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Open AccessSafeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration
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 integratio...
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Open AccessComprehensive assessment of miniature CRISPR-Cas12f nucleases for gene disruption
Because of their small size, the recently developed CRISPR-Cas12f nucleases can be effectively packaged into adeno-associated viruses for gene therapy. However, a systematic evaluation of the editing outcomes ...
