Single-vector AAV delivery of En-ISPmu1 and donor DNA for in vivo targeted genome integration
Alessio Conci, Ilaria Bonuzzi, Matteo Ciciani, Federica Esposito, Pasquale Piccolo, Fanny Collaud, Giuseppe Ronzitti, Alberto Auricchio, Nicola Segata, Anna Cereseto
The paper describes the development of En-ISPmu1, a modified TnpB nuclease, and demonstrates its ability to achieve targeted genome integration in mouse liver with a single AAV vector.
bioRxiv · Oct 28, 2026 · PreprintBioengineering