cardiomyopathy (DCM) is the second most common cause for heart failure with no cure except a high-risk heart transplantation. Approximately 30% of DCM patients harbor heritable mutations which are amenable to CRISPR-based gene therapy 1 . However, challenges related to delivery of the editing complex and off-target concerns hamper the broad applicability of CRISPR agents in the heart 2 . We employed a co...

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Literature Corpus work: 78c0727f-8678-5845-8709-b316b20ea7af
DOI: 10.1101/2022.12.13.520227

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