Experiment Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Experiment Condition cc47-SaCas9, Male Assay Description: Editing efficiency calculated by counting fluorescent cells (RFP) compared to DAPI stained nuclei Tissue Measured: heart Cell Type : cardiac muscle cell Measurement Type: Editing Efficiency Record ID 15000001247 Editor SauCas9 Delivery System Delivery System Subtype Guide Ai9 SaCas9 Guide B; Ai9 SaCas9 Guide A Vector AAVcc47-SaCas9-Ai9; Model Species Mouse Model Name B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J Sample Sex Male Application Method intravenous, tail vein Application Site liver and heart Dosage 1.0x10^14 vg copies/kg (vg/kg) (2.0x10^12/20g mouse) Injection Frequency once Injection Rate Injection Volume 200 microliters Days post injection 4 weeks Editor Format AAV vector encoded Antidote Id Antidote Description Measured Values Samples Size: 6   Replicate Result (Editing Efficiency) Measurement Units: % of cells Mean 2.37  1 1.93  2 3.69  3 1.08  4 3.85  5 1.3  6 2.35