Experiment Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Experiment Condition Arm1a: BI28 Neuron Specific Promoter, Experimental Animals, Females Assay Description: Identification of of GFP-positive nuclei (green) Tissue Measured: brain Cell Type : neuron Measurement Type: Delivery Efficiency Record ID 15000001451 Editor SaCas9 Delivery System Delivery System Subtype Guide L1-modified; L2-modified Vector BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2; BI28:AAV-GFAP-SaCas9-WPRE3-pA; Model Species Mouse Model Name B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J Application Method Application Site brain Dosage 0.25E+11 viral genomes/gram for each virus Injection Frequency once Injection Rate Injection Volume 5 microliters/gram Days post injection 4 weeks Editor Format ssDNA, packaged in two AAVs with neuron-specific promoter Antidote Id Antidote Description Measured Values Samples Size: 5   Replicate Result (Delivery Efficiency) Measurement Units: Signal Mean 5 of 5 present  1 Present  2 Present  3 present  4 present  5 present