Therapeutic Knock-in Genome Editing Using Single AAV Vectors in Mouse Models of Inherited Liver Disease
Khishigjargal Batjargal, Tomoki Togashi, Yuji Kashiwakura, Nemekhbayar Baatartsogt, Kosuke Tsuchida, Takahiro Sato, Morisada Hayakawa, Kiwako Tsukida, Kazuhiro Muramatsu, Atsushi Hoshino, Osamu Nureki, 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.
bioRxiv · Jul 30, 2025 · PreprintBioengineering