Experiment Independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Experiment Condition 306-S10 Assay Description: On-target editing analyzed by confocal imaging of whole mouse cochlea for direct fluorescence RFP (tdTomato) compared to Hoeschst stained nuclei. Off-target editing analysis: peripheral tissues were fixed, cryosectioned and imaged directly for RFP (tdTomato). Presence or absence of signal above background was recorded for each tissue assessed. For 306-O12B, On-target N=6; Off-target N=5 (2 males, 3 females); for 306-S10, On-target N=6; Off-target N=1 (female); and for Uninjected Control, On-target and Off-target N=2. Tissue Measured: skin of body Measurement Type: Editing Efficiency Record ID 15000003264 Editor CleanCap® Cas9 mRNA (5moU) Delivery System 306-S10 Delivery System Subtype Nanoparticle Guide sg298 Vector Model Species Mouse Model Name B6.Cg-Gt(ROSA)26Sor^tm14(CAG-tdTomato)Hze/J Application Method canalostomy injection Application Site inner ear Dosage Injection Frequency once Injection Rate Injection Volume 1 microliter Days post injection 6 days Editor Format Lipid Nanoparticle Antidote Id Antidote Description Measured Values Samples Size: 0   Replicate Result (Editing Efficiency) Measurement Units: Signal Mean 0 of 1 present  1 absent