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 |
|
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  |