| 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 |
|
| 8 weeks |
| 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  |