Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV Vectors
Raed Ibraheim, Phillip W.L. Tai, Aamir Mir, Nida Javeed, Jiaming Wang, Tomás Rodríguez, Suk Namkung, Samantha J. Nelson, Eraj Shafiq Khokhar, Esther Mintzer, Stacy A. Maitland, Zexiang Chen, Yueying Cao, Emmanouela Tsagkaraki, Scot A. Wolfe, , 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.
Nature Communications · Nov 1, 2021Peer-reviewedMolecular Biology