Ontology highlight
ABSTRACT:
SUBMITTER: Yin J
PROVIDER: S-EPMC9780342 | biostudies-literature | 2022 Dec
REPOSITORIES: biostudies-literature
Nature communications 20221222 1
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 treatment of age-related macular degeneration (AMD) disease in a mouse model. AAV delivery ...[more]