Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration
Undesired chromosomal translocations, vector integrations, and large deletions remain a problem for therapeutic gene editing in vivo. Here, the authors compare the CRISPR-Cas9TX variant with CRISPR-Cas9 and show elimination of chromosomal translocations and reduction of AVV integration when targetin...
Main Authors: | , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Portfolio
2022-12-01
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Series: | Nature Communications |
Online Access: | https://doi.org/10.1038/s41467-022-35640-4 |