BioengineeringbioRxivPreprint
Therapeutic Knock-in Genome Editing Using Single AAV Vectors in Mouse Models of Inherited Liver Disease
Khishigjargal Batjargal, Tomoki Togashi, Yuji Kashiwakura…·Tsukasa Ohmori
This paper develops a single AAV vector system for liver-targeted gene knock-in therapy in mouse models of inherited liver diseases, achieving efficient therapeutic gene insertion without germline transmission.
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