Experiment Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Experiment Condition AAVcc47-mCherry Assay Description: Native fluorescence was quantified using Image J. For each animal, 6 images were taken from 2 sections and used to calculate corrected total cell fluorescence using the formula: Integrated Density – (Area of selected image X Mean fluorescence of background readings). Tissue Measured: skeletal muscle tissue Measurement Type: Delivery Efficiency Record ID 15000000346 Editor Delivery System Delivery System Subtype Guide Vector AAVcc47-mCherry; Model Species Mouse Model Name C57BL/6 Application Method intravenous Application Site Dosage 5e13 vg/kg Injection Frequency 1X Injection Rate Injection Volume 200 microliters Days post injection 4 weeks Editor Format AAV vector encoded Antidote Id Antidote Description Measured Values Samples Size: 3   Replicate Result (Delivery Efficiency) Measurement Units: Corrected Total Fluorescence Mean 2794378491.38  1 3084055640.12133  2 2800961697.68067  3 2498118136.33733

Representative image of AAVcc47-mCherry delivery in skeletal muscle