Experiment Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Experiment Condition Arm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Males Assay Description: Quantitative assessment of tdTomato-positive cells per DAPI-stained nucleus in a specific brain region Tissue Measured: cerebral cortex Cell Type : astrocyte Measurement Type: Editing Efficiency Record ID 15000001454 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 astrocyte-specific promoter Antidote Id Antidote Description Measured Values Samples Size: 6   Replicate Result (Editing Efficiency) Measurement Units: % Area Mean 38.67  1 35.0  2 45.9  3 33.3  4 41.8  5 29.7  6 46.3