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Navigating the prime editing strategy to treat cardiovascular genetic disorders in transforming heart health.

Seyed Younes HosseiniRahul MallickPetri MäkinenSuo Ctc Bladder Committee
Published in: Expert review of cardiovascular therapy (2024)
The ideal PE for the heart should be tissue-specific, regulatable, less immunogenic, high transducing, and safe. However, low efficiency, sup-optimal PE architecture, the large size of required elements, the unclear role of transcriptomics on the process, unpredictable off-target effects, and its context-dependency are subjects that need to be considered. It is also of great importance to see how beneficial or detrimental cell cycle or epigenomic modifier is to bring changes into cardiac cells. The PE delivery is challenging due to the size, multi-component properties of the editors and liver sink.
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