Fig. 7: Base editing mice Pcsk9 in vivo using PNLM-pcABE.
From: Protein-nucleic acid language model-assisted design of precise and compact adenine base editor
a Schematic diagram of delivering LNPs for in vivo editing of Pcsk9 in mice. The splice donor sequence of exon 1 of the mouse Pcsk9 gene was targeted by PNLM-pcABE. The blue sequence area represents the Exon1 of Pcsk9, and the yellow represents the Exon2. The splice donor site “GT” is shown in red, and the neighboring adenine is in green. Blood was collected on the 2nd day before injection and on the 7th, 14th, and 21st days after injection, respectively. b The A-to-G editing efficiency of Pcsk9 in mice 3-weeks after the delivery of LNPs packaged with ABE8e / PNLM-pcABE and sgRNA. Data are mean ± s.d. (n = 3 different mice) c The normalized ratio of A6 / A4 A-to-G base editing efficiency of ABE8e and PNLM-pcABE at the Pcsk9 site in Fig. 7b. Data are mean ± s.d. (n = 3 different mice). d, e The expression of PCSK9 and LDL-C in plasma of adult mice before and after the delivery of LNPs packaged with ABE8e/PNLM-pcABE and sgRNA. Data are mean ± s.d. (n = 3 different mice). Source data are provided as a Source data file.
