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Article

Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration

22 Dec 2022

Abstract excerpt

Ensuring genome safety during gene editing is crucial for clinical translation of the high-efficient CRISPR-Cas9 toolbox. Therefore, the undesired events including chromosomal translocations, vector integrations, and large deletions arising during therapeutic gene editing remain to be adequately addressed or tackled in vivo. Here, we apply CRISPR-Cas9TX in comparison to CRISPR-Cas9 to target Vegfa for the...

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PMID: 36550137
DOI: 10.1038/s41467-022-35640-4

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