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Description

Concomitant therapies and adverse events were reviewed and blood samples were collected for safety analysis and an electrocardiogram (ECG) performed

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However, the review notes that these results, while promising, remain preliminary and require confirmation in larger outcome studies

**At time of manufacturing

Comparison of the CAG-C9 and EFS-C9 lines showed that the CAG-C9 lines had higher expression of Cas9 mRNA and protein (compare Supplementary information, Fig