Single-AAV CRISPR editing of skeletal muscle in non-human primates with NanoCas, an ultracompact nuclease
Benjamin J. Rauch, Aaron DeLoughery, Renan B. Sper, Sean Chen, Sophia Yunanda, Megan Masnaghetti, Ning Chai, Jason Ching‐Yao Lin, Alexander Neckelmann, Ymer M. Bjornson, David Páez-Espino, Alyssa Sancio, Christopher Schmitt, Clarissa Scholes, Ria Shah, , Lucas B. Harrington
This paper develops NanoCas, a compact CRISPR nuclease, and demonstrates its efficacy in editing non-liver tissues in non-human primates.
bioRxiv · Jan 29, 2025 · PreprintBioengineering