A compact base editor rescues AATD-associated liver and lung disease in mouse models.
Gao J, Bamidele N, Pires-Ferreira D, Zheng C, Destefano A, Cheng H, Tang Q, Cao Y, Xie J, Gao G, Gruntman A, Sontheimer E, Flotte TR, Xue W
Mol Ther. 2025 Nov 5;33(11):5817-5828. doi: 10.1016/j.ymthe.2025.08.040.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 256758 | PiZ ABE | for human piz allele editing |