Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV Vectors
Raed Ibraheim, Phillip W.L. Tai, Aamir Mir…·Erik J. Sontheimer
This paper develops single-AAV vectors for precise in vivo genome editing using Nme2Cas9 with HDR templates or multiple sgRNAs, showing efficacy in treating HT-I and MPS-I mouse models.