Exploring Prime Editing strategies for genetic correction of a Ceroid Neuronal Lipofuscinosis type 2 disease causing variant
Heshadi Primrose Primrose Mandalawatta, Rajendra KC, Kirsten A. Fairfax, Alex William Hewitt
This study uses prime editing to correct a common TPP1 mutation in cultured cells and eVLPs, achieving on-target editing efficiencies of up to 27.36% and 12.6% in vivo.
bioRxiv · Oct 2, 2025 · PreprintMolecular Biology