Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases.
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DOI: 10.1172/jci.insight.128863
Abstract: Non-integrative AAV-mediated gene therapy in the liver is effective in adult patients, but faces limitations in pediatric settings due to episomal DNA loss during hepatocyte proliferation. Gene targeting is a promising approach by permanently modifying… read more here.
Keywords: aav mediated; gene targeting; coupling aav; mice ... See more keywords