| Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. |
| cc47-SaCas9, Female |
| Fluorescent imaging for tdTomato+ cells |
| ovary |
| Editing Efficiency |
| 15000001290 |
|
| SauCas9 |
|
|
| Ai9 SaCas9 Guide B; Ai9 SaCas9 Guide A |
| AAVcc47-SaCas9-Ai9; |
|
| Mouse |
| B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J |
|
| 8 weeks |
| Female |
|
| 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: | 5 |
| |
| Mean |
1 Multiple red cells; 2 No red cells; 1 A few red cells; 1 Significant number of red cells  |
| 1 |
Multiple red cells  |
| 2 |
Significant number of red cells  |
| 3 |
No red cells  |
| 4 |
No red cells  |
| 5 |
A few red cells  |