Genome-wide off target detection of Un1Cas12f1 in mouse models
Hypercompact Cas12f1 were demonstrated with eukaryotic nuclease activity showing great potential as AAV-based gene therapy system. Our present study provided definitive results for developing Cas12f1-based therapy for in vivo diseases treatment with tyrosineamia mouse model. We conducted genome-wide off-target analysis using PEM-seq for gene knockout with Cas12f1. PEM-seq detected no off-target editing events for Cas12f1-mediated gene editing.