| Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. | 
            | cc47-Cre Pos control, Male | 
            
                | Editing efficiency calculated by counting fluorescent cells (RFP) compared to DAPI stained nuclei | 
            
                | liver | 
            
            
                | hepatocyte | 
            
            
                | Editing Efficiency | 
            | 15000001292 | 
            | 
 | 
            
                | Cre recombinase | 
                |  | 
                |  | 
                |  | 
            | AAVcc47-Cre; | 
            | 
 | 
    | Mouse | 
        | B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J | 
            | 
 | 
            
            
            | Male | 
            
            | 
 | 
                | intravenous, tail vein | 
                | liver and heart | 
                | 1.0x10^14 vg copies/kg (vg/kg) (2.0x10^12/20g mouse) | 
                | once | 
                |  | 
                | 200 microliters | 
                | 4 weeks | 
                | AAV vector encoded | 
                |  | 
                |  | 
                | 
 | 
            
            
                | Measured Values | 
            
                | Samples Size: | 2 | 
            |  | 
            
                
                
            
            
                 
                     
                     
                     
                    
            
                
                | Mean | 2 of 2 Significant red cells  | 
            
            
                
                | 1 | Significant red cells  | 
            
            
                
                | 2 | Significant red cells  |