In vivo base editing rescues liver pathophysiology and peroxisome dysfunction in a mouse model of Zellweger spectrum disorder
Gao XD, Presa M, Duby JE, Ryan J, Piec P-A, Hsu A, Banskota S, Jiang AY, Chen L, Newby GA, Di Pietro E, Levy JM, Steele BH, Lecordier S, Qin F, Moser AB, Xie J, Gao G, Braverman NE, Zuberi AR, Hacia JG, Lutz CM, Liu DR
Nat. Biomed. Eng (2026). https://doi.org/10.1038/s41551-026-01651-5
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 247654 | AAV N-terminus ABE8e-V106W targeting Pex1-G844D | AAV genome encoding the N-terminus of ABE8e-V106W editor and Sp sgRNA cassette |
| 247655 | AAV C-terminus ABE8e-V106W targeting Pex1-G844D | AAV genome encoding the C-terminus of ABE8e-V106W editor and Sp sgRNA cassette |
| 247667 | Lentivirus ABE8e-V106W targeting PEX1-G843D | Lentivirus genome encoding ABE8eV106W editor and Sp sgRNA cassette targeting PEX1-G843D mutation |
| 247668 | Lentivirus ABE8e-V106W with non-targeting sgRNA | Lentivirus genome encoding ABE8eV106W editor and Sp sgRNA cassette non-targeting control |